News

Girl, 3, becomes youngest patient to receive groundbreaking treatment
A three-year-old girl has become the youngest patient in the UK to receive a groundbreaking gene therapy for a rare, ...
Devdiscourse4h
Key Rulings & Measures Shape US Economic and Regulatory Landscape
Recent developments in U.S. domestic affairs include a legal victory for Anthropic in an AI training lawsuit, Treasury ...
Mother’s delight as gene therapy offers hope to three-year-old
But after receiving the new gene therapy, called Upstaza, Gunreet has made progress that her mother thought would never be ...
FDA investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...
Jefferies Maintains a Buy on Taysha Gene Therapies (TSHA) With an $8 PT
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the 13 Best Long-Term Penny Stocks to Buy According to Analysts.
Fusion superkine and focused ultrasound could enable targeted, noninvasive therapy for glioblastoma
Researchers at VCU Massey Comprehensive Cancer Center and the VCU Institute of Molecular Medicine (VIMM) have discovered a ...
Sarepta Viral Vector Gets FDA Platform Technology Designation
Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops ...
The Scientist9h
Weaving the Fabric of Cancer Research with Synthetic DNA
Modern DNA synthesis platforms empower researchers to explore the molecular world, from next-generation sequencing to cell ...
Exosome therapy offers a promising new approach to sensorineural hearing loss
Sensorineural hearing loss (SNHL) is the most prevalent form of permanent hearing loss, affecting over 6% of the global ...
Scientists use gene editing to correct harmful mitochondrial mutations in human cells
In a step toward treating mitochondrial diseases, researchers in the Netherlands have successfully edited harmful mutations ...
Healio12h
Cell-based, gene therapy skin grafts heal large epidermolysis bullosa wounds
Genetically engineered skin grafts exhibited a long-term ability to heal large, chronic wounds in patients with recessive ...
Fierce Biotech12h
Sangamo preps FDA application for Fabry gene therapy on heels of phase 1/2 registrational data
Sangamo Therapeutics’ investigational gene therapy appears to improve kidney function at 52 weeks for patients with a rare genetic disorder.  | Sangamo Therapeutics’ investigational gene therapy ...