Approaches in Cell Therapy and Regenerative Medicine

September 23, 2020
Approaches in Cell Therapy and Regenerative Medicine
Webinar


Location

Zoom Webinar

 

 


Overview

In the last decade, the science of cell therapy and regenerative medicine has made remarkable progress. Work in this area has also resulted in numerous engineering successes, enabling sophisticated tools that open the door to new approaches for discovery and development of therapeutics. Particularly noteworthy is the recent progress in cell and molecular biology, including in stem cell research and gene editing. In this webinar, we will hear about new tools and the new directions they make possible as well as how each is being commercialized.

How can small molecules be identified that evoke cell or tissue regeneration by design? How can we engineer cells and tissue-growth in situ with a structure suitable for implantation? How can we physically gain access to the interior of cells for both discovery and engineering purposes? And how can the immune system be mapped with single-cell biology to accelerate discovery?

  • Overview

    In the last decade, the science of cell therapy and regenerative medicine has made remarkable progress. Work in this area has also resulted in numerous engineering successes, enabling sophisticated tools that open the door to new approaches for discovery and development of therapeutics. Particularly noteworthy is the recent progress in cell and molecular biology, including in stem cell research and gene editing. In this webinar, we will hear about new tools and the new directions they make possible as well as how each is being commercialized.

    How can small molecules be identified that evoke cell or tissue regeneration by design? How can we engineer cells and tissue-growth in situ with a structure suitable for implantation? How can we physically gain access to the interior of cells for both discovery and engineering purposes? And how can the immune system be mapped with single-cell biology to accelerate discovery?

Register

Agenda

11:00am

Towards Druggable Tissue Regeneration
Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School
Principal Faculty, Harvard Stem Cell Institute
Affiliate faculty, Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology
Jeffrey M. Karp
Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School
Principal Faculty, Harvard Stem Cell Institute
Affiliate faculty, Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology

Dr. Jeff Karp is a Professor of Medicine at Brigham and Women’s Hospital, Harvard Medical School. He is also a principal faculty member at the Harvard Stem Cell Institute, and an affiliate faculty member at the Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology.

He works in the fields of drug delivery, medical devices, stem cell therapeutics, and tissue adhesives. He has published over 125 peer-reviewed papers, with >21,500 citations, and has given over 300 invited lectures. He has over 100 issued or pending national and international patents. Several technologies developed in his lab have led to multiple products currently in development or on the market and for the launch of eight companies that have raised over $400 million in funding. Technologies include high-tech skincare (Skintifique, products sold in pharmacies throughout EU), tissue adhesives (Tissium, EU Approval in 2017) and 3D printed biomedical devices, immunomodulation with biologically responsive materials (Alivio Tx), small molecule regenerative therapeutics ($FREQ – NASDAQ), cannabinoid therapeutics (Molecular Infusions acquired by Suterra Wellness in 2019), biomedical devices to improve child safety (Landsdowne Labs), needles that automatically stop at their target (Bullseye Therapeutics), and a bioengineered luminal coating for controlled GI targeting (Altrix Bio).

Karp has received >50 awards and honors. Most recently Jeff received the highest award from the Society For Biomaterials for innovation – the Clemson Award for Applied Research.  Boston Magazine recognized Karp as one of 11 Boston Doctors Making Medical Breakthroughs. The Boston Business Journal recognized him as a Champion in Healthcare Innovation and MIT’s Technology Review Magazine (TR35) also recognized Karp as being one of the top innovators in the world (three members from his laboratory have subsequently received this award). Karp was elected to the American Institute for Medical and Biological Engineering’s College of Fellows in 2013, a fellow of the Biomedical Engineering Society (BMES) in 2018, and a Fellow of the Royal Society of Chemistry and the Canadian Academy of Engineering in 2019. His work has been selected by Popular Mechanic's as one of the Top 20 New Biotech Breakthroughs that Will Change Medicine. He gave a commencement speech at his high school in 2011, and a TEDMED talk in 2014 on bioinspired medical innovation, and since 2015 has been a member of the TEDMED Editorial Advisory Board. In 2015 and 2016, he received Breakthrough Awards from the Kenneth Rainin Foundation, and in 2015 was a commencement speaker at the University of Toronto Faculty of Dentistry and Pharmacy. Karp also won an internal Shark Tank award judged by Kevin O’Leary (from ABC’s Shark Tank). In 2018 Karp gave a TEDx talk on Radical Simplicity. Furthermore, in 2019 Karp was a grade 8 commencement speech at the Talcott Mountain Academy in Connecticut.

