An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

The 23-year-old man received a gene therapy transfusion Wednesday at Manning Family Children's Hospital that doctors say should cure his sickle cell disease. It’s a condition he has lived with his ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Please provide your email address to receive an email when new articles are posted on . This is the first biologics license application submitted for a gene-agnostic retinal disease gene therapy.

Ocugen lines up key gene therapy catalysts through 2027, with pivotal data, filings, and milestones across three eye programs set to drive stock volatility.

Ocugen OCGN is building its ocular portfolio around a modifier gene therapy idea: instead of designing a therapy for a single genetic defect, the platform is intended to work across many underlying ...

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...

Current GT strategies employ viral vectors (eg, murine g-retroviruses or lentiviruses) to introduce genetic material into isolated HSCs. However, gene editing (eg, CRISPR-based GT) is under active ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for ...

Belief BioMed ("BBM") today announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by the Pharmaceutical Administration Bureau of Macao Special ...

The only drug-related adverse events observed are typically anticipated with gene therapy administration, the company noted. ・It added that a proactive, short-course immune modulation regimen in ...