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

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin ...

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 ...

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

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 ...

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

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 ...

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.” ...

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 ...