The FDA in 2022 approved Amvuttra to treat adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis, a rare debilitating disease that damages nerves, and analysts have predicted ...

Scholar Rock yesterday reported that its experimental treatment for spinal muscular atrophy succeeded in a Phase 3 trial, ...

A new RNAi biotech is breaking ground with $135 million and John Maraganore, Ph.D., holding the key to the city. | A new RNAi ...

City Therapeutics, based in Cambridge, is developing new medicines that use RNA interference, or RNAi, to silence genes and ...

The time to re-enter the RNAi revolution is now,” said Robert Nelsen, the managing director of Arch, which led the Series A funding round for City Therapeutics.

In a report released today, Michael Ulz from Morgan Stanley maintained a Hold rating on Arrowhead Pharmaceuticals (ARWR – Research ...

Intellia Therapeutics, Inc. NTLA announced that it has initiated the pivotal phase III HAELO study evaluating NTLA-2002 for ...

Ultragenyx Pharmaceutical RARE announced that the FDA has granted the Breakthrough Therapy designation to its investigational candidate, setrusumab (UX143), as a treatment to reduce the risk of ...

As the U.S. stock market faces turbulence with major indexes tumbling due to rising oil prices and Treasury yields, investors are keenly observing potential opportunities amidst the volatility. In ...

Dianthus Therapeutics' DNTH103 is in phase 2 trials for gMG, CIDP, and MMN, offering a differentiated, convenient dosing ...

Harnessing next-generation siRNA engineering to improve and expand the therapeutic reach of RNAi-based medicines ...