Shares of biotech firms focused on gene editing have been heating up this month, largely on the back of the FDA moving forward with more gene therapy approvals. Though the scope of Duchenne muscular dystrophy patients Sarepta’s SRP-9001 may be able to treat has been scaled back by regulators, an approval remains on the table for next month. That follows news that Krystal’s Vyjuvek, a treatment for dystrophic epidermolysis bullosa, would become the first FDA-approved topical gene therapy.
Venture capital interest in gene editing is ramping up again lately as well. Both of 2023’s largest private biotech fundraising efforts have been announced throughout the past week. ReNAgade Therapeutics and ElevateBio each raised $300 million and $401 million in series A and D rounds, respectively.
Related ETF: ARK Genomic Revolution ETF (ARKG)
As MRP noted last December, US Food and Drug Administration (FDA) approvals for genetic medicine became quite common in the latter half of 2022. Per BiopharmaDive, the FDA had approved more gene therapies for inherited diseases in the prior four months than it had in the five years prior. Increased confidence shown toward gene therapies at the FDA, as well as within capital markets, has continued in 2023 and seems to be picking up steam recently.
Earlier this month, shares of Sarepta Therapeutics surged on news that a slim majority of an FDA advisory committee, consisting of 8 out of 14 advisers, voted in favor of supporting an accelerated approval for Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) – known as SRP-9001. Reuters notes that the FDA often follows the advice of its expert advisers and is slated to make a decision on accelerated approval by June 22.
That date was initially May 29, but the FDA decided to delay the deadline on Wednesday, pummeling the stock price. Moreover, Sarepta has signaled that if the FDA does approve the gene therapy, it will do so for a far narrower group of patients than the company had hoped. Barron’s notes that Sarepta had tested SRP-9001 in patients 4 to 7 years old, and asked for approval of the gene therapy in all DMD patients who can still walk, a category that could include some patients as old as 13. However, accelerated approval…
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