Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...

Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite ...

Grace Science, LLC, a biotechnology company founded to develop novel therapies based on the function of NGLY1, announced ...

Key takeaways• Researchers at The Ohio State University College of Medicine and College of Engineering developed a novel gene ...

Scientists have developed a new gene therapy that quiets pain at its source in the brain—without the addictive risks of opioids. Using AI to map how pain is processed, they created a targeted “off ...

Company entered into an asset purchase agreement with Johnson & Johnson (J&J) to acquire all interests in botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP ...

RMAT designation enables intensified FDA engagement and potential accelerated pathways, implying preliminary clinical evidence for RTx-015 despite absent peer-reviewed efficacy details, endpoints, and ...

Clinically meaningful improvements in xerostomia symptoms measured by PRO Xerostomia Questionnaire (XQ) maintained out to 3 years post treatment ...

University of Virginia School of Medicine scientists have used a next-generation form of gene editing to fix the underlying cause of a severe form of epilepsy in lab mice.

Rocket Pharmaceuticals drops despite the FDA's accelerated approval of gene therapy Kresladi in severe leukocyte adhesion ...

At a first meeting on pediatric gene and cell therapy trials, participants discussed when enrolling children before adults is ethical and when to wait.

Presentation to include 3-year data from all cohorts of the Phase 1 AQUAx Clinical Study- Cohort-level data and individual patient data from ...