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 ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

Korro Bio's RNA editing platform, OPERA, targets genetic diseases by performing precise, transient single-base edits on RNA, reducing the risks associated with DNA editing. Lead asset KRRO-110 aims to ...

The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA ...

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 ...

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 ...

DNA is the backbone of the genetic code so, logically, decoding and understanding the genetic sequence along with epigenetic modifications came first. Yet, RNA and the emerging field of ...

Advancements in genome editing technologies the growing emphasis on safe and accurate gene correction, and strong biotechnology R&D investment drive the base editing market.Austin, Texas, Jan. 21, ...

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 ...