The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.
Using interactions between viral peptides and human proteins as a starting point, researchers from Enyo Pharma Inc., the University of Lyon and other institutions were able to bootstrap themselves to a mitochondria-targeting small molecule that showed activity in a mouse model of nonalcoholic steatohepatitis (NASH) with chronic kidney disease.
Korro Bio Inc. has highlighted new data for KRRO-110, its first development candidate for the potential treatment of α-1 antitrypsin deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
Researchers at Myrobalan Therapeutics Inc. and Myrobalan Therapeutics Nanjing Co. Ltd. have divulged macrophage colony-stimulating factor 1 receptor (CSF-1R; CD115; c-Fms) inhibitors reported to be useful for the treatment of cancer, autoimmune diseases, neurodegeneration, obesity, metabolic diseases, and inflammatory disorders.
The use of α-glucosidase inhibitors reduces postprandial glucose levels and increases insulin sensitivity, and is thus ideal for combination with insulin therapy.
Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Fractyl Health Inc. has nominated RJVA-001 as the first clinical type 2 diabetes candidate from its Rejuva gene therapy platform, which is designed to deliver locally administered genetic medicines to the pancreas.
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