Small Molecule Therapy
FDA grants fast-track designation to small molecule for Fragile X
The FDA has granted fast-track designation to an investigational small molecule to treat individuals with Fragile X syndrome, according to the manufacturer.
Annovis secures US patent for buntanetap
Annovis Bio Inc. has secured a U.S. patent for methods of treating acute traumatic brain injuries with its lead drug candidate, an oral, small molecule neurotoxic protein inhibitor.
Mission Therapeutics receives $5.2M to advance novel small-molecule Parkinson’s therapy
Mission Therapeutics has been awarded $5.2 million from The Michael J. Fox Foundation for Parkinson’s Research and Parkinson’s UK to advance a selective, small molecule, brain penetrant USP30 inhibitor for Parkinson’s disease.
Deucravacitinib ‘very likely’ best small molecule for systemic lupus erythematosus
Deucravacitinib is “very likely to be the best” targeted small-molecule drug for the treatment of systemic lupus erythematosus, according to data published in Arthritis Research & Therapy.
Novel small molecule in development for Parkinson’s-related GBA1 mutations
Vanqua Bio has announced its lead candidate for Parkinson’s disease, a small molecule allosteric activator of glucocerebrosidase, will enter clinical development in the first quarter of 2024.
Smaller dose, new regimen recommended for pexidartinib in TGCT after post-marketing data
A reduced dosage and updated regimen are now available for the oral small molecule pexidartinib in the treatment of adults with symptomatic tenosynovial giant cell tumor, according to a press release from the drug manufacturer.
VIDEO: ‘A lot of potential’ for targeting protein degradation
In this video, Stan Lipkowitz, MD, PhD, chief of the women's malignancies branch in the National Cancer Institute's Center for Cancer Research, discusses new research into targeting protein degradation.
FDA accepts avacopan NDA for ANCA-associated vasculitis
The FDA has accepted a new drug application from ChemoCentryx Inc. for its orally administered small-molecule drug avacopan for the treatment of ANCA-associated vasculitis, according to a company press release.
Discovery of novel fetal hemoglobin repressor may provide ‘new avenues’ for sickle cell disease treatment
ORLANDO — Researchers identified the protein nuclear factor IX as a novel repressor of fetal hemoglobin, a discovery that could lead to the development of new treatments for sickle cell disease, according to data presented at ASH Annual Meeting and Exposition.