Solid Biosciences Inc. has received FDA clearance of its IND application for SGT-003, the company’s next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD). The planned first-in-human phase I/II trial will enroll pediatric patients with DMD to receive SGT-003 as a one-time intravenous infusion.
ΔF508-CFTR is the most common CFTR mutation in cystic fibrosis (CF), which leads to destabilization of CFTR’s first nucleotide-binding domain (NBD1), contributing centrally to defective ΔF508-CFTR folding, trafficking and function.
Researchers from Nkgen Biotech Inc. have developed an anti-HER2 CAR NK cell therapy, named SNK-02, as a potential immunotherapeutic for HER2-overexpressing tumors. SNK-02 was tested in a xenografted murine model of cancer.
Researchers from Adicet Bio Inc. reported on the preclinical development of ADI-925, an engineered Chimeric Adapter (CAd) γδ1 T-cell therapy that targets major histocompatibility complex class I chain-related protein A/B (MICA/MICB) and ULBP1-6 expressed in tumor cells.
A new derivative of coumarin, a natural plant product abundant in cinnamon, could hold the key to healthy aging. Researchers at the Buck Institute have shown that it extended life span and prevented neurodegenerative disease in worms and mice. The drug, a TFEB gene inducer called MIC, promoted mitochondria recycling (mitophagy) but also interacted with lysosomes, which could have multiple applications. The scientists published the results of this aging and mitophagy study on Nov. 13, 2023, in Nature Aging.
Vectory Therapeutics BV has closed a €129 million ($138 million) series A financing to advance its vectorized antibody programs in neurodegenerative diseases.
Mindset Pharma Inc. has described indoline derivatives acting as 5-HT2A receptor agonists reported to be useful for the treatment of anhedonia, anorexia nervosa, epilepsy, fragile X syndrome, obsessive-compulsive disorder, post-traumatic stress, stroke and Alzheimer’s disease, among others.
Rome Therapeutics Inc. has divulged didehydro-3’-deoxy-4’-ethynylthymidines acting as LINE-1 retrotransposable element ORF1 protein (L1RE1) and/or HERV-K reverse transcriptase inhibitors reported to be useful for the treatment of cancer, autoimmune disease and neurological disorders.
Nimbus Clotho Inc. has identified CTP synthase 1 (CTPS1) inhibitors reported to be useful for the treatment of psoriasis, lichen planus, lupus nephritis, systemic lupus erythematosus, multiple sclerosis, myasthenia gravis, autoimmune lymphoproliferative syndrome, and graft-vs.-host disease.