What can a molecule used for hematopoietic stem cell expansion teach us about pediatric brain tumors? Check out the incredible stories below highlighting the convergence of chemical design and cancer genetics on the same molecular solution👇

Today in Science Magazine, together with my former colleagues at NIBR, we report the discovery and characterization of first molecular glue degraders of the WIZ transcription factor (TF) for fetal hemoglobin derepression and therapeutic consideration in Sickle Cell Disease. 1/10

A major medical milestone late last year was the first #CRISPR genome editing approval (Casgevy) vs sickle cell disease. But it's very complicated and expensive. How about an epigenetic modifier pill with the same target, as seen here Science Magazine? science.org/doi/10.1126/sc…

Gee WIZ: Novartis’ “Glue Degrader” Drug Candidate for Sickle Cell Shows Promise in Animal Studies — My piece for Genetic Engineering & Biotechnology News on exciting new work from Pamela Ting Jay Bradner, M.D. and colleagues #sicklecell genengnews.com/news/gee-wiz-n… via Genetic Engineering & Biotechnology News

Excited to see this published in Science Magazine—our team’s paper on the discovery of molecular glue degraders to induce fetal hemoglobin—a potential avenue toward creating an accessible oral therapy for #sicklecelldisease. Congrats to Pamela Ting, Jay Bradner, M.D. and all involved

WIZ is identified as HbF repressor via a phenotypic screen, leading to the discovery of its molecular glue degraders dWIZ-1 and dWIZ-2 which induce HbF in erythroblasts, offering an promising oral therapy option for sickle cell disease.#NBThighlight science.org/doi/10.1126/sc…

Last week, Novartis scientists published a beautiful paper on a potentially game-changing treatment for sickle cell disease (SCD). The article is paywalled, so I thought I'd summarize it in a brief thread! Check it out. 🧵⤵️ science.org/doi/10.1126/sc…

It’s incredible that we can treat sickle cell disease with CRISPR, but it’d be extremely useful to have a pill that does the same thing. Work from Pamela Ting Jay Bradner, M.D. in Science Magazine show it may be possible with a molecular glue small molecule!