Gene therapy
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.
A push to reshore some drug production and progress in advanced manufacturing technologies have been prominent trends this year, industry leaders say.
Of all the stories we published this year, these deep dives by BioSpace editors stand out as relevant re-reads going into the New Year.
Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector.
PRESS RELEASES