Gene therapy
After years of contraction, investors see biotech reentering a growth cycle driven by scientific progress, asset quality and renewed conviction in oncology, obesity and neuroscience innovation.
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.
Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for this year.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
Aurora joins the clutch of companies linked to Nobel Prize winner and CRISPR trailblazer Jennifer Doudna.
Rampart Bioscience was working on a platform to deliver gene therapies without the need for viral vectors.
Industry leaders are focused on the resilience of key starting material supply and the knock-on effects of automation in the new year.
With a pair of Phase III trial flops, Ultragenyx will explore cost reductions as analysts turn attention to an upcoming Angelman syndrome readout.
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