Results from a three-year trial suggest an experimental gene therapy for Huntington's disease can slow the progression of the ...

After three patients died, the maker of an investigational gene therapy stopped shipments of the drug after it refused to ...

Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the ...

There's a potential breakthrough for people living with hemophilia. The condition usually means regular infusions to prevent bleeding, and even then, patients have to be extremely careful to avoid ...

In a trial of people with early Huntington's, the newly developed therapy slowed the progression of symptoms by 75%.

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

Clinical trials of a groundbreaking new gene therapy for Huntington's disease have shown that it slows progression of the condition by 75 per cent after three years, researchers say.

A study led by Vincenzo Condello and Christofer Juhlin of the Karolinska Institutet explores why some papillary thyroid ...

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease that could help protect the brain from damage and preserve cognitive function ...

At the European Cell & Gene Therapy Summit 2025, funding challenges and innovation drove discussions on the sector’s future.

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Being proactive — especially when it comes to testing, process development and preparing for commercialization — can greatly decrease the risk that your CGT will receive a CRL.