If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

Crispr Therapeutics is an emerging gene editing company focused on the development of Crispr/Cas9-based therapeutics. The company's proprietary platform specializes in clustered regularly interspaced ...

Siren said proceeds will go toward investigational new drug (IND)-enabling animal studies and clinical-grade production of ...

Hathway’s technology uses natural epigenetic processes to dial gene activity up or down, rather than rewriting the genetic code. The company is using this to develop therapies for rare diseases and ...

Gene therapy developer CRISPR Therapeutics AG (CRSP) is navigating some regulatory and commercial dynamics. Its flagship gene ...

Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney, Australia announced the results of the longest follow-up study to ...

Japanese pharmaceutical company Takeda and domestic biotech company Belief BioMed jointly announced on Thursday that the gene therapy drug Dalnacogene Ponparvovec Injection has received approval ...

And so we went to conversion therapy," he said. Yang explained that his parents gave him an ultimatum. Either he does the conversion therapy and could attend school with his sister at New York ...

In May 2018, North Carolina awarded the Novartis gene therapy division (then called AveXis, later renamed Novartis Gene Therapies) an initial job development investment grant to build a new ...