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 ...

Researchers using next-generation DNA sequencing have identified four specific genes whose mutations are linked to the ...

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: ...

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 ...

One assay applies to bioprocesses that use the HEK293 human embryonic kidney cell line, often used in gene therapy development.

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