Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...

ProQR Therapeutics is advancing its Axiomer ADAR-mediated RNA editing pipeline, aiming to file a Clinical Trial Application for its lead program AX-0810, targeting NTCP for cholestatic diseases, by Q2 ...

Gene-editing technologies show great promise for medical treatments and research, with the potential to cure thousands of ...

Genetic editing holds promise to treat incurable diseases, but the most popular method - CRISPR - sometimes does more harm than good. A new study from University of California San Diego and Yale ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

Hello, and welcome to Wave Life Sciences 2025 Research Day. [Operator Instructions] Also, as a reminder, this conference is being recorded today. I will now turn the call over to Kate Rausch, Vice ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...

ProQR Therapeutics announces a Phase 1 trial of AX-0810 to evaluate safety and target engagement in cholestatic liver disease. The initiation of the Phase 1 clinical trial for AX-0810 is contingent ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...