Hypochondroplasia (HCH) is a skeletal dysplasia similar to achondroplasia (ACH) but with milder features, that affects particularly the ossification of proximal long bones of arms and legs. Around 70% to 80% of cases of HCH are caused by N540K alterations in the FGFR3 gene. At the recent 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference, researchers from Tyra Biosciences Inc. presented preclinical proof-of-concept data of TYRA-300, an oral FGFR3-selective inhibitor in a model of HCH.
Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.
Senya Pharmaceuticals Inc. has described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurological disorders.
Shenzhen Salubris Pharmaceuticals Co. Ltd. has divulged myeloperoxidase inhibitors reported to be useful for the treatment of cardiovascular disorders.
Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. has synthesized antibody-drug conjugates consisting of anti-B7-H3 antibodies covalently linked to cytotoxic drugs reported to be useful for the treatment of cancer.
Pharma Foods International Co. Ltd. (PFI) has announced the achievement of a milestone under its licensing agreement with Mitsubishi Tanabe Pharma Corp. for a new investigational therapeutic antibody for autoimmune diseases.
Metabolic dysfunction-associated steatotic liver disease (MASLD) has a prevalence of about 25% in the adult population, with steatosis present in >5% of hepatocytes, hepatocyte ballooning and fibrosis as the main hallmarks. Scientists have tested effects of the galectin-3 inhibitor modified citrus pectin (MCP) in ApoE knockout mice fed a western diet.
Medipal Holdings Corp. and JCR Pharmaceuticals Co. Ltd. have announced the completion of the clinical trial notification review by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for a phase I/II study of JR-446 for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome type B).
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