Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...

These 10 stocks could mint the next wave of millionaires › Ocugen (NASDAQ:OCGN), a biotechnology company developing gene therapies and regenerative medicine solutions for eye diseases, released its ...

Former FDA Commissioner Dr. Scott Gottlieb joins 'Squawk Box' to discuss news of President Trump ordering pharma companies to ...

Gain insights from Ocugen's Q2 2025 earnings call, highlighting clinical advancements, strategic partnerships, BLA milestones for 2026-2027, and ...

Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.

If successful, the access model for sickle cell disease gene therapies could serve as a blueprint for other such treatments ...

In a first-of-its-kind clinical trial, UCLA scientists have shown it's possible to reprogram a patient's blood-forming stem ...

Prasad, a longtime critic of the FDA’s leniency in drug approvals, joined the agency in May. WASHINGTON — The Food and Drug ...

Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s executive leadership team will host the call, which ...