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.

A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...

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

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

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

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.

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...