Cure Rare Disease (CRD) announced that the FDA has approved the first-in-human IND for CRD-TMH-001, its first therapeutic candidate using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy.
Alexion Pharmaceuticals has synthesized new macrocycle compounds acting as complement factor B inhibitors reported to be useful for the treatment of age-related macular degeneration, multiple sclerosis, rheumatoid arthritis, retinal degeneration, chronic obstructive pulmonary disease (COPD), paroxysmal nocturnal hemoglobinuria and cardiovascular disorders.
WuXi Biologics (Shanghai) and WuXi Biologics Ireland have divulged antibody-drug conjugates (ADCs) comprising anti-TROP2 antibodies covalently linked to cytotoxic drugs via linker reported to be useful for the treatment of cancer.
RP Scherer Technologies has synthesized new antibody-drug conjugates (ADCs) comprising camptothecine or camptothecine derivatives linked to a polypeptide such as an antibody through a linker reported to be useful for the treatment of cancer.
Vellore Institute of Technology University (VIT) and BG Negev Technologies and Applications have described new curcumin or piperine analogues acting as NF-kappaB (NFKB) activation inhibitors and/or quorum sensing signaling inhibitors reported to be useful for treatment of cancer, fungal and bacterial infections and inflammatory disorders.