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 ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

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

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

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 ...

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

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

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

The $150 million will also go toward Atsena’s preclinical pipeline and expand the use of the biotech’s spreading AAV.SPR ...

Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts ...