Past Event

2022 MIT Health Science Technologies Conference

The Future of Cell & Gene Therapies

April 12, 2022
2022 MIT Health Science Technologies Conference
Conference

Location

Boston Marriott Cambridge
50 Broadway
Cambridge, MA 02142

Education Partner

MIT Professional Education log

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 


Overview

Spring 2022 ILP's in-person conferences will be open to fully vaccinated individuals only, excepting those individuals who have a medical condition or religious exemption. Conference attendees will use an application developed at MIT -- Tim Tickets -- to grant campus access or scan into an event.


The pharmaceutical industry is under growing pressure from patent expirations of major drugs, cost-constrained health care systems, and a demanding regulatory environment. A focus for the industry is to tackle these challenges while increasing the output and quality of cost-effective, new medicines without incurring unsustainable R&D cost. Through new technology and new approaches there is an aspiration to make such improvements while also bringing ever-improved therapeutics to patients faster. In this conference, we will lay out many of the challenges the industry is facing and explore potential solutions coming from academic research and startup endeavors, from early discovery through manufacturing technologies, to clinical studies and beyond.


REGISTRATION FEE

Registration is closed, onsite registration will be a available at the conference

  • ILP Member: Complimentary
  • General Public: $1,350 
  • Current MIT Faculty/Staff/Student: Complimentary

 * Startup Exchange Member: Complimentary Send email for code.
 * MIT Alum: 70% discount Send email for a discount code.
 * Sloan Exec Ed & Professional Education Member:  70% discount Send email for a discount code.


Visiting MIT: https://www.mit.edu/visitmit/

Where to Stay: https://institute-events.mit.edu/visit/where-to-stay

Registration Questions: ocrevents@mit.edu

  • Overview

    Spring 2022 ILP's in-person conferences will be open to fully vaccinated individuals only, excepting those individuals who have a medical condition or religious exemption. Conference attendees will use an application developed at MIT -- Tim Tickets -- to grant campus access or scan into an event.


    The pharmaceutical industry is under growing pressure from patent expirations of major drugs, cost-constrained health care systems, and a demanding regulatory environment. A focus for the industry is to tackle these challenges while increasing the output and quality of cost-effective, new medicines without incurring unsustainable R&D cost. Through new technology and new approaches there is an aspiration to make such improvements while also bringing ever-improved therapeutics to patients faster. In this conference, we will lay out many of the challenges the industry is facing and explore potential solutions coming from academic research and startup endeavors, from early discovery through manufacturing technologies, to clinical studies and beyond.


    REGISTRATION FEE

    Registration is closed, onsite registration will be a available at the conference

    • ILP Member: Complimentary
    • General Public: $1,350 
    • Current MIT Faculty/Staff/Student: Complimentary

     * Startup Exchange Member: Complimentary Send email for code.
     * MIT Alum: 70% discount Send email for a discount code.
     * Sloan Exec Ed & Professional Education Member:  70% discount Send email for a discount code.


    Visiting MIT: https://www.mit.edu/visitmit/

    Where to Stay: https://institute-events.mit.edu/visit/where-to-stay

    Registration Questions: ocrevents@mit.edu


Agenda

8:00 AM - 9:00 AM

Registration with Light Breakfast
9:00 AM - 9:20 AM

Welcome and Introduction
Executive Director (Interim), MIT Corporate Relations
John Roberts
Executive Director (Interim)

John Roberts has been Executive Director of MIT Corporate Relations (Interim) since February 2022. He obtained his Ph.D. in organic chemistry at MIT and returned to the university after a 20-year career in the pharmaceutical industry, joining the MIT Industrial Liaison Program (ILP) in 2013.  Prior to his return, John worked at small, medium, and large companies, holding positions that allowed him to exploit his passions in synthetic chemistry, project leadership, and alliance management while growing his responsibilities for managing others, ultimately as a department head. As a program director at MIT, John built a portfolio of ILP member companies, mostly in the pharmaceutical industry and headquartered in Japan, connecting them to engagement opportunities in the MIT community. Soon after returning to MIT, John began to lead a group of program directors with a combined portfolio of 60-80 global companies. In his current role, John oversees MIT Corporate Relations which houses ILP and MIT Startup Exchange.

Program Director, MIT Corporate Relations
Sheryl Greenberg
Program Director, MIT Corporate Relations

Sheryl Greenberg initiates and promotes the interactions and development of relationships between academic and industrial entities to facilitate the transfer of new ideas and technologies between MIT and companies, and has created numerous successful partnerships. By understanding the business, technology, and commercial problems within a company, and understanding the technologies and expertise of MIT researchers, Greenberg identifies appropriate resources and expertise to foster new technology applications and collaborative opportunities.

Prior to MIT, Greenberg created and directed the Office of Technology Transfer at Brandeis University. In the process of managing intellectual property protection, marketing, and licensing, she has promoted the successful commercialization of technologies as diverse as new chemicals and manufacturing, biotechnology, food compositions, software, and medical devices. She facilitated the founding and funding of new companies, as well as creating a profitable technology transfer program. She also facilitated the patenting, marketing, and licensing of Massachusetts General Hospital technologies. In addition to her cellular, biochemical, and genetic research experience in academic and corporate environments, she has also created intellectual property for medical uses. Greenberg has been an independent intellectual property and business development consultant, is a U.S. Patent Agent, and has previously served the Juvenile Diabetes Research Foundation as Co-Chair of the Islet Research Program Advisory Committee and grant reviewer. She currently also mentors startup companies and facilitates partnering them with large life science and healthcare companies.

Presentation
9:20 AM - 9:50 AM
Director of Translational Research, Immune Effector Cell Therapies​
Principal Investigator of a Synthetic Biology/Cellular Engineering/mRNA laboratory, Dana-Farber Cancer Institute
Eric Smith
Director of Translational Research, Immune Effector Cell Therapies​
Principal Investigator of a Synthetic Biology/Cellular Engineering/mRNA laboratory

Dr. Smith is Director of Translational Research for Immune Effector Cell Therapies and PI of a Synthetic Biology/Cellular Engineering/mRNA laboratory at Dana-Farber Cancer Institute (DFCI) as well as a member of the faculty at Harvard Medical School.  He completed his MD/PhD and internal medicine residency at the Mount Sinai School of Medicine and oncology fellowship at Memorial Sloan Kettering Cancer Center. His previous work identified myeloma specific targets with limited “on-target/off-tumor” potential, including the novel target for the immunotherapy of myeloma, GPRC5D.  He engineered optimized fully human CAR and antibodies therapies targeting both GPRC5D and BCMA licensed to BMS and Sanofi.  There are 7 clinical trials investigating CAR constructs stemming from this work. Currently the Smith Lab focuses on advancing the field of gene, cellular, and mRNA immunotherapies for cancer.

CAR T cell therapies for relapsed/refractory hematologic malignancies induce frequent deep clinical responses in patients who failed all other treatment options. While these results are game changing, the field is only scratching the surface of the potential benefit gene and cell therapies can provide.  Future advances include discovery of novel target antigens, extension of CAR T cell clinical benefit to solid tumors, logic gated engineering designs, advances in manufacturing, targeted in situ gene delivery, and off-the-shelf immunologically stealth approaches. The above will be broadly addressed and work from our group developing and translating CAR T cell therapies targeting the novel antigen GPRC5D for myeloma and “OR” gating strategies for hematologic malignancies will be highlighted as examples.

Presentation
9:50 AM - 10:20 AM
Instructor and Postdoctoral Associate in Biological Engineering, MIT
Deepak Mishra
Instructor and Postdoctoral Associate in Biological Engineering, MIT

Synthetic biologist with 10+ years of experience across bacteria, yeast, mammalian, and iPSC-derived organoids developing genetic networks encompassing transcriptional, translational, and post-translational regulation.

Co-author of 4 manuscripts including first-author publications in Science and Nature Biotechnology, launched 1 SynBio product in collaboration with ATCC, and awarded 2 provisional patents. Consulting experience as a key opinion leader for both Flagship Pioneering and M12. Co-lectured and co-taught 110 students over 5 years post-doctorate, mentored 80 undergraduates directly in independent research projects, and served as a teaching assistant for 160 undergraduates over 11 years at MIT. Additional honors include being a MIT Siebel Scholar, a MIT Energy Institute Fellow, a NSF GRFP recipient, Caltech's Institute Teaching Award, and former captain of Caltech's NCAA D.III varsity soccer team.

Career interests are aligned with tenure-track faculty positions in synthetic biology with specific expertise in mixed hybrid modality network design, ultrafast protein networks, interrogation of natural networks using computational algorithms, development and maturation of iPSC-derived drug/disease organoid models, lab automation, and the leveraging of of top-down systems biology with bottom-up synbio composition.

Organoids are tiny, self-organized three-dimensional (3D) tissue cultures erived from stem cells that allow recapitulation of architecture, composition, and physiology of organs. Organoids may be formed by combining individually differentiated cell populations or via the introduction of external cues to human induced pluripotent stem cells (hiPSCs) to mimic stages of human development. However, such approaches may limit intercellular interactions or faithful mimicry of a mature organ’s structure. We employ synthetic gene circuits to genetically engineer human induced pluripotent stem cells (hIPSCs) with transcriptional and post-transcriptional regulatory elements. These networks govern multi-step differentiation of hiPSCs in a cell-specific, semi-automated manner to form 3D liver organoids. Our recent advances in creation and subsequent use of these organoids in toxicology, drug metabolism, and implantation will be presented and discussed.

10:20 AM - 10:50 AM
Postdoctoral Associate, Darrell Irvine Lab, Koch Institute for Integrative Cancer Research at MIT
Parisa Yousefpour
Postdoctoral Associate, Darrell Irvine Lab

Parisa Yousefpour is a postdoctoral researcher in the Darrell Irvine Lab at the Koch Institute for Integrative Cancer Research at MIT. Her research focuses on regulating gene expression from self-replicating RNAs for cancer immunotherapy and vaccine development, and she has been awarded the Ludwig Fellowship from the Koch Institute and the Ruth L. Kirschstein NRSA (F32) Grant from the National Institutes of Health. Yousefpour’s PhD work focused on biomolecular engineering and development of recombinant biopolymer-based drug delivery systems for cancer and diabetes treatment. Yousefpour holds an integrated BS-MS degree in Biotechnology from the University of Tehran, Iran, and a PhD in Biomedical Engineering from Duke University. At Duke, she was awarded the Howard Clark Fellowship in Biomedical Engineering and the Bass Instructional Fellowship.

Self-replicating RNAs termed replicons have begun to be explored as a promising platform technology for vaccines and gene therapy.  Upon delivery to host cells, the replicon copies itself and therefore, allows for prolonged and increased transgene expression with a small initial dose.  We employ the replicon platform for 1) vaccine development for sustained antigen expression coupled with the intrinsic adjuvanticity of replicons, and 2) cancer immunotherapy to stimulate multiple synergistic pathways of antitumor immunity. In addition, harnessing synthetic biology tools, we are developing next generation replicon platforms that incorporate microRNA-based classifier and small-molecule responsive gene circuits for internal and external regulation of transgene expression, respectively.  Our recent advances and directions on gene delivery with replicons will be presented and discussed.

10:50 AM - 11:10 AM

Networking Break
11:10 AM - 11:15 AM

MIT Professional Education
Myriam Joseph
Assistant Director, Marketing & Business Development, MIT Professional Education
11:15 AM - 12:15 PM

MIT Startup Exchange Lightning Talks
Program Manager, MIT Startup Exchange
Ariadna Rodenstein
Program Manager

Ariadna Rodenstein is a Program Manager at MIT Startup Exchange. She joined MIT Corporate Relations as an Events Leader in September 2019 and is responsible for designing and executing startup events, including content development, coaching and hosting, and logistics. Ms. Rodenstein works closely with the Industrial Liaison Program (ILP) in promoting collaboration and partnerships between MIT-connected startups and industry, as well as with other areas around the MIT innovation ecosystem and beyond. 

Prior to working for MIT Corporate Relations, she worked for over a decade at Credit Suisse Group in New York and London, in a few different roles in event management and as Director of Client Strategy. Ms. Rodenstein has combined her experience in the private sector with work at non-profits as a Consultant and Development Director at New York Immigration Coalition, Immigrant Defense Project, and Americas Society/Council of the Americas. She also served as an Officer on the Board of Directors of the Riverside Clay Tennis Association in New York for several years. Additionally, she earned her B.A. in Political Science and Communications from New York University, with coursework at the Instituto Tecnológico y de Estudios Superiores de Monterrey in Mexico City, and her M.A. in Sociology from the City University of New York.

The future in cell therapy discovery and manufacturing
Paulo Garcia
CEO & Co-founder, Kytopen
Paulo Garcia
CEO & Co-founder

Paulo Garcia is a Biomedical Engineer that  co-invented the Flowfect™ technology to realize high-throughput, automated, and scalable non-viral cell engineering. His professional career has been centered around impacting the life sciences and healthcare via engineering innovation as demonstrated through 27 peer-reviewed manuscripts, 7 issued US patents, and several more pending patents. There is nothing that motivates Dr. Garcia more than knowing that the ongoing efforts will help patients suffering from devastating diseases worldwide. At Kytopen, we exist to accelerate the translation from the bench to the clinic and ultimate impact patient’s lives. Prior to being CEO & Co-Founder  at Kytopen, he was a Research Scientist in the Laboratory for Energy and Microsystems Innovation (Prof. Buie’s laboratory) in Mechanical Engineering at MIT.

Making personalized cell therapies scalable
Marinna Madrid
Co-founder, Cellino
Marinna Madrid
Co-founder

Marinna Madrid is a co-founder at Cellino, a venture capital-backed early-stage biotech company. Cellino is making personalized regenerative medicines economically viable at scale for the first time. Marinna received her PhD and MA in Applied Physics from Harvard University, where she co-invented laser-based intracellular delivery techniques. She received her BSc in Biophysics from University of California, Los Angeles, after transferring from Riverside Community College. She is the recipient of the Harvard Graduate Prize Fellowship, the Catalyst Accelerator Grant from Harvard Medical School, and is on the Forbes 30 Under 30 2019 list for Healthcare.

Pioneering tRNA/mRNA-based gene therapy platforms
Gopi Shanker
Chief Scientific Officer, Tevard Biosciences
Gopi Shanker
Chief Scientific Officer

Gopi Shanker is the Chief Scientific Officer of Tevard Biosciences. He has over two decades of broad drug discovery experience and a strong background across multiple drug modalities including small molecules, biologics and gene therapies for various CNS indications including schizophrenia, depression, Alzheimer’s disease, Parkinson’s disease, chronic pain and migraine as well as several rare neurodevelopmental and neurodegenerative diseases.

Prior to joining Tevard, Gopi was at the Novartis Institutes for Biomedical Research (NIBR), where he most recently served in the capacity of Head of Neuroscience with responsibility for strategic oversight of the Novartis neuroscience portfolio, including psychiatric, neurodevelopmental and neurological disorders. At Novartis, Gopi advanced multiple new therapeutic programs through late preclinical and clinical development and was responsible for expanding Novartis’ portfolio in psychiatry as well as developing a portfolio of gene therapy programs targeting neurodevelopmental disorders. Prior to joining Novartis, Gopi led several drug discovery programs at Amgen and Regeneron.

Gopi completed a Howard Hughes Medical Institute postdoctoral fellowship at the Icahn School of Medicine at Mount Sinai in New York. He earned his Ph.D. from the Indian Institute of Science in Bangalore.

Cell therapy manufacturing, enabled by robotics
Fred Parietti
Founder & CEO, Multiply Labs
Fred Parietti
Founder & CEO

Fred (Federico) Parietti is specialized in the control and design of advanced robotic systems. Since 2016, Fred has served as Founder and Chief Executive Officer of Multiply Labs. Multiply Labs is an advanced Series-A startup based in San Francisco developing the first demonstration of fully-automated robotic manufacturing in the pharmaceutical industry to produce personalized therapeutics. The company has raised over $20M from top investors and is engaged with top pharmaceutical customers around the world. Fred earned his PhD from MIT, where he worked on robotic exoskeletons advised by Dr. Harry Asada. He strongly believes that robotic technology will help humankind transcend its limits - making us smarter, stronger and healthier. Prior to obtaining his PhD, Fred developed advanced robotic systems as a researcher at Carnegie Mellon University, ETH Zurich, and Politecnico di Milano and Politecnico di Torino where he earned his M.S. and B.S., respectively. Fred has co-authored over 35 articles with a total of 1424 citations and was awarded over 10 patents (with an additional 15+ provisional patents filed). His work has been featured on New Scientist, Fortune, Fox News, IEEE Spectrum, The Verge, The Smithsonian, MIT News, Tech Crunch, Endpoint News and Fierce Pharma. 

Single-stranded DNA vectors for gene insertions and replacement
Floris Engelhardt
Floris Engelhardt
CEO

Floris Engelhardt is CEO of Kano Therapeutics which she co-founded with her former PostDoc advisor Prof. Mark Bathe (MIT). She holds a bachelor’s degree in Biochemisty and Molecular Biology from the Friedich-Schiller University in Jena, a master’s degree in Molecular Biotechnology and a Doctoral Degree in Physics from the Technical University of Munich. Her work has been focused on single-stranded DNA production and Nucleic Acid Nanostructures for gene and cell therapy.

Simplifying the front-end workflows in genomic sequencing
Udayan Umapathi
Co-founder & CEO, Volta Labs
Udayan Umapathi
Co-founder & CEO

Udayan Umapathi is the co-founder and CEO of bioautomation company Volta Labs, Inc. Drawing from over a decade of industry and research experience at the MIT Media Lab, Hasso Plattner Institute, Cypress Semiconductors and other startups, Umapathi holds a depth of expertise in engineering, molecular biology, human-computer interaction design, and entrepreneurship. Prior to starting Volta, Umapathi was a researcher at the MIT Media Lab. His work at the Lab has led to several patents and publications leading up to commercialization by Fortune 500 companies. Umapathi holds multiple Master's degrees from the MIT Media Lab’s Tangible Media Group and Purdue University. Prior to the Media Lab, he has been part of several early stage startups and the semiconductor industry and shipping and consumer-focused products.

Dynamic single-cell analysis at scale
Daniel Meyer
CEO, CellChorus
Daniel Meyer
CEO

Daniel Meyer is the CEO of CellChorus. He is the former Chief Operating Officer and was a board member of Genospace, which was funded by Thomson Reuters/Clarivate and acquired by HCA Healthcare (NYSE:HCA), the largest private hospital company in the United States and one of the largest oncology clinical care and clinical trial organizations in the United States, having led more than 450 first-in-man clinical trials and having been a clinical trial leader in the majority of approved cancer therapies over the last ten years.

Mr. Meyer was also a member of the early stage healthcare and life sciences investment teams at Arboretum Ventures and PJC.vc, where he focused on investments in life sciences, medical devices, health care information technology, and health care services. Mr. Meyer holds an MBA from the Tuck School of Business at Dartmouth and a BA from Middlebury College.

Simplifying access to bioprocessing data
Elisabeth Maida
Co-founder & CEO, Fathom Data
Elisabeth Maida
Co-founder & CEO

Liz Maida is the co-founder and CEO of Fathom and was previously the co-founder and CEO of Uplevel Security (acquired by McAfee in 2019). Uplevel applied graph theory and machine learning to enhance the efficiency and effectiveness of security operations teams. Prior to McAfee, Liz served in multiple executive roles at Akamai Technologies focused on technology strategy and new product development. Liz holds a BS from Princeton University and dual MS degrees from MIT. Her graduate school research examined the application of graph theory to network interconnection.

12:15 PM - 1:25 PM

Lunch with Startups Exhibit

Exhibit only startup

LiquiGlide: Friction-reducing coating technology to enhance cell/gene delivery performance

1:30 PM - 2:00 PM
Ran Zheng
CEO

Ran is a biotechnology industry veteran with more than 25 years of experience in biotechnology operations across multiple geographies. Prior to joining Landmark Bio, Ran most recently served as Chief Technical Officer at Orchard Therapeutics, a commercial-stage global gene therapy company specializing in hematopoietic stem cell based gene therapies. In this role, she established the technical operations function and manufacturing network, and advanced the company’s product pipeline, including the approval of Libmeldy® by EMA - the first gene therapy product for metachromatic leukodystrophy. Ran has also held leadership positions at several major biotechnology companies including Genzyme (now Sanofi) and Amgen. At Amgen, Ran played a key role in building differentiating capabilities in manufacturing for clinical supply and commercial product launch to enable speed to clinic and speed to market strategies for Amgen’s innovative products.

We have entered the golden age of biology with an unprecedented explosion of discoveries that could lead to life changing therapies and have profound impact on human health. However, the shortage of manufacturing capacity and expertise in recent years has constrained therapeutic translation especially for the life sciences innovators who explore emerging modalities and unique technologies. Manufacturing scalability, reproducibility and the cost of goods remain as major challenges, while the evolving science and regulatory landscape require continuous learning. 

In 2021, Harvard and MIT partnered with Cytiva, Fujifilm Diosynth Biotechnologies and Alexandria Real Estate Equities to bring the best of academia, the life sciences industry, and world-class research hospitals together to accelerate life sciences innovation. Landmark Bio was established to break down the barriers in novel therapeutics development and industrialization. We collaborate with academics, startups and drug developers to take groundbreaking discoveries from bench to clinics by providing capacity and expertise in CMC development and manufacturing. Uniquely positioned as pre-platform and technology agnostic, we help design, de-risk, and develop early innovations into future platforms. Our cross-sector partnership offers an unparalleled academic, industry, hospitals and investor network with a magnetic pull to power biomanufacturing innovation. As a mission-driven organization, we embrace risk and opportunities at the bleeding-edge in order to advance emerging technologies, demonstrate therapeutic potential and improve human health.

2:00 PM - 2:30 PM
Professor of Electrical Engineering and Professor of Biological Engineering
Jongyoon Han
Professor of Electrical Engineering and Professor of Biological Engineering

Dr. Jongyoon Han is currently a professor in the Department of Electrical Engineering and Computer Science and the Department of Biological Engineering, Massachusetts Institute of Technology. He received B.S.(1992) and M.S.(1994) degree in physics from Seoul National University, Seoul, Korea, and Ph.D. degree in applied physics from Cornell University in 2001. He was a research scientist in Sandia National Laboratories (Livermore, CA), until he joined the MIT faculty in 2002. He received NSF CAREER award (2003) and Analytical Chemistry Young Innovator Award (ACS, 2009). His research is mainly focused on applying micro/nanofabrication techniques to a very diverse set of fields and industries, including biosensing, desalination / water purification, biomanufacturing, dentistry, and neuroscience. He is currently the lead PI for MIT’s participation for NIIMBL (The National Institute for Innovation in Manufacturing Biopharmaceuticals).

One of the critical challenges in cell therapy is the lack of reliable, specific, and non-destructive quality attributes, which are sorely needed for all aspects of biomanufacturing of these cells, including donor selection, in-process quality monitoring, and release testing. Most biological and biochemical assays are destructive or perturbative, which limits their utility, especially for autologous cell therapy. In this talk, I will showcase some of the emerging ideas of label-free, biophysical critical quality attributes (CQAs) we have been working on, including magnetic, electrical, and mechanical signatures of cells. Once the strong correlation with biochemical cell phenotypes, these will serve an important role to improve the overall production of both allogeneic and autologous cell therapy products.