In addition to his research goals, Karp is dedicated to developing the careers of the next generation bioengineers at the forefront of regenerative medicine. He was selected as the Outstanding Faculty Undergraduate Mentor among all faculty at MIT and he received the HST McMahon Mentoring award for being the top mentor of Harvard-MIT students. To date, 22 trainees from his laboratory have secured faculty positions.

One of the holy grails in medicine is to achieve tissue regeneration. Traditionally involving complex manipulation and manufacturing of cells outside of the body, stem cell approaches are very promising. Avoiding the complexity, we have focused on delivering combinations of small molecules to target stem cells and progenitor cells in the body. Through this work we identified small molecules that can regenerate hair cells in the inner ear to functionally restore hearing.  Hearing loss affects hundreds of millions of people and cochlear implants and hearing aids have severe limitations. This platform technology formed the basis for a startup company called Frequency Therapeutics in 2015 that IPOed on the NASDAQ in 2019. The company advances regenerative small molecule therapeutics through targeting and manipulating stem cells and progenitor cells in situ.

11:25am

Startups pitches
  • Cellino: Image-guided, laser-driven manufacturing of iPSCs and iPSC-derived tissues
  • Kytopen: The Future in Non-Viral Delivery for Cell Therapy Manufacturing
  • Immunai: Comprehensive mapping of the immune system with single-cell biology and AI
Marinna Madrid, Co-Founder, Cellino
Marinna Madrid
Marinna Madrid, Co-Founder, Cellino

Marinna Madrid is a co-founder at Cellino, a venture capital-backed early-stage biotech company. Cellino is combining machine learning, laser processing, and stem cell biology to develop and manufacture autologous iPSC-derived cell and tissue therapies at scale.

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, where she co-invented a cellular organelle isolation technique. 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, along with her co-founders.

CEO and Co-founder, Kytopen
Paulo Garcia
CEO and Co-founder, Kytopen

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.

Scientific co-founder of Immunai
Danny Wells
Scientific co-founder of Immunai

Danny Wells is the scientific co-founder of Immunai where he helps define the company vision and overall strategy. He is a recognized leader in the application of genomics - in particular, single cell genomics - to the study of cancer immunotherapy resistance. He has published papers in Nature, Nature Medicine, and Nature Immunology. He completed his PhD in applied math at Northwestern and his postdoc in cell biology at Berkeley.

11:50am

Panel Discussion: Challenges in the field
Marinna Madrid, Paulo Garcia, Danny Wells
12:05pm

Breakout Discussions
  • Agenda
    11:00am

    Towards Druggable Tissue Regeneration
    Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School
    Principal Faculty, Harvard Stem Cell Institute
    Affiliate faculty, Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology
    Jeffrey M. Karp
    Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School
    Principal Faculty, Harvard Stem Cell Institute
    Affiliate faculty, Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology

    Dr. Jeff Karp is a Professor of Medicine at Brigham and Women’s Hospital, Harvard Medical School. He is also a principal faculty member at the Harvard Stem Cell Institute, and an affiliate faculty member at the Broad Institute and at the Harvard-MIT Division of Health Sciences and Technology.

    He works in the fields of drug delivery, medical devices, stem cell therapeutics, and tissue adhesives. He has published over 125 peer-reviewed papers, with >21,500 citations, and has given over 300 invited lectures. He has over 100 issued or pending national and international patents. Several technologies developed in his lab have led to multiple products currently in development or on the market and for the launch of eight companies that have raised over $400 million in funding. Technologies include high-tech skincare (Skintifique, products sold in pharmacies throughout EU), tissue adhesives (Tissium, EU Approval in 2017) and 3D printed biomedical devices, immunomodulation with biologically responsive materials (Alivio Tx), small molecule regenerative therapeutics ($FREQ – NASDAQ), cannabinoid therapeutics (Molecular Infusions acquired by Suterra Wellness in 2019), biomedical devices to improve child safety (Landsdowne Labs), needles that automatically stop at their target (Bullseye Therapeutics), and a bioengineered luminal coating for controlled GI targeting (Altrix Bio).

