Excited to share 2 preprints, together showing how mechanisms of a molecular glue & E3 ligase cancer mutations serendipitously converge to cause neomorphic protein degradation in distinct contexts. Highlights below: 1/17
#1: biorxiv.org/content/10.110…
#2: biorxiv.org/content/10.110…

One of the best aspects of #ASGCT2024 : reuniting with former lab members and collaborators.
With Gregory Newby (now a professor at Johns Hopkins), Xiao Wang, Samagya Banskota (now a professor at Boston University), Meirui An, Mandana Arbab (now a professor at Harvard

Coming up soon at #ASGCT2024 : Hear from Drs. Beverly Davidson (Children's Hospital) + Philip Gregory (@Regeneron) during today's George Stamatoyannopoulos Memorial Lecture and from David R. Liu during the Outstanding Achievement Award Presentation! bit.ly/3HFSrdi

Alyssa is the first patient to receive a base edited therapeutic (triply base edited CAR-T cells that cleared her T-cell leukemia), doing well now more than two years post-treatment thanks to the efforts of Waseem Qasim’s team and GOSH.

My #ASGCT2024 talk Thursday May 9 is actually titled “Continuous evolution of genome editing systems for targeted gene-sized DNA integration in mammalian cells”, rather than “Speaker” 😅 and will describe two unpublished projects. ASGCT

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…