One class of small molecules of recent prevalence, which practitioners tend to claim in this manner, are heterobifunctional compounds.

The switch from fetal γ-globin to adult β-globin is controlled by BCL11A. Genetic manipulation of BCL11A improves disease outcomes by turning γ-globin expression back on to compensate for ...

About Fulcrum Therapeutics Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare ...

April 22, 2025 — At Argonne National Laboratory near Chicago researchers are using AI and high performance computing to develop new methods aimed at accelerating drug discovery and enhancing drug ...

Critical to the development of the fetal hemoglobin switching approach were genome-wide association studies performed 15 years earlier by Stuart Orkin, MD, and colleagues that identified BCL11A as ...

Dearden felt divinely guided to study small molecules in spectrometry, he said. "I collected my poster, got in the car to go home and I had a revelation, I really did," Dearden said.

Drug development powered by artificial intelligence is countering incentives from the Inflation Reduction Act and making small molecules more attractive in the complex inflammatory & immunology ...

Among small-molecule drugs, upadacitinib and deucravacitinib demonstrate the best efficacy for treatment of psoriatic arthritis, according to data published in Therapeutic Advances in ...

Molecular glue -based degraders, small molecules that shunt proteins of interest to the cell’s garbage disposal system, have been making waves in oncology.

The process of identifying promising small molecule drug candidates that target cancer checkpoints may become faster and smarter through virtual screening, according to Weill Cornell Medicine ...

New small molecule could treat sickle cell disease in adults that don't respond to hydroxyurea, alone Date: July 31, 2024 Source: Boston Medical Center Summary: Researchers found a small molecule ...

A surprise set of small molecule candidates In the Esser-Kahn lab, Knight set out to find out whether any small molecules—drugs that can easily enter cells—could also induce trained immunity.