Will consider data that includes "the experience of individuals," FDA commissioner says ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...

RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

When Doug Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

PDE6A-associated RP, a rare form of inherited retinal disease (IRD), is characterized by nyctalopia, visual field defects, and significant loss of visual acuity (VA). The disease primarily affects the ...

Ultragenyx Pharmaceutical's employees were already braced for changes as the biopharma considered how to rebound from a pair ...

Catalent is cutting more roles at its gene therapy manufacturing facility in Harmans, Maryland. | Catalent is cutting more roles at its gene therapy manufacturing facility in Harmans, Maryland. This ...

During a February 2026 appearance on the What Matters With Liz podcast , Winkler, 80, admitted that his Emmy-winning role on ...

Real-world evidence links higher injection frequency to better VA outcomes and reduced CST fluctuations, reinforcing ...

Q4 2025 earnings call recap: revenue beat, 2026 guidance, margin expansion outlook, and product pipeline catalysts.