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Thanks to gene therapies, previously untreatable diseases are now treatable and patient quality of life has improved exponentially. Yet, gene therapy regulatory challenges, along with R&D, manufacturing, and the realities of the patient populations, are keeping the game-changing sector from realizing its full potential.
During the BIO International Convention in June 2022, Pfizer’s Robert Smith moderated a panel discussion, Realizing the Promise of Gene Therapy: Current Challenges, Opportunities and Trends. The panelists included:
- Janet Lambert, CEO of the Alliance for Regenerative Medicine
- Debra Miller, CEO & Founder of CureDechenne
- Ruhi Khan, Chief Business Officer at Caribou Biosciences, Inc.
- Albert Hwang, MD and Managing Director for the Healthcare Investment Banking at Morgan Stanley
- Peter Marks, MD, PhD, and the Director at the Centers for Biologics Evaluation & Research (CBER), US Food & Drug Administration (FDA)
The gene therapy regulatory landscape today
The landscape of gene therapy development is changing, creating a variety of dynamic and unique challenges.
“Our global data of about 3100 companies worldwide has shown that a little over half of gene therapy-focused biotech companies are in the United States, about 30% are in the South Pacific, and a little under 20% are in Europe, which is considerably less than there used to be,” said Janet Lambert of the Alliance for Regenerative Medicine.
But this is a profoundly complicated global market, which means that raising capital, shoring up and standardizing manufacturing, and pairing manufacturing needs with robust and actively up-to-date regulation are paramount.
“Regulatory delays when it comes to manufacturing issues is a big pain point,” added Lambert.
The FDA’s Peter Marks agreed: “Manufacturing and regulatory interface is still a major pain point,” he said. “What the United States regulatory agencies can do is to find ways to standardize processes to try to decrease regulation. And if we could find a way that we didn’t have to reinvent the manufacturing process each time, maybe that would be a way of decreasing regulatory burden and therefore allow rare disease gene therapy development to move forward faster.”
How gene therapy regulatory challenges impact patients
Debra Miller of CureDechenne pointed out a major barrier for the gene therapy field: gene therapies are largely for small patient populations who have very rare diseases. This exacerbates manufacturing and regulatory issues.
However, she acknowledged that the heart of the field is patient-focused: “The biotech and biopharma companies we work with really do focus on what is important for the patient,” she said. “It is something that I am obviously very pleased about. In terms of where we are now, it’s two steps forward and one step back. The development of gene therapy is still slow, especially when you are a parent, and there are overwhelming challenges associated with the science, manufacturing, as well as patient toxicity. These are things we really have to think about as we invest, support, and move forward. Regardless, we’ve come really far, but there is still a long way to go.”
“One challenge in the oncology space is that when regulations are lagging, sponsors are forced to pigeonhole development into outdated or inconsistent guidelines,” said Ruhi Khan of Caribou Biosciences, “which then burdens the FDA more as they need to catch up.”
These barriers trickle down to the patient level as well when it comes to the affordability of and access to gene therapies. This is where institutions like the FDA, CMS, and Medicaid come in.
“It is heartbreaking to see all of this work go into gene therapies and then ultimately see them taken off of the market because they were not successfully delivered to the patients,” Lambert said.
However, the overwhelming consensus was that, despite the challenges, the gene therapy field is committed to opening up communication lines to improve the field and save lives.
“The sector is changing,” said Lambert, “But it is still a strong sector with a very strong pipeline and clinical trials are indicating that things are changing in meaningful ways.”