    Karp has received >50 awards and honors. Most recently Jeff received the highest award from the Society For Biomaterials for innovation – the Clemson Award for Applied Research.  Boston Magazine recognized Karp as one of 11 Boston Doctors Making Medical Breakthroughs. The Boston Business Journal recognized him as a Champion in Healthcare Innovation and MIT’s Technology Review Magazine (TR35) also recognized Karp as being one of the top innovators in the world (three members from his laboratory have subsequently received this award). Karp was elected to the American Institute for Medical and Biological Engineering’s College of Fellows in 2013, a fellow of the Biomedical Engineering Society (BMES) in 2018, and a Fellow of the Royal Society of Chemistry and the Canadian Academy of Engineering in 2019. His work has been selected by Popular Mechanic's as one of the Top 20 New Biotech Breakthroughs that Will Change Medicine. He gave a commencement speech at his high school in 2011, and a TEDMED talk in 2014 on bioinspired medical innovation, and since 2015 has been a member of the TEDMED Editorial Advisory Board. In 2015 and 2016, he received Breakthrough Awards from the Kenneth Rainin Foundation, and in 2015 was a commencement speaker at the University of Toronto Faculty of Dentistry and Pharmacy. Karp also won an internal Shark Tank award judged by Kevin O’Leary (from ABC’s Shark Tank). In 2018 Karp gave a TEDx talk on Radical Simplicity. Furthermore, in 2019 Karp was a grade 8 commencement speech at the Talcott Mountain Academy in Connecticut.

    In addition to his research goals, Karp is dedicated to developing the careers of the next generation bioengineers at the forefront of regenerative medicine. He was selected as the Outstanding Faculty Undergraduate Mentor among all faculty at MIT and he received the HST McMahon Mentoring award for being the top mentor of Harvard-MIT students. To date, 22 trainees from his laboratory have secured faculty positions.

    One of the holy grails in medicine is to achieve tissue regeneration. Traditionally involving complex manipulation and manufacturing of cells outside of the body, stem cell approaches are very promising. Avoiding the complexity, we have focused on delivering combinations of small molecules to target stem cells and progenitor cells in the body. Through this work we identified small molecules that can regenerate hair cells in the inner ear to functionally restore hearing.  Hearing loss affects hundreds of millions of people and cochlear implants and hearing aids have severe limitations. This platform technology formed the basis for a startup company called Frequency Therapeutics in 2015 that IPOed on the NASDAQ in 2019. The company advances regenerative small molecule therapeutics through targeting and manipulating stem cells and progenitor cells in situ.

    11:25am

    Startups pitches
    • Cellino: Image-guided, laser-driven manufacturing of iPSCs and iPSC-derived tissues
    • Kytopen: The Future in Non-Viral Delivery for Cell Therapy Manufacturing
    • Immunai: Comprehensive mapping of the immune system with single-cell biology and AI
    Marinna Madrid, Co-Founder, Cellino
    Marinna Madrid
    Marinna Madrid, Co-Founder, Cellino

    Marinna Madrid is a co-founder at Cellino, a venture capital-backed early-stage biotech company. Cellino is combining machine learning, laser processing, and stem cell biology to develop and manufacture autologous iPSC-derived cell and tissue therapies at scale.

    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, where she co-invented a cellular organelle isolation technique. 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, along with her co-founders.

    CEO and Co-founder, Kytopen
    Paulo Garcia
    CEO and Co-founder, Kytopen

    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.

    Scientific co-founder of Immunai
    Danny Wells
    Scientific co-founder of Immunai

    Danny Wells is the scientific co-founder of Immunai where he helps define the company vision and overall strategy. He is a recognized leader in the application of genomics - in particular, single cell genomics - to the study of cancer immunotherapy resistance. He has published papers in Nature, Nature Medicine, and Nature Immunology. He completed his PhD in applied math at Northwestern and his postdoc in cell biology at Berkeley.

    11:50am

    Panel Discussion: Challenges in the field
    Marinna Madrid, Paulo Garcia, Danny Wells
    12:05pm

    Breakout Discussions