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.

Aridis Pharmaceuticals announced preclinical data for its inhaled monoclonal antibody (MAb) cocktail, AR-701.

Polycystic ovary syndrome (PCOS) appears to result from increase of GnRH activity and LH release, as well as elevated androgen production.

Patrys announced new preclinical data for its lead asset, PAT-DX1, being evaluated in animal model of high-grade glioma.

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.

Schroedinger has patented fused heterocycles acting as Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer.

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.

GentiBio announced that it has entered into a collaboration with Bristol Myers Squibb to develop new engineered regulatory T cell (Treg) therapies to re-establish immune tolerance and repair tissue in patients living with inflammatory bowel diseases (IBD).