2:30 PM - 3:00 PM
Core Member, Broad Institute of MIT and Harvard
Investigator, McGovern Institute for Brain Research, MIT
James and Patricia Poitras Professor of Neuroscience at MIT
Associate Professor, Departments of Brain and Cognitive Sciences and Biological Engineering, MIT
New York Stem Cell Foundation-Robertson Investigator
Feng Zhang
Core Member, Broad Institute of MIT and Harvard
Investigator, McGovern Institute for Brain Research, MIT
James and Patricia Poitras Professor of Neuroscience at MIT
Associate Professor, Departments of Brain and Cognitive Sciences and Biological Engineering, MIT
New York Stem Cell Foundation-Robertson Investigator

Feng Zhang attended Harvard University as an undergraduate, where he earned an A.B. in Chemistry and Physics in 2004. He transitioned to Stanford for his Ph.D. in Chemistry and Biophysics, which he obtained in 2009 while working in Karl Deisseroth’s research group for his work  pioneering optogenetics in conjunction with Ed Boyden. After a postdoc in George Church’s lab, he moved to MIT in January of 2011, where he is now both an Associate Professor of Biological Engineering and a Core Institute Member of the Broad Institute of MIT and Harvard.

Feng Zhang has received a number of honors, including the NIH Pioneer Award and the Perl-UNC Neuroscience Prize, along with Ed Boyden and thesis advisor Deisseroth. He is additionally a Searle Scholar.

Many powerful molecular biology tools have their origin in nature, and, often, microbial life. From restriction enzymes to CRISPR-Cas9, microbes utilize a diverse array of systems to get ahead evolutionarily. We are interested in exploring this natural diversity through bioinformatics, biochemical, and molecular work to better understand the fundamental ways in which living organisms sense and respond to their environment and ultimately to harness these systems to improve human health. Building on our demonstration that Cas9 can be repurposed for precision genome editing in mammalian cells, we began looking for novel CRISPR-Cas systems that may have other useful properties. This led to the discovery of several new CRISPR systems, including the CRISPR-Cas13 family that target RNA, rather than DNA. We developed a toolbox for RNA modulation based on Cas13, including methods for precision base editing. We are expanding our biodiscovery efforts to search for new microbial proteins that may be adapted for applications beyond genome and transcriptome modulation, capitalizing on the growing volume of microbial genomic sequences and building on our bioengineering expertise. We are particularly interested in identifying new therapeutic modalities and vehicles for delivering cellular and molecular cargo. We hope that this combination of tools and delivery modes will accelerate basic research into human disease and open up new therapeutic possibilities.

3:00 PM - 3:20 PM

Networking Break
3:20 PM - 3:50 PM
Professor of Biology, Koch Institute for Integrative Cancer Research
MIT Department of Biology
Jianzhu Chen
Professor of Biology, Koch Institute for Integrative Cancer Research
MIT Department of Biology

Jianzhu Chen is Professor of Biology at Koch Institute for Integrative Cancer Research and Department of Biology at Massachusetts Institute of Technology (MIT). Dr. Chen received a Ph.D. degree from Stanford University. He was a postdoctoral fellow and then an instructor at Harvard Medical School before he joined the faculty at MIT. Dr. Chen’s research seeks fundamental understanding of the immune system as well as its application in disease intervention. Over the years, Dr. Chen has made significant contributions to a broad area of research in immunology, cancer research, infectious diseases, and animal models of human diseases. His research on lymphocyte homeostasis and immunological memory challenged the prevailing paradigm at the time and revealed unexpected effect of lymphopenia-induced proliferation on memory T cell development. Dr. Chen pioneered innovative research in development of novel mouse models through genetic manipulations and cell complementation and in development of novel anti-microbial, including surface coatings that inactivate microbes on contact, universal anti-influenza siRNAs, and polymer-attached antivirals that minimize drug resistance of influenza viruses. Recently, Dr. Chen’s research activity has focused on development of tumor-specific CAR-NK cells and re-programming macrophages for disease intervention, including cancer, metabolic diseases and infectious diseases.

Natural Killer (NK) cells and CD8+ cytotoxic T cells are two types of immune cells that can kill target cells through similar cytotoxic mechanisms. With the remarkable success of chimeric antigen receptor-engineered T (CAR-T) cells for treating hematological malignancies, there is a rapidly growing interest in developing CAR-engineered NK (CAR-NK) cells for cancer therapy. Compared to CAR-T cells, CAR-NK cells could offer some significant advantages, including (1) better safety, such as a lack of or minimal cytokine release syndrome and neurotoxicity in autologous setting and graft-versus-host disease in allogeneic setting, (2) multiple mechanisms for activating cytotoxic activity, and (3) high feasibility for “off-the-shelf” manufacturing. We are developing the next generation of CAR-NK cells by combining tumor-specific CAR, additional armors, and cytokine-induced memory-like (CIML) NK cells, with a goal to achieve better tumor-specific targeting, enhanced proliferation and persistence in vivo, resistance to the suppressive tumor microenvironment, and ultimately an effective and durable anti-tumor response in patients.

Presentation
3:50 PM - 4:20 PM
Faculty Research Officer
Gilliland Professor, MIT Department of Chemical Engineering
Richard Braatz
Faculty Research Officer
Gilliland Professor

Richard D. Braatz is the Edwin R. Gilliland Professor of Chemical Engineering at MIT, where he conducts research into advanced biomanufacturing systems. He leads process data analytics, mechanistic modelling, and control systems activities for projects on monoclonal antibody, vaccine, and gene therapy manufacturing. Dr. Braatz received an M.S. and Ph.D. from the California Institute of Technology and was a Visiting Scholar at Harvard University before moving to MIT. He has collaborated with more than 20 companies including Novartis, Pfizer, Merck, Bristol-Myers Squibb, Biogen, Amgen, Takeda, and Abbott Labs. Honors include the AIChE PD2M Award for Outstanding Contribution to QbD for Drug Substance, the AIChE Excellence in Process Development Research Award, the Technical Innovation Award from the International Society of Automation, the IEEE Control Systems Society Transition to Practice Award, and election to the U.S. National Academy of Engineering.

Ph.D. student in chemical engineering at MIT
Tam Nguyen
Ph.D. student in chemical engineering at MIT

Tam Nguyen is a Ph.D. student in chemical engineering at MIT, where she does research in biopharmaceutical manufacturing systems. Her main research is in mechanistic modeling for AAV-based transfection and its use in improving understanding, optimization, and control of AAV capsid production. Tam received a Bachelors of Science in Chemical Engineering from the University of Massachusetts Amherst and a Masters of Science in Chemical Engineering Practice from MIT. Richard Braatz is the Edwin R. Gilliland Professor of Chemical Engineering at MIT, where he conducts research into advanced biopharmaceutical manufacturing systems. In this role, he leads process data analytics, mechanistic modeling, and control systems for many projects, including on monoclonal antibody, viral vaccine, and gene therapy manufacturing within the Center of Biomedical Innovation, and on protein crystallization, continuous lyophilization, and therapeutic protein formulation within the Department of Chemical Engineering.

Recombinant adeno-associated virus (rAAV) is one of the most commonly used platforms for in vivo gene therapy treatments. The reduced toxicity, robust and long-term transgene expression, and ability to transduce both dividing and non-dividing cells as well as target a wide range of tissues have made rAAV the most widely used viral vector. However, the standard method of producing rAAV via transient transfection of mammalian cells, specifically human embryonic kidney 293 (HEK293) cells, typically has low yield and generates a high portion of empty particles, laying extra burden on downstream processing. To elucidate the mechanisms of rAAV synthesis in HEK293 suspension-adapted cells, we have developed a mechanistic model based on the published understanding of the underlying biology and existing data. Quantitative analysis suggests the misaligned dynamics of capsid and viral DNA production result in the high ratio of empty particles. Through a model-based strategy, we explored a novel transfection method using low-dose multiple transfections in HEK293 cell culture that successfully increased the ratio of full to empty capsids in the viral harvest without compromising the viral titer. Molecular analysis through a next-generation rAAV production model attributed the improvements to changes in the kinetics of viral protein expression and DNA replication. Here, we demonstrate that the use of multiple transfection times is a practical method for increasing the genome titer and improving the percentage of full capsids for rAAV production. Our results also demonstrated the capability to manipulate product composition from an operational standpoint.

