Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.

With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit.  | Rocket ...

Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Five-year clinical study results show this revolutionary one-time treatment allowed 94% of patients to stop routine factor IX prophylaxis DETROIT, March 23, 2026 /PRNewswire/ -- The Barbara Ann ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working ...

Rocket Pharmaceuticals (NASDAQ:RCKT) said the U.S. Food and Drug Administration has granted accelerated approval to KRESLADI ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

It involves substances “not for human use.” ...