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

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 ...

A new gene therapy treatment developed by uniQuire has shown promise in reducing the progression of Huntington's Disease by ...

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

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

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Half of all babies diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency die within their first week of life Getty KJ Muldoon was diagnosed with severe carbamoyl phosphate ...

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

A new randomized study finds that a lab test that reads tumor genes can identify which patients with recurrent prostate ...

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 ...

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.