Presentation
4:20 PM - 4:50 PM
Associate Professor of Biological Engineering, MIT Department of Biological Engineering
Michael Birnbaum
Associate Professor of Biological Engineering

Michael obtained an A.B. in Chemical and Physical Biology at Harvard University in 2008. He then moved to Stanford University, where he completed his Ph.D. in Immunology in 2014. At Stanford, he worked in Professor K. Christopher Garcia’s laboratory, studying the molecular mechanisms of T cell receptor recognition, cross-reactivity, and activation. He then conducted postdoctoral research in Professor Carla Shatz’s laboratory, studying novel roles for immune receptors expressed by neurons in neural development and neurodegenerative disease. Michael joined the Department of Biological Engineering in 2016 as an Assistant Professor.

Cell and gene immunotherapies are revolutionizing how we treat disease, with multiple FDA-approved therapies that have transformed cancer treatments. However, advances in gene delivery, manufacturing, and therapeutic cargoes are still required to increase the impact and scope of these promising approaches. The Birnbaum laboratory is working to develop approaches that improve the specificity of cellular engineering, and the potency of cells once engineered.

4:50 PM - 6:00 PM

Closing Remarks and Networking Reception
  • Agenda
    8:00 AM - 9:00 AM

    Registration with Light Breakfast
    9:00 AM - 9:20 AM

    Welcome and Introduction
    Executive Director (Interim), MIT Corporate Relations
    John Roberts
    Executive Director (Interim)

    John Roberts has been Executive Director of MIT Corporate Relations (Interim) since February 2022. He obtained his Ph.D. in organic chemistry at MIT and returned to the university after a 20-year career in the pharmaceutical industry, joining the MIT Industrial Liaison Program (ILP) in 2013.  Prior to his return, John worked at small, medium, and large companies, holding positions that allowed him to exploit his passions in synthetic chemistry, project leadership, and alliance management while growing his responsibilities for managing others, ultimately as a department head. As a program director at MIT, John built a portfolio of ILP member companies, mostly in the pharmaceutical industry and headquartered in Japan, connecting them to engagement opportunities in the MIT community. Soon after returning to MIT, John began to lead a group of program directors with a combined portfolio of 60-80 global companies. In his current role, John oversees MIT Corporate Relations which houses ILP and MIT Startup Exchange.

    Program Director, MIT Corporate Relations
    Sheryl Greenberg
    Program Director, MIT Corporate Relations

    Sheryl Greenberg initiates and promotes the interactions and development of relationships between academic and industrial entities to facilitate the transfer of new ideas and technologies between MIT and companies, and has created numerous successful partnerships. By understanding the business, technology, and commercial problems within a company, and understanding the technologies and expertise of MIT researchers, Greenberg identifies appropriate resources and expertise to foster new technology applications and collaborative opportunities.

    Prior to MIT, Greenberg created and directed the Office of Technology Transfer at Brandeis University. In the process of managing intellectual property protection, marketing, and licensing, she has promoted the successful commercialization of technologies as diverse as new chemicals and manufacturing, biotechnology, food compositions, software, and medical devices. She facilitated the founding and funding of new companies, as well as creating a profitable technology transfer program. She also facilitated the patenting, marketing, and licensing of Massachusetts General Hospital technologies. In addition to her cellular, biochemical, and genetic research experience in academic and corporate environments, she has also created intellectual property for medical uses. Greenberg has been an independent intellectual property and business development consultant, is a U.S. Patent Agent, and has previously served the Juvenile Diabetes Research Foundation as Co-Chair of the Islet Research Program Advisory Committee and grant reviewer. She currently also mentors startup companies and facilitates partnering them with large life science and healthcare companies.

    Presentation
    9:20 AM - 9:50 AM
    Director of Translational Research, Immune Effector Cell Therapies​
    Principal Investigator of a Synthetic Biology/Cellular Engineering/mRNA laboratory, Dana-Farber Cancer Institute
    Eric Smith
    Director of Translational Research, Immune Effector Cell Therapies​
    Principal Investigator of a Synthetic Biology/Cellular Engineering/mRNA laboratory

    Dr. Smith is Director of Translational Research for Immune Effector Cell Therapies and PI of a Synthetic Biology/Cellular Engineering/mRNA laboratory at Dana-Farber Cancer Institute (DFCI) as well as a member of the faculty at Harvard Medical School.  He completed his MD/PhD and internal medicine residency at the Mount Sinai School of Medicine and oncology fellowship at Memorial Sloan Kettering Cancer Center. His previous work identified myeloma specific targets with limited “on-target/off-tumor” potential, including the novel target for the immunotherapy of myeloma, GPRC5D.  He engineered optimized fully human CAR and antibodies therapies targeting both GPRC5D and BCMA licensed to BMS and Sanofi.  There are 7 clinical trials investigating CAR constructs stemming from this work. Currently the Smith Lab focuses on advancing the field of gene, cellular, and mRNA immunotherapies for cancer.

    CAR T cell therapies for relapsed/refractory hematologic malignancies induce frequent deep clinical responses in patients who failed all other treatment options. While these results are game changing, the field is only scratching the surface of the potential benefit gene and cell therapies can provide.  Future advances include discovery of novel target antigens, extension of CAR T cell clinical benefit to solid tumors, logic gated engineering designs, advances in manufacturing, targeted in situ gene delivery, and off-the-shelf immunologically stealth approaches. The above will be broadly addressed and work from our group developing and translating CAR T cell therapies targeting the novel antigen GPRC5D for myeloma and “OR” gating strategies for hematologic malignancies will be highlighted as examples.

    Presentation
    9:50 AM - 10:20 AM
    Instructor and Postdoctoral Associate in Biological Engineering, MIT
    Deepak Mishra
    Instructor and Postdoctoral Associate in Biological Engineering, MIT

    Synthetic biologist with 10+ years of experience across bacteria, yeast, mammalian, and iPSC-derived organoids developing genetic networks encompassing transcriptional, translational, and post-translational regulation.

    Co-author of 4 manuscripts including first-author publications in Science and Nature Biotechnology, launched 1 SynBio product in collaboration with ATCC, and awarded 2 provisional patents. Consulting experience as a key opinion leader for both Flagship Pioneering and M12. Co-lectured and co-taught 110 students over 5 years post-doctorate, mentored 80 undergraduates directly in independent research projects, and served as a teaching assistant for 160 undergraduates over 11 years at MIT. Additional honors include being a MIT Siebel Scholar, a MIT Energy Institute Fellow, a NSF GRFP recipient, Caltech's Institute Teaching Award, and former captain of Caltech's NCAA D.III varsity soccer team.

    Career interests are aligned with tenure-track faculty positions in synthetic biology with specific expertise in mixed hybrid modality network design, ultrafast protein networks, interrogation of natural networks using computational algorithms, development and maturation of iPSC-derived drug/disease organoid models, lab automation, and the leveraging of of top-down systems biology with bottom-up synbio composition.

    Organoids are tiny, self-organized three-dimensional (3D) tissue cultures erived from stem cells that allow recapitulation of architecture, composition, and physiology of organs. Organoids may be formed by combining individually differentiated cell populations or via the introduction of external cues to human induced pluripotent stem cells (hiPSCs) to mimic stages of human development. However, such approaches may limit intercellular interactions or faithful mimicry of a mature organ’s structure. We employ synthetic gene circuits to genetically engineer human induced pluripotent stem cells (hIPSCs) with transcriptional and post-transcriptional regulatory elements. These networks govern multi-step differentiation of hiPSCs in a cell-specific, semi-automated manner to form 3D liver organoids. Our recent advances in creation and subsequent use of these organoids in toxicology, drug metabolism, and implantation will be presented and discussed.

    10:20 AM - 10:50 AM
    Postdoctoral Associate, Darrell Irvine Lab, Koch Institute for Integrative Cancer Research at MIT
    Parisa Yousefpour
    Postdoctoral Associate, Darrell Irvine Lab

    Parisa Yousefpour is a postdoctoral researcher in the Darrell Irvine Lab at the Koch Institute for Integrative Cancer Research at MIT. Her research focuses on regulating gene expression from self-replicating RNAs for cancer immunotherapy and vaccine development, and she has been awarded the Ludwig Fellowship from the Koch Institute and the Ruth L. Kirschstein NRSA (F32) Grant from the National Institutes of Health. Yousefpour’s PhD work focused on biomolecular engineering and development of recombinant biopolymer-based drug delivery systems for cancer and diabetes treatment. Yousefpour holds an integrated BS-MS degree in Biotechnology from the University of Tehran, Iran, and a PhD in Biomedical Engineering from Duke University. At Duke, she was awarded the Howard Clark Fellowship in Biomedical Engineering and the Bass Instructional Fellowship.

    Self-replicating RNAs termed replicons have begun to be explored as a promising platform technology for vaccines and gene therapy.  Upon delivery to host cells, the replicon copies itself and therefore, allows for prolonged and increased transgene expression with a small initial dose.  We employ the replicon platform for 1) vaccine development for sustained antigen expression coupled with the intrinsic adjuvanticity of replicons, and 2) cancer immunotherapy to stimulate multiple synergistic pathways of antitumor immunity. In addition, harnessing synthetic biology tools, we are developing next generation replicon platforms that incorporate microRNA-based classifier and small-molecule responsive gene circuits for internal and external regulation of transgene expression, respectively.  Our recent advances and directions on gene delivery with replicons will be presented and discussed.

    10:50 AM - 11:10 AM

    Networking Break
    11:10 AM - 11:15 AM

    MIT Professional Education
    Myriam Joseph
    Assistant Director, Marketing & Business Development, MIT Professional Education
    11:15 AM - 12:15 PM

    MIT Startup Exchange Lightning Talks
    Program Manager, MIT Startup Exchange
    Ariadna Rodenstein
    Program Manager

    Ariadna Rodenstein is a Program Manager at MIT Startup Exchange. She joined MIT Corporate Relations as an Events Leader in September 2019 and is responsible for designing and executing startup events, including content development, coaching and hosting, and logistics. Ms. Rodenstein works closely with the Industrial Liaison Program (ILP) in promoting collaboration and partnerships between MIT-connected startups and industry, as well as with other areas around the MIT innovation ecosystem and beyond. 

    Prior to working for MIT Corporate Relations, she worked for over a decade at Credit Suisse Group in New York and London, in a few different roles in event management and as Director of Client Strategy. Ms. Rodenstein has combined her experience in the private sector with work at non-profits as a Consultant and Development Director at New York Immigration Coalition, Immigrant Defense Project, and Americas Society/Council of the Americas. She also served as an Officer on the Board of Directors of the Riverside Clay Tennis Association in New York for several years. Additionally, she earned her B.A. in Political Science and Communications from New York University, with coursework at the Instituto Tecnológico y de Estudios Superiores de Monterrey in Mexico City, and her M.A. in Sociology from the City University of New York.

    The future in cell therapy discovery and manufacturing
    Paulo Garcia
    CEO & Co-founder, Kytopen
    Paulo Garcia
    CEO & Co-founder

    Paulo Garcia is a Biomedical Engineer that  co-invented the Flowfect™ technology to realize high-throughput, automated, and scalable non-viral cell engineering. His professional career has been centered around impacting the life sciences and healthcare via engineering innovation as demonstrated through 27 peer-reviewed manuscripts, 7 issued US patents, and several more pending patents. There is nothing that motivates Dr. Garcia more than knowing that the ongoing efforts will help patients suffering from devastating diseases worldwide. At Kytopen, we exist to accelerate the translation from the bench to the clinic and ultimate impact patient’s lives. Prior to being CEO & Co-Founder  at Kytopen, he was a Research Scientist in the Laboratory for Energy and Microsystems Innovation (Prof. Buie’s laboratory) in Mechanical Engineering at MIT.

    Making personalized cell therapies scalable
    Marinna Madrid
    Co-founder, Cellino
    Marinna Madrid
    Co-founder

    Marinna Madrid is a co-founder at Cellino, a venture capital-backed early-stage biotech company. Cellino is making personalized regenerative medicines economically viable at scale for the first time. Marinna received her PhD and MA in Applied Physics from Harvard University, where she co-invented laser-based intracellular delivery techniques. She received her BSc in Biophysics from University of California, Los Angeles, after transferring from Riverside Community College. She is the recipient of the Harvard Graduate Prize Fellowship, the Catalyst Accelerator Grant from Harvard Medical School, and is on the Forbes 30 Under 30 2019 list for Healthcare.

    Pioneering tRNA/mRNA-based gene therapy platforms
    Gopi Shanker
    Chief Scientific Officer, Tevard Biosciences
    Gopi Shanker
    Chief Scientific Officer

    Gopi Shanker is the Chief Scientific Officer of Tevard Biosciences. He has over two decades of broad drug discovery experience and a strong background across multiple drug modalities including small molecules, biologics and gene therapies for various CNS indications including schizophrenia, depression, Alzheimer’s disease, Parkinson’s disease, chronic pain and migraine as well as several rare neurodevelopmental and neurodegenerative diseases.

    Prior to joining Tevard, Gopi was at the Novartis Institutes for Biomedical Research (NIBR), where he most recently served in the capacity of Head of Neuroscience with responsibility for strategic oversight of the Novartis neuroscience portfolio, including psychiatric, neurodevelopmental and neurological disorders. At Novartis, Gopi advanced multiple new therapeutic programs through late preclinical and clinical development and was responsible for expanding Novartis’ portfolio in psychiatry as well as developing a portfolio of gene therapy programs targeting neurodevelopmental disorders. Prior to joining Novartis, Gopi led several drug discovery programs at Amgen and Regeneron.

    Gopi completed a Howard Hughes Medical Institute postdoctoral fellowship at the Icahn School of Medicine at Mount Sinai in New York. He earned his Ph.D. from the Indian Institute of Science in Bangalore.

    Cell therapy manufacturing, enabled by robotics
    Fred Parietti
    Founder & CEO, Multiply Labs
    Fred Parietti
    Founder & CEO

    Fred (Federico) Parietti is specialized in the control and design of advanced robotic systems. Since 2016, Fred has served as Founder and Chief Executive Officer of Multiply Labs. Multiply Labs is an advanced Series-A startup based in San Francisco developing the first demonstration of fully-automated robotic manufacturing in the pharmaceutical industry to produce personalized therapeutics. The company has raised over $20M from top investors and is engaged with top pharmaceutical customers around the world. Fred earned his PhD from MIT, where he worked on robotic exoskeletons advised by Dr. Harry Asada. He strongly believes that robotic technology will help humankind transcend its limits - making us smarter, stronger and healthier. Prior to obtaining his PhD, Fred developed advanced robotic systems as a researcher at Carnegie Mellon University, ETH Zurich, and Politecnico di Milano and Politecnico di Torino where he earned his M.S. and B.S., respectively. Fred has co-authored over 35 articles with a total of 1424 citations and was awarded over 10 patents (with an additional 15+ provisional patents filed). His work has been featured on New Scientist, Fortune, Fox News, IEEE Spectrum, The Verge, The Smithsonian, MIT News, Tech Crunch, Endpoint News and Fierce Pharma. 

    Single-stranded DNA vectors for gene insertions and replacement
    Floris Engelhardt
    Floris Engelhardt
    CEO

    Floris Engelhardt is CEO of Kano Therapeutics which she co-founded with her former PostDoc advisor Prof. Mark Bathe (MIT). She holds a bachelor’s degree in Biochemisty and Molecular Biology from the Friedich-Schiller University in Jena, a master’s degree in Molecular Biotechnology and a Doctoral Degree in Physics from the Technical University of Munich. Her work has been focused on single-stranded DNA production and Nucleic Acid Nanostructures for gene and cell therapy.

    Simplifying the front-end workflows in genomic sequencing
    Udayan Umapathi
    Co-founder & CEO, Volta Labs
    Udayan Umapathi
    Co-founder & CEO

    Udayan Umapathi is the co-founder and CEO of bioautomation company Volta Labs, Inc. Drawing from over a decade of industry and research experience at the MIT Media Lab, Hasso Plattner Institute, Cypress Semiconductors and other startups, Umapathi holds a depth of expertise in engineering, molecular biology, human-computer interaction design, and entrepreneurship. Prior to starting Volta, Umapathi was a researcher at the MIT Media Lab. His work at the Lab has led to several patents and publications leading up to commercialization by Fortune 500 companies. Umapathi holds multiple Master's degrees from the MIT Media Lab’s Tangible Media Group and Purdue University. Prior to the Media Lab, he has been part of several early stage startups and the semiconductor industry and shipping and consumer-focused products.

    Dynamic single-cell analysis at scale
    Daniel Meyer
    CEO, CellChorus
    Daniel Meyer
    CEO

    Daniel Meyer is the CEO of CellChorus. He is the former Chief Operating Officer and was a board member of Genospace, which was funded by Thomson Reuters/Clarivate and acquired by HCA Healthcare (NYSE:HCA), the largest private hospital company in the United States and one of the largest oncology clinical care and clinical trial organizations in the United States, having led more than 450 first-in-man clinical trials and having been a clinical trial leader in the majority of approved cancer therapies over the last ten years.

    Mr. Meyer was also a member of the early stage healthcare and life sciences investment teams at Arboretum Ventures and PJC.vc, where he focused on investments in life sciences, medical devices, health care information technology, and health care services. Mr. Meyer holds an MBA from the Tuck School of Business at Dartmouth and a BA from Middlebury College.

    Simplifying access to bioprocessing data
    Elisabeth Maida
    Co-founder & CEO, Fathom Data
    Elisabeth Maida
    Co-founder & CEO

    Liz Maida is the co-founder and CEO of Fathom and was previously the co-founder and CEO of Uplevel Security (acquired by McAfee in 2019). Uplevel applied graph theory and machine learning to enhance the efficiency and effectiveness of security operations teams. Prior to McAfee, Liz served in multiple executive roles at Akamai Technologies focused on technology strategy and new product development. Liz holds a BS from Princeton University and dual MS degrees from MIT. Her graduate school research examined the application of graph theory to network interconnection.

    12:15 PM - 1:25 PM

    Lunch with Startups Exhibit

    Exhibit only startup

    LiquiGlide: Friction-reducing coating technology to enhance cell/gene delivery performance

    1:30 PM - 2:00 PM
    Ran Zheng
    CEO

    Ran is a biotechnology industry veteran with more than 25 years of experience in biotechnology operations across multiple geographies. Prior to joining Landmark Bio, Ran most recently served as Chief Technical Officer at Orchard Therapeutics, a commercial-stage global gene therapy company specializing in hematopoietic stem cell based gene therapies. In this role, she established the technical operations function and manufacturing network, and advanced the company’s product pipeline, including the approval of Libmeldy® by EMA - the first gene therapy product for metachromatic leukodystrophy. Ran has also held leadership positions at several major biotechnology companies including Genzyme (now Sanofi) and Amgen. At Amgen, Ran played a key role in building differentiating capabilities in manufacturing for clinical supply and commercial product launch to enable speed to clinic and speed to market strategies for Amgen’s innovative products.

    We have entered the golden age of biology with an unprecedented explosion of discoveries that could lead to life changing therapies and have profound impact on human health. However, the shortage of manufacturing capacity and expertise in recent years has constrained therapeutic translation especially for the life sciences innovators who explore emerging modalities and unique technologies. Manufacturing scalability, reproducibility and the cost of goods remain as major challenges, while the evolving science and regulatory landscape require continuous learning. 

    In 2021, Harvard and MIT partnered with Cytiva, Fujifilm Diosynth Biotechnologies and Alexandria Real Estate Equities to bring the best of academia, the life sciences industry, and world-class research hospitals together to accelerate life sciences innovation. Landmark Bio was established to break down the barriers in novel therapeutics development and industrialization. We collaborate with academics, startups and drug developers to take groundbreaking discoveries from bench to clinics by providing capacity and expertise in CMC development and manufacturing. Uniquely positioned as pre-platform and technology agnostic, we help design, de-risk, and develop early innovations into future platforms. Our cross-sector partnership offers an unparalleled academic, industry, hospitals and investor network with a magnetic pull to power biomanufacturing innovation. As a mission-driven organization, we embrace risk and opportunities at the bleeding-edge in order to advance emerging technologies, demonstrate therapeutic potential and improve human health.

    2:00 PM - 2:30 PM
    Professor of Electrical Engineering and Professor of Biological Engineering
    Jongyoon Han
    Professor of Electrical Engineering and Professor of Biological Engineering

    Dr. Jongyoon Han is currently a professor in the Department of Electrical Engineering and Computer Science and the Department of Biological Engineering, Massachusetts Institute of Technology. He received B.S.(1992) and M.S.(1994) degree in physics from Seoul National University, Seoul, Korea, and Ph.D. degree in applied physics from Cornell University in 2001. He was a research scientist in Sandia National Laboratories (Livermore, CA), until he joined the MIT faculty in 2002. He received NSF CAREER award (2003) and Analytical Chemistry Young Innovator Award (ACS, 2009). His research is mainly focused on applying micro/nanofabrication techniques to a very diverse set of fields and industries, including biosensing, desalination / water purification, biomanufacturing, dentistry, and neuroscience. He is currently the lead PI for MIT’s participation for NIIMBL (The National Institute for Innovation in Manufacturing Biopharmaceuticals).

    One of the critical challenges in cell therapy is the lack of reliable, specific, and non-destructive quality attributes, which are sorely needed for all aspects of biomanufacturing of these cells, including donor selection, in-process quality monitoring, and release testing. Most biological and biochemical assays are destructive or perturbative, which limits their utility, especially for autologous cell therapy. In this talk, I will showcase some of the emerging ideas of label-free, biophysical critical quality attributes (CQAs) we have been working on, including magnetic, electrical, and mechanical signatures of cells. Once the strong correlation with biochemical cell phenotypes, these will serve an important role to improve the overall production of both allogeneic and autologous cell therapy products.

    2:30 PM - 3:00 PM
    Core Member, Broad Institute of MIT and Harvard
    Investigator, McGovern Institute for Brain Research, MIT
    James and Patricia Poitras Professor of Neuroscience at MIT
    Associate Professor, Departments of Brain and Cognitive Sciences and Biological Engineering, MIT
    New York Stem Cell Foundation-Robertson Investigator
    Feng Zhang
    Core Member, Broad Institute of MIT and Harvard
    Investigator, McGovern Institute for Brain Research, MIT
    James and Patricia Poitras Professor of Neuroscience at MIT
    Associate Professor, Departments of Brain and Cognitive Sciences and Biological Engineering, MIT
    New York Stem Cell Foundation-Robertson Investigator

    Feng Zhang attended Harvard University as an undergraduate, where he earned an A.B. in Chemistry and Physics in 2004. He transitioned to Stanford for his Ph.D. in Chemistry and Biophysics, which he obtained in 2009 while working in Karl Deisseroth’s research group for his work  pioneering optogenetics in conjunction with Ed Boyden. After a postdoc in George Church’s lab, he moved to MIT in January of 2011, where he is now both an Associate Professor of Biological Engineering and a Core Institute Member of the Broad Institute of MIT and Harvard.

    Feng Zhang has received a number of honors, including the NIH Pioneer Award and the Perl-UNC Neuroscience Prize, along with Ed Boyden and thesis advisor Deisseroth. He is additionally a Searle Scholar.

