REGENXBIO
NEWS
Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.
In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs.
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
Gene therapies developed by Janssen, Atsena, REGENXBIO and AbbVie showed promise against eye diseases this weekend at the American Academy of Ophthalmology 2022 meeting.
It’s been a busy week for research on rare diseases, with several clinical trials from AnaptysBio, Regenxbio, Sangamo and more posting results from ongoing activities.
CRD’s CRD-TMH-001 is expected to upregulate an alternate form of the dystrophin protein with CRISPR technology and stabilize or reverse the symptoms associated with DMD.
Ayala announced positive data for its desmoid tumor treatment, while Vertex and Dyne announced the FDA had lifted the hold on their clinical studies.
BioSpace looks at several companies developing new gene therapies in the wake of the FDA recommending two of bluebird bio’s lentiviral vector gene therapies.
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