From The Editor | January 14, 2021

A Recap Of ARM's Cell And Gene Therapy State Of The Industry And Panel Discussion


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


During JP Morgan 2021, ARM’s CEO Janet Lambert and her team crafted an extremely informative Cell and Gene Therapy State of the Industry address. During her address, Lambert provided a look back at 2020’s highlights and challenges. She stated that COVID-19 provided disruptions to clinical trials. Regulatory was slammed with COVID-19-related demands and had less bandwidth for cell and gene therapies, and the political climate posed problems to sector. The sector had a recording-setting year in terms of financing with $20 billion in investment in this space — more than double in 2019. Public financing was especially strong in 2020, and IPOs were double the previous record set in 2018.

Lambert stated that 2021 will be a fantastic year in science, technology, and clinical progress. The sector will continue to overcome manufacturing challenges. She urged the necessity for the social context of our sector’s work, and one way of doing that is by requiring racial equality in clinical trials. She noted there is still much work to be done around patient access, as new political and regulatory leaders still have much to do to educate lawmakers and to shape an effective policy for it.

Panel: Looking Ahead – What to Expect for the Cell & Gene Therapy Sector in 2021 and Beyond

Terry-Ann (TA) Burrell, CFO at Beam Therapeutics moderated the discussion that followed Lambert’s Cell and Gene State of the Industry address. Marianne De Backer, Member of Executive Committee and EVP, Head of Strategy and Business Development & Licensing, Pharmaceuticals Division, Bayer; Sandy Macrae, President & CEO, Sangamo Therapeutics; Ken Mills, CEO, REGENXBIO; and Adrian Rawcliffe, CEO, Adaptimmune answered TA’s questions about what is anticipated for the cell and gene therapy sector in 2021 — big data readouts, technology trends, important commercialization challenges, etc.

TA kicked off by asking the panelists about the sector’s status is in the lifecycle of precision genetic medicines. Rawcliffe explained that cell and gene therapies are defined by the opportunity on a one-off basis to new or regain of function, and that as a modality, it fits into is biopharmaceuticals. “We have yet to convince everyone that there is still a lot of hard work to convert our amazing technologies into products that impact lives on a global scale.”

Valuation Success

Just as Lambert stated in her address, the panelists agree that the sector has seen and is poised for continued valuation growth. TA asked panelists to share their thoughts on the valuation potential is from a strategic perspective.

De Backer stated that the sector has modalities that allows it to treat diseases at their roots. There is an increased level of enthusiasm for financing, and this is an area that will alter standard of care for multiple diseases. “But when we are thinking about valuation, how can we think about structures with partners? We have tried to focus on value and clinical and technological milestones and others in this field. There is endless innovation to come, and the sector will be a seller’s market for many years to come.”

Mills referenced the breadth of tools that are available to bring therapies to life and that a growth area is larger indications as we are moving into millions of patient populations. “Think about how many more tools — and not just capital — we are accelerating along the growth curve much faster than traditional biologics,” says Mills.

Overcoming Challenges to Gene Therapy

Gene therapies have faced some challenges this year. Regarding larger indications, regulatory advancements need to be realized to move forward. “There will be three forms of medicine — small molecules to treat something; vaccines to prevent something; and then genomic medicines to prevent something,” states Macrae. “Those are the three modalities, that if we come back in ten years’ time, that’s what we will be talking about in terms of established modalities to address disease. Gene therapy is great, but it is only a transient place to pass through. For example, the number of diseases in the liver that have large enough patient populations that you can treat and take forward is tiny. There are 7,000 diseases in the liver, and only 20 are big enough, and all the companies are going to the same place. The future is not gene therapy, it is long-term editing and changes to the genome that allows the patient to walk away from their disease and lead a normal life.”

Macrae went on to address the regulatory environment commending Dr. Peter Marks and CBER for their work in the sector. He explained that the sector is moving on from a time from when it was all the science to now, which is all about the clinical results and producing clinical data. “Once you start to produce the clinical data, no one cares about which form of science produced it,” Macrae explains. “All that matters is that the patient feels better or live longer, and that’s how it should be. We’re all throwing up our hands to challenges to our dream programs simply because we have to prove they work and show them they work in patients. There are three fundamental things to remember —this is the future, the future is beyond deliver, and the future is determined by showing clinical efficacy.”

The Fast Track Keeps Moving Even Faster

The panel discussed how the regulatory environment can possibly keep up so that companies can fast track their therapies. The group discussed that it used to be that between ideation and IND, you lost 90% of products. With cell and gene therapy products, that is not the case. “You usually get them all the way to IND,” explains Macrae. “I do believe we need to be absolutely certain of what we’re doing in the clinic. The agency is doing a fantastic job. We have to show clinical data, and the agency always asks reasonable questions.”

Rawcliffe stated that the Agency will always require the data, and in the cell and gene therapy sector specifically, 80% of that is CMC and 20% is clinical data because the clinical data happens in smaller numbers because of the size of the signal. “There are many companies being born that have a piece of the puzzle. The key question they are asking is what does this piece of this puzzle do? A lot of the investment in early-stage companies is now going into integrated companies.”