Here, the authors describe a scalable, CRISPR activation screening framework to identify regulatory element-gene pairs in diverse cell types including cancer cells and neurons. Continuous directed ...

The startup’s technology combines an AAV — a common gene therapy delivery tool — with an immunotherapy approach now widely used in oncology. The idea is to give patients a one-time treatment that ...

The product candidate is a modifier gene therapy for broad retinitis pigmentosa indication.

Gene delivery is a process by which foreign DNA is transferred to host cells for applications such as genetic research or gene therapy. Gene delivery methods can be mechanical (e.g. microinjection ...

Headlines,CRISPR Therapeutics,(NASDAQ:CRSP),has achieved a significant regulatory milestone with its gene-editing therapy, Casgevy, now approved in multiple countries, but has faced challenges in ...

A new start­up at­tempt­ing to de­vel­op AAV gene ther­a­pies to treat can­cer emerged from stealth Thurs­day morn­ing with $26 mil­lion in seed fund­ing — and a … ...

Vironexis Biotherapeutics has unveiled with $26 million and a clinical-trial-ready gene therapy that has already nabbed FDA fast track and rare pediatric disease tags. The San Diego-based biotech ...

"The era of gene therapy for neuromuscular diseases has begun, and the complexity of these diseases requires us to constantly innovate to improve drug candidates targeting muscle. The new ...

Soo-Kyung Lee hoped to change that through gene therapy. However, some had doubts that because of FOXG1’s role in brain development in utero, administering a therapy after birth may not have a ...

SINGAPORE – Patients here who require certain costly cell, tissue and gene therapy products (CTGTPs) will now be eligible for subsidies, starting with one product for blood cancers. This opens ...

It's gene therapy research at Children's Hospital of Philadelphia that's being called a game changer. The first patient to get the therapy outside a research setting is a Cherry Hill college ...

BridgeBio Pharma is slashing its gene therapy budget and pulling back from the modality after seeing the results of a phase 1/2 clinical trial. CEO Neil Kumar, Ph.D., said the data “are not yet ...