Ten out of 11 kids who received gene therapy for a rare variant of congenital deafness enjoyed significant improvements when ...

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

CROs can contribute to site collaboration by also identifying and supporting non-academic institutions capable of conducting ...

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

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

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

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

Despite the challenges, including what CSL CEO Paul McKenzie has described as a fragmented U.S. healthcare system, the blood ...

Maria Lowe, Ph.D., associate vice president of Pharmaceutical Intelligence at Institute for Clinical and Economic Review ...

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 ...

Verve Therapeutics said Monday that initial data show that its investigational gene-editing therapy lowered cholesterol without inducing serious side effects.

Gene therapy companies have faced numerous setbacks over the last few years. What will it take for gene therapies to fulfill ...