Despite the challenges, including what CSL CEO Paul McKenzie has described as a fragmented U.S. healthcare system, the blood product specialist remains hopeful that Hemgenix is here to stay. “I hope ...

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 ...

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

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

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 ...

Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as ...

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

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

Lexeo Therapeutics has posted updated data in Friedreich ataxia (FA) patients, linking a gene therapy to improvements on ...

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

Beam Therapeutics Inc. (NASDAQ:BEAM) on Saturday presented additional data from the Phase 1/2 clinical trial of BEAM-302 in ...