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 ...

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

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

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.

The 23-year-old man received a gene therapy transfusion Wednesday at Manning Family Children's Hospital that doctors say should cure his sickle cell disease. It’s a condition he has lived with his ...

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

Current GT strategies employ viral vectors (eg, murine g-retroviruses or lentiviruses) to introduce genetic material into isolated HSCs. However, gene editing (eg, CRISPR-based GT) is under active ...

Ocugen OCGN is building its ocular portfolio around a modifier gene therapy idea: instead of designing a therapy for a single genetic defect, the platform is intended to work across many underlying ...

Scientists have developed a method to reprogram cancer-fighting immune cells directly inside the body, potentially eliminating the need for complex lab-based manufacturing. Early results show rapid ...

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 ...