Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. IND clearance enables initiation of a phase I/II trial, with a clinical trial application (CTA) submission to the EMA also planned in the coming months.
Clarity Pharmaceuticals Ltd. has expanded its pipeline with a new fibroblast activation protein (FAP)-targeted radiopharmaceutical for the diagnosis and treatment of cancer. Clarity’s targeted copper theranostic targeting FAP was developed by combining an industry-leading FAP inhibitor with proprietary SAR chelator technology, which enables the use of copper-64 (64Cu) for imaging and copper-67 (67Cu) for the targeted treatment of various cancers.
Bristol Myers Squibb Co. has selected the first novel target for amyotrophic lateral sclerosis (ALS) that was identified and validated by Insitro Inc. under the companies’ 2020 collaboration agreement to discover new therapies for ALS. The selection triggers a milestone payment to Insitro.
Genescience Pharmaceuticals Co. Ltd. has identified fibroblast growth factor receptor 2 (FGFR2) and/or FGFR3 inhibitors reported to be useful for the treatment of cancer, osteochondrodysplasia and achondroplasia.
Scientists at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged compounds acting as Toll-like receptor 7 (TLR7) and TLR8 agonists reported to be useful for the treatment of cancer.
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has synthesized sodium-bile acid cotransporter (SLC10A1; NTCP) inhibitors reported to be useful for the treatment of cardiovascular, fatty acid oxidation, liver and gastrointestinal disorders.
RSS
