The first prime editing clinical trial has been cleared by U.S. FDA to begin: prime editing of HSCs to treat chronic granulomatous disease (CGD). Congratulations to Prime Medicine on achieving this key milestone only 4.5 years after we published the first prime editing paper.

This Thursday April 25 from 4:00-6:30 pm at the Broad Institute Julia Vitarello (Mila's mom Mila's Miracle), Casey McPherson (Rose's dad ToCureARose), Termeer Foundation, Cookies4Cures, Broad Institute, and others will host 'Rock n' Rare', a special event of inspirational stories

Published today, our reasons for optimism that success rates in drug discovery can be improved by picking the right targets using human genetics: nature.com/articles/s4158…

I break the key findings down panel by panel in this thread: x.com/cureffi/status…

Excited to share that UK regulators have cleared the clinical trial application for BEAM-302 as a potential treatment for alpha-1 antitrypsin deficiency! We are on track to initiate the trial in the first half of 2024.

globenewswire.com/news-release/2…

An exciting new report in Nature Biotechnology from Xiao Wang’s team led by Hongyu Chen shows that mRNAs with branched poly-A tails yield much higher levels, and prolonged production, of the corresponding proteins compared with conventional single-tailed mRNA. This discovery

A terrific profile of someone who exemplifies leading by example, both personally and professionally. Congratulations, Carolyn Bertozzi for your well-deserved successes.🍻

A new PACE-based system generates protein degradation tags that interact with an otherwise-inert small molecule to trigger the rapid breakdown of a cell’s native proteins, with molecular glue systems that can become useful new research tools. broad.io/Pace-News-0314…