Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4. Read More
Xtalpi Inc. has announced regulatory clearances in Australia and Taiwan for phase I trials of PEP-08, a next-generation PRMT5 inhibitor candidate developed under its AI drug discovery collaboration with Pharmaengine Inc., for solid tumors. This milestone triggered a payment to Xtalpi under the partnership agreement.
Secreted gonadotropin-releasing hormone (GnRH) acts on the anterior pituitary gland to initiate puberty. Mutations in the protein immunoglobulin superfamily member 10 (IGSF10) have been linked to puberty delay, but the reason has been unclear. Read More
Hearing loss is a major global health challenge, affecting over 5% of the population and largely lacking effective biological therapies. Mutations in KCNQ4, which encodes the Kv7.4 potassium channel essential for outer hair cell function, are a leading cause of autosomal dominant, non-syndromic hearing loss (DFNA2). Read More
HCW Biologics Inc. has developed second-generation, tissue factor-targeting T-cell engagers (TCEs) to treat solid tumors, particularly pancreatic cancer and glioblastoma, constructed with its novel proprietary TRBC product discovery and development platform technology. Read More
Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease. Read More
Mutations that activate phosphorylation of certain Rab GTPases by LRRK2 have been linked to a subset of genetic cases of Parkinson’s disease, and PPM1M is one of the phosphatases responsible for reversing phosphorylation by LRRK2. Read More
Researchers from Cyclone Therapeutics Inc. and the Scripps Research Institute have disclosed tetracycline derivatives reported to be useful for the treatment of fragile X syndrome, rheumatoid arthritis and Alzheimer’s disease. Read More
Aligos Therapeutics Inc. has patented programmed cell death 1 (PDCD1; PD-1; CD279) and/or PD-1 ligand 1 (PD-L1; CD274) and/or PD-1/PD-L1 interaction inhibitors reported to be useful for the treatment of liver cancer (hepatocellular carcinoma) and hepatitis B. Read More
Researchers from the University of California, Davis have designed a novel gene therapy vector that selectively targets and kills cells infected with Kaposi’s sarcoma-associated herpesvirus (KSHV). Read More
The Ohio State University has synthesized DNA topoisomerase II (bacterial) inhibitors reported to be useful for the treatment of bacterial infections. Read More
Chong Kun Dang Pharmaceutical Corp. has prepared and tested glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of fibrosis, neurodegeneration, nutrition, endocrine, immunological, inflammatory, psychiatric and renal disorders, among others. Read More
Researchers from the Institute of Medicinal Biotechnology in China reported the preclinical characterization of a series of benzene sulfonamide derivatives developed as androgen receptor (AR) antagonists. Read More
