Etranacogene dezaparvovec (Hemgenix), the only commercially available gene therapy for hemophilia B, is temporarily ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

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

OCGN's gene-agnostic eye therapy aims to treat multiple retinal mutations, with key filings and pivotal data in 2026???2027 set to test its broad platform.

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

He is the first person in the state to receive LYFGENIA, a one-time gene therapy designed to keep the red blood cells that ...

The only drug-related adverse events observed are typically anticipated with gene therapy administration, the company noted. ・It added that a proactive, short-course immune modulation regimen in ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

Nemours Children's Hospital is now offering Lyfgenia gene therapy. The treatment aims to reduce pain crises and hospital visits for young patients.

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a ...