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

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

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Genetic factors are thought to play a major role in the development of autism – but for decades what they are has proven elusive. Now scientists are starting to uncover clues.

A team at CNIC has developed an innovative gene therapy to treat arrhythmogenic right ventricular cardiomyopathy type 5 ...

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

Hemophilia represents a rare, lifelong bleeding disorder characterized by the blood's inability to clot normally. While management strategies have advanced ...

Investigators from Mass General Brigham and Beth Israel Deaconess Medical Center have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without ...

From STAT’s Matthew Herper: Pfizer said this morning that it would stop development of danuglipron, its experimental oral GLP-1 medicine to treat obesity, focusing its efforts on another medicine with ...

Genflow Biosciences Signs MSA with Heureka Labs, Launching AI-Powered Partnership in Gene Therapy Research ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy ...