Gene therapy
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
The AAV pullback comes amid Biogen’s aggressive cost-cutting campaign, which put some 1,000 jobs on the chopping block with the goal of generating $1 billion in savings by 2025.
Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the development of cell and gene therapies.
The FDA is hoping to repurpose GSK’s Wellcovorin for cerebral folate deficiency; Pfizer acquired fast-moving weight-loss startup Metsera for nearly $5 billion after suffering a hat trick of R&D failures; psychedelics are primed for M&A action and Eli Lilly may be next in line; RFK Jr.’s revamped CDC advisory committee met last week with confounding results; and Stealth secured its Barth approval.
Bluebird bio has re-emerged after a private equity buyout as Genetix Biotherapeutics, marking a return to its roots and a new path forward for manufacturing.
The White House is clamping down on pharma’s ability to buy new molecules from Chinese biotechs; Sanofi, Merck and others abandon the U.K. after the introduction of a sizeable levy; Novo CEO Maziar Mike Doustdar lays off 9,000 while the company presents new data at EASD; Capsida loses a patient in a gene therapy trial; and CDER Director George Tidmarsh walks back comments on FDA adcomms.
A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
Capsida has yet to disclose the exact cause of death. The patient had received the gene therapy CAP-002 for a type of epilepsy.
As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy.
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