CRISPR Cas9 gene therapy explained with DNA scissors, hereditary diseases treatment, and designer babies ethical dilemmas ...

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 ...

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

A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.

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 ...

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 ...

Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin ob ...

Technologies for the development of new RNA treatments and gene therapy are marking new milestones in Italy, with the ...

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 ...

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

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 ...