    Many powerful molecular biology tools have their origin in nature, and, often, microbial life. From restriction enzymes to CRISPR-Cas9, microbes utilize a diverse array of systems to get ahead evolutionarily. We are interested in exploring this natural diversity through bioinformatics, biochemical, and molecular work to better understand the fundamental ways in which living organisms sense and respond to their environment and ultimately to harness these systems to improve human health. Building on our demonstration that Cas9 can be repurposed for precision genome editing in mammalian cells, we began looking for novel CRISPR-Cas systems that may have other useful properties. This led to the discovery of several new CRISPR systems, including the CRISPR-Cas13 family that target RNA, rather than DNA. We developed a toolbox for RNA modulation based on Cas13, including methods for precision base editing. We are expanding our biodiscovery efforts to search for new microbial proteins that may be adapted for applications beyond genome and transcriptome modulation, capitalizing on the growing volume of microbial genomic sequences and building on our bioengineering expertise. We are particularly interested in identifying new therapeutic modalities and vehicles for delivering cellular and molecular cargo. We hope that this combination of tools and delivery modes will accelerate basic research into human disease and open up new therapeutic possibilities.

    3:00 PM - 3:20 PM

    Networking Break
    3:20 PM - 3:50 PM
    Professor of Biology, Koch Institute for Integrative Cancer Research
    MIT Department of Biology
    Jianzhu Chen
    Professor of Biology, Koch Institute for Integrative Cancer Research
    MIT Department of Biology

    Jianzhu Chen is Professor of Biology at Koch Institute for Integrative Cancer Research and Department of Biology at Massachusetts Institute of Technology (MIT). Dr. Chen received a Ph.D. degree from Stanford University. He was a postdoctoral fellow and then an instructor at Harvard Medical School before he joined the faculty at MIT. Dr. Chen’s research seeks fundamental understanding of the immune system as well as its application in disease intervention. Over the years, Dr. Chen has made significant contributions to a broad area of research in immunology, cancer research, infectious diseases, and animal models of human diseases. His research on lymphocyte homeostasis and immunological memory challenged the prevailing paradigm at the time and revealed unexpected effect of lymphopenia-induced proliferation on memory T cell development. Dr. Chen pioneered innovative research in development of novel mouse models through genetic manipulations and cell complementation and in development of novel anti-microbial, including surface coatings that inactivate microbes on contact, universal anti-influenza siRNAs, and polymer-attached antivirals that minimize drug resistance of influenza viruses. Recently, Dr. Chen’s research activity has focused on development of tumor-specific CAR-NK cells and re-programming macrophages for disease intervention, including cancer, metabolic diseases and infectious diseases.

    Natural Killer (NK) cells and CD8+ cytotoxic T cells are two types of immune cells that can kill target cells through similar cytotoxic mechanisms. With the remarkable success of chimeric antigen receptor-engineered T (CAR-T) cells for treating hematological malignancies, there is a rapidly growing interest in developing CAR-engineered NK (CAR-NK) cells for cancer therapy. Compared to CAR-T cells, CAR-NK cells could offer some significant advantages, including (1) better safety, such as a lack of or minimal cytokine release syndrome and neurotoxicity in autologous setting and graft-versus-host disease in allogeneic setting, (2) multiple mechanisms for activating cytotoxic activity, and (3) high feasibility for “off-the-shelf” manufacturing. We are developing the next generation of CAR-NK cells by combining tumor-specific CAR, additional armors, and cytokine-induced memory-like (CIML) NK cells, with a goal to achieve better tumor-specific targeting, enhanced proliferation and persistence in vivo, resistance to the suppressive tumor microenvironment, and ultimately an effective and durable anti-tumor response in patients.

    Presentation
    3:50 PM - 4:20 PM
    Faculty Research Officer
    Gilliland Professor, MIT Department of Chemical Engineering
    Richard Braatz
    Faculty Research Officer
    Gilliland Professor

    Richard D. Braatz is the Edwin R. Gilliland Professor of Chemical Engineering at MIT, where he conducts research into advanced biomanufacturing systems. He leads process data analytics, mechanistic modelling, and control systems activities for projects on monoclonal antibody, vaccine, and gene therapy manufacturing. Dr. Braatz received an M.S. and Ph.D. from the California Institute of Technology and was a Visiting Scholar at Harvard University before moving to MIT. He has collaborated with more than 20 companies including Novartis, Pfizer, Merck, Bristol-Myers Squibb, Biogen, Amgen, Takeda, and Abbott Labs. Honors include the AIChE PD2M Award for Outstanding Contribution to QbD for Drug Substance, the AIChE Excellence in Process Development Research Award, the Technical Innovation Award from the International Society of Automation, the IEEE Control Systems Society Transition to Practice Award, and election to the U.S. National Academy of Engineering.

    Ph.D. student in chemical engineering at MIT
    Tam Nguyen
    Ph.D. student in chemical engineering at MIT

    Tam Nguyen is a Ph.D. student in chemical engineering at MIT, where she does research in biopharmaceutical manufacturing systems. Her main research is in mechanistic modeling for AAV-based transfection and its use in improving understanding, optimization, and control of AAV capsid production. Tam received a Bachelors of Science in Chemical Engineering from the University of Massachusetts Amherst and a Masters of Science in Chemical Engineering Practice from MIT. Richard Braatz is the Edwin R. Gilliland Professor of Chemical Engineering at MIT, where he conducts research into advanced biopharmaceutical manufacturing systems. In this role, he leads process data analytics, mechanistic modeling, and control systems for many projects, including on monoclonal antibody, viral vaccine, and gene therapy manufacturing within the Center of Biomedical Innovation, and on protein crystallization, continuous lyophilization, and therapeutic protein formulation within the Department of Chemical Engineering.

    Recombinant adeno-associated virus (rAAV) is one of the most commonly used platforms for in vivo gene therapy treatments. The reduced toxicity, robust and long-term transgene expression, and ability to transduce both dividing and non-dividing cells as well as target a wide range of tissues have made rAAV the most widely used viral vector. However, the standard method of producing rAAV via transient transfection of mammalian cells, specifically human embryonic kidney 293 (HEK293) cells, typically has low yield and generates a high portion of empty particles, laying extra burden on downstream processing. To elucidate the mechanisms of rAAV synthesis in HEK293 suspension-adapted cells, we have developed a mechanistic model based on the published understanding of the underlying biology and existing data. Quantitative analysis suggests the misaligned dynamics of capsid and viral DNA production result in the high ratio of empty particles. Through a model-based strategy, we explored a novel transfection method using low-dose multiple transfections in HEK293 cell culture that successfully increased the ratio of full to empty capsids in the viral harvest without compromising the viral titer. Molecular analysis through a next-generation rAAV production model attributed the improvements to changes in the kinetics of viral protein expression and DNA replication. Here, we demonstrate that the use of multiple transfection times is a practical method for increasing the genome titer and improving the percentage of full capsids for rAAV production. Our results also demonstrated the capability to manipulate product composition from an operational standpoint.

    Presentation
    4:20 PM - 4:50 PM
    Associate Professor of Biological Engineering, MIT Department of Biological Engineering
    Michael Birnbaum
    Associate Professor of Biological Engineering

    Michael obtained an A.B. in Chemical and Physical Biology at Harvard University in 2008. He then moved to Stanford University, where he completed his Ph.D. in Immunology in 2014. At Stanford, he worked in Professor K. Christopher Garcia’s laboratory, studying the molecular mechanisms of T cell receptor recognition, cross-reactivity, and activation. He then conducted postdoctoral research in Professor Carla Shatz’s laboratory, studying novel roles for immune receptors expressed by neurons in neural development and neurodegenerative disease. Michael joined the Department of Biological Engineering in 2016 as an Assistant Professor.

    Cell and gene immunotherapies are revolutionizing how we treat disease, with multiple FDA-approved therapies that have transformed cancer treatments. However, advances in gene delivery, manufacturing, and therapeutic cargoes are still required to increase the impact and scope of these promising approaches. The Birnbaum laboratory is working to develop approaches that improve the specificity of cellular engineering, and the potency of cells once engineered.

    4:50 PM - 6:00 PM

    Closing Remarks and Networking Reception