RJVA-002 achieved ~30% weight loss at five weeks in a translational obesity model; weight loss not yet plateauedPreclinical ...

MIT scientists have found a way to make gene editing far safer and more accurate — a breakthrough that could reshape how we ...

Rocket has pulled an approval application to the FDA, with the biotech considering offloading the asset via external ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its ...

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...

Stunning success in halting Huntington's disease with gene therapy offers new hope to those with the disorder—and potential ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

The library was constructed by generating a variety of AAV capsid variants through the insertion of random peptides at a specific site in the capsid. Specifically, a 7-mer peptide display library ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

How cell and gene therapy developers can lessen the chances of receiving FDA complete response letters through the use of ...