Industry Insights
Latest in gene therapy and rare disease
Legend scientific founder returns to ASCO with new ambition for high-yield, non-gene-editing CAR-T platform
Nine years after wowing the audience at the American Society of Clinical Oncology annual meeting with a CAR-T candidate that would become Carvykti—now the world’s most successful cell therapy—Legend B
AstraZeneca, after phase 3 miss, posts subgroup data behind pitch for rare disease approval
AstraZeneca has shared data it believes can rescue anselamimab from a phase 3 failure, linking the rare disease drug candidate to a 62% improvement in survival in a prespecified subgroup.
FDA extends Beren’s rare disease review after info request, further stretching saga
The FDA has delayed its approval decision on Beren Therapeutics’ rare disease drug candidate, extending the asset’s long path toward the U.S. market.
FDA delays verdict on Beren’s rare disease drug by three months
Beren Therapeutics is seeking approval in Neimann-Pick disease type C, an application the FDA was set to decide on by August 17. The new target action date is November 17.
PRINCE: A Small-Molecule Switch for Safer Gene Editing
Researchers developed PRINCE, a small-molecule-controlled CRISPR system enabling precise, long-term regulation of gene editing. Its compact version, Little Prince, showed therapeutic potential in mous