Cell and gene therapy sector remains strong despite challenges: ARM CEO

June 30, 2022 -- The cell and gene therapy (CGT) sector is “quite strong” despite the regulatory, investment, and manufacturing challenges facing the industry, according to Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM).

"It wouldn't be realistic to not comment on the fact that the financing environment right now is challenging," Lambert told ScienceBoard.net. "A high interest environment always creates challenges for investors in biotech and I think we're seeing that play out. There are definite financing challenges for companies in the field."

At the same time, Lambert noted that the CGT sector in 2021 saw record investment of nearly $23 billion, a total of six new therapies approved, and several scientific breakthroughs.

In the last four years, Lambert said more than $60 billion of investment has flowed into the industry serving as "fuel in the tank for all the scientific progress that we're trying to achieve."

As evidence of the progress being made for cell, gene, and tissue-engineered therapies, Lambert pointed to the fact that there are 2,400 ongoing clinical trials globally, half of which are commercial trials and the other half sponsored by academic institutions. In addition, she said there are currently almost 150 phase III trials "to where they could realistically reach patients" with a "breadth of indications" for both prevalent and rare diseases.

"We're hoping that this is going to be a record-breaking year for new approvals for gene therapies targeting rare diseases," Lambert commented. "That said, we do see more and more clinical development focused on more prevalent diseases."

When it comes to challenges, Lambert said manufacturing continues to be a major obstacle for the gene therapy sector. Borrowing from a model created for monoclonal antibodies, ARM has released a similar document called "A-Gene," a best-practices guide to integrating quality by design principles in gene therapy around chemistry, manufacturing, and controls (CMC) programs.

"That was modeled after a sector-wide effort in the monoclonal antibodies space a couple of decades ago," Lambert said, who added that the gene therapy sector today is "in the same place" where monoclonal antibodies were in years past due to a lack of standardized methodologies and training. She also noted a number of regulatory "holdups" for gene therapies trying to come to market "have been the result of CMC challenges, questions raised by regulators."

Under Lambert's leadership over the past five years, ARM's membership has doubled to more than 425 members and the organization's budget and full-time staff has tripled.

In late April, ARM announced that after five years at the helm Lambert would be stepping down as CEO of the organization. ARM's board is currently conducting a search for Lambert's successor. Asked what's next for her, Lambert said she's looking forward to spending more time with her two almost-college-age children and an aging show horse.

Watch the video below to learn more.

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