CRISPR gene therapy trial for sickle cell disease begins, offering hope for a potential cure by targeting the genetic root of ...

A drug developed by Sanofi raised platelet counts and reduced bleeding episodes in patients with a rare autoimmune disorder ...

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell ...

New genetic medicines for sickle cell disease were hailed as a milestone. Now researchers are working on ways to widen access ...

Sites in Los Angeles and near San Francisco California are enrolling people with severe SCD for a trial to test a gene ...

Genome editing with various CRISPR-Cas molecule complexes has progressed rapidly in recent years. Hundreds of labs around the ...

The CRISPR-SCD trial is the first clinical attempt to use a non-viral delivery approach to directly correct the sickle cell ...

相关研究结果近期发表在Cell期刊上，论文标题为“The CRISPR-associated adenosine deaminase Cad1 converts ATP to ITP to provide antiviral immunity”。 在这篇论文中 ...

Cathie Wood says a "cure" can generate up to 20x more value than traditional drugs, which appears to explain why two of her firm's funds snapped up shares of the Casgevy co-developer until this past ...

Vertex Pharmaceuticals Inc. and bluebird bio Inc. — are the first to take part in a new Medicaid model intended to expand ...

The biotech industry has lagged the broader market so far in 2024. The SPDR S&P Biotech ETF, an industry benchmark, is up 9% ...

Preliminary data demonstrate that CTX112™ has the potential to provide meaningful clinical benefit with a well-tolerated safety profile using a ...