Super impressed by GenerationBio's #ESGCT2023 data! First example I'm aware of of actively targeted LNPs for mRNA delivery leveraging scFvs for cell targeting, instead of dealing with all of the challenges that come with mAbs. See more here: generationbio.com/wp-content/upl…

1. Let's have a quick comparison between Verve-101 and Verve-102. Two differences: 1⃣ The ionizable amino lipid Acuitas (101) ➡️ Novartis (102) 2⃣ Adding GalNAc to the LNP The adenine base editor (ABE) and gRNA stay the same Other LNP components also stay the same

Remarkable to see that our discoveries over 15 years ago would enable such tremendous advances for sickle cell disease and thalassemia!!! Incredible to think that the final sentence of our 2008 paper would be prophetic (science.org/doi/10.1126/sc…)... statnews.com/2023/11/16/u-k…

A truly inspiring 2023 ABRCMS meeting in Phoenix. Love to spread enthusiasm about the physician-scientist career and the The Harvard/MIT MD-PhD Program program with our amazing students representing history of science, neuroscience, engineering, and immunology. Got to see some saguaros too!

Glad to have made a small contribution to this work! Stabilization of nebulized LNPs is an important step towards achieving effective pulmonary mRNA delivery, with implications for a number of diseases

An important perspective on the recent approvals of gene editing therapies for hemoglobinopathies - certainly a milestone, but a long way to go to ensuring access to all in need. In vivo or small molecule approaches are a must! nytimes.com/2023/12/08/hea…

Bioengineered spider silk without critters nature.com/articles/s4158…

Bone marrow transplant advance could widen use of this less buzzy cure for sickle cell disease statnews.com/2023/12/12/sic… via STAT

Excited to have received the support of the NIH to push forward our efforts developing a non-viral, protein-based targeted gene editor delivery - more to come!

statnews.com/2024/01/18/ege…

Happening next Wednesday! If you’re interested in therapeutic gene editing, or the realities of clinical translating a novel modality, tune in! Details below.

Exclusive: After a months-long fight with Texas Medicaid over coverage of a gene therapy, Afghan refugees now have a chance to save their infant son. trib.al/qTXeRLS

Exclusive: Backed by MIT’s Andrew Lo, Quantile Health raises $6M to bolster gene therapy access - endpts.com/exclusive-back…