Cell and Gene Therapy
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The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September.
Johnson & Johnson secured rights to prizlon-cel in 2023 for $245 million upfront. The asset in June last year showed a 100% response rate as a second-line option in a Phase 1b study for 10 patients with relapsed or refractory large B cell lymphoma.
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
In November 2025, one patient died after receiving Intellia Therapeutics’ investigational gene silencer for transthyretin amyloidosis.
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor.
As part of the collaboration, Johnson & Johnson will have an exclusive option to acquire Sail Biomedicines for an additional $2.58 billion.
ElevateBio will be the primary source of Kyverna’s autologous CAR T cell therapy for anticipated U.S. and global launches in autoimmune indications.
In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor’s assertion that a Phase 3 trial met its primary outcome measures.
Safety will be the key determinant of in vivo CAR T therapies, according to H.C. Wainwright’s Mitchel Kapoor, who noted that convenience and efficacy won’t be enough to impress investors in this space. Eli Lilly, AbbVie, AstraZeneca and more are up for the challenge.
Poised to become the first company to achieve a regulatory filing for a cell therapy in autoimmune disease, Kyverna Therapeutics is building a pipeline-in-a-product to change the treatment paradigm.