The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got ...

When even one tiny section of a person’s genetic code becomes mutated or modified, cells can begin to act out of ...

AMT-130 is administered through magnetic resonance imaging-guided, convection-enhanced stereotactic neurosurgical delivery into the striatum.

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

Additionally, improving CAR-T biological efficiency could reduce dosing needs, further cutting costs and expanding access.

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

The FDA granted regenerative medicine advanced therapy designation to ATSN-201, a gene therapy candidate for the treatment of ...

Gene therapy is an area of intensive research and development in the retina space, with promising prospects and significant challenges. The benefits and pitfalls of gene therapy were discussed by two ...

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...