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

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

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

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

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 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 ...

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

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

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

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 ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology. It uses electronics to step in as a replacement for the biological ear that ...

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science ...