The New Wave of Cell and Gene Therapies

Bruce Levine

Bruce Levine, PhD 
President, ISCT
University of Pennsylvania
United States

A Q&A with Dr. Bruce Levine, ISCT President

Dr. Levine’s upcoming presidential plenary on May 26 at ISCT 2021 New Orleans VIRTUAL is titled Integrating Science, Regulatory Oversight, and Commercialization for the New Wave of Cell and Gene Therapies. 

Below, an exclusive interview giving you a sneak peek at the vision and objectives behind this exciting plenary, and the leaders that Dr. Levine has invited to discuss the future of cell and gene therapy:

Plenary Speakers

Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research
Food and Drug Administration
United States 

Peter Marks, MD, PhD, is the director of the Center for Biologics Evaluation and Research within the Food and Drug Administration. Dr. Marks and center staff are responsible for assuring the safety and effectiveness of biological products including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies. 

Arie Belldegrun, MD, FACS
Executive Chairman, Allogene
Founder and Past CEO of Kite Pharmaceuticals
United States

Arie Belldegrun, MD, FACS, is a scientific entrepreneur with a distinguished history in founding several successful ventures in the field of life sciences. Dr. Belldegrun’s leadership of Kite Pharmaceuticals led to a $11.9 billion acquisition from Gilead Sciences and the landmark approval of Yescarta, one of the first commercially available CAR-T therapies. 

Matthew Porteus, MD, PhD 
Co-Founder, CRISPR Therapeutics & Graphite Bio
Stanford University
United States

Matthew Porteus, MD, PhD, is a distinguished scientist and clinician whose current research targets genetic therapies to cure or treat infant diseases and genetic diseases that affect muscle, through cutting-edge work at the Porteus Lab, and through scientific-commercial initiatives with CRISPR Therapeutics and Graphite Bio. Dr. Porteus attends pediatric patients undergoing hematopoietic stem cell transplantation at the Lucille Packard Children’s Hospital. 

This past year has shown significant press attention in the cell and gene therapy space, with increased visibility for barriers in getting late-stage clinical products to the market,. It has also been a record breaking year for funding and number of active cell and gene therapy trials in the space.

Forecasts demonstrate that approvals-wise, we are looking at 10-20 items being considered per year by 2025. For some time now, we have been lining up to build theresearch and the knowledge needed for cell and gene therapies to flourish, but now, the pivotal approvals and studies are beginning to happen. The questions we can ask now pivot towards how approval should be approached, how reimbursement might look, how patients can get access to therapies, and so on. 

Our speakers demonstrated a keen ability, in their respective areas, to adapt to the unexpected. They have a finger on the pulse for where we need to go. Each of them is a proven leader in their respective sphere of regulatory, commercial, and academic expertise, strengthened by experience across other parts of the translational chain:

  • Before his leading role in regulation, Dr. Marks had significant experience as a clinician-scientist, then in commercial roles in the development of hematological and oncological products, then working in a senior clinical role at a hospital.
  • Along with the mantle of business leadership, Dr. Belldegrun is an accomplished oncologist and urologist, with more than 500 publications related to urological cancers, immunotherapy, gene therapy, and cancer vaccines. 
  • Complimenting his scientific lab leadership, Dr. Porteus engages in commercial enterprise through co-founder roles in CRISPR Therapeutics and Graphite Bio, both companies working on the development of first-in-class genetic therapies. 

There are some very pertinent questions that any stakeholder in this sector might want to ask – for example, what does it take to go from a start-up conducting clinical trials to becoming a Gilead company in three years? How do we best address the challenge of regulatory oversight and approval despite an ongoing pandemic? What does the cutting edge of cell therapy need in order to scale up so that patients can access them? What have we learned about potency data?

That’s the tip of the iceberg. 

Since March, almost all of us have been operating in reduced capacity, distanced work environments, and missing the in-person connectedness that drives our work. 

We continue to find a way by focusing on the patients – how do we overcome challenges and build resilience as a community so that patients in need can access these therapies?
We focus on the bigger picture, and find other roads to reach it. 

Our speakers bring some very useful personal experiences to the table. Arie had been diagnosed with COVID-19 earlier in the year, while Peter has continued to navigate the balance between issuing Emergency Use Authorizations, political barriers, and the extreme demand for therapeutics targeted at addressing the pandemic. Matthew, meanwhile, has adapted the practices of both a research and clinical practice to a new normal with great success.

ISCT brings together a resilient community and a strong breadth of expertise. Our annual meetings offer a safe sphere of discussion to focus on challenges that lie ahead for the sector at large, with perspectives from industry, academic, and regulation. ISCT meetings are among the most open I have experienced; there is a sense of inclusiveness where you don’t need to know an inside group or be invited to get deeply involved. 

The community is cohesive and very supportive. Our #ISCTTogether campaign, and a series called “Reports from the Front Lines” speak to this – where we look at how we can support each other as people, rather than just as professionals. 

This year was challenging for everyone in the sector, but ISCT broke records thanks to its membership – and that group gives me optimism for the path forward. It’s also an inclusive Society. You don’t need to be invited or part of any exclusive ‘in-group’ to join as a member, and to attend meetings, discuss collaborations, and network in this community.  

We have time to plan, this year!

The COVID era has been a heavy weight, but I think this meeting is going to bring together a deep sense of community. We’re looking to build on top of the cutting-edge virtual platform to bring attendees an experience that brings both key lessons and enjoyment. 

ISCT 2020 Paris Virtual was a resounding success in this regard, and opened the doors for an audience larger than ever before to join us from around the globe, at the same time. We have a strong baseline to build on – and going forward, I’m hoping that ISCT 2021 sets the stage for us to reconnect and hit the ground running for 2022. 

What does the future hold? A hybrid event? Evolved meetings? 
We’re a truly global society, and the future of our meetings has never looked better for that.