Arkuda Therapeutics Inc. has patented new fused tricyclic mucolipin-1 (MCOLN1; TRPML1) blockers reported to be useful for the treatment of cancer, liver diseases, amyotrophic lateral sclerosis, cardiovascular disorders, frontotemporal dementia, Alzheimer’s, Huntington’s and Parkinson’s disease.
Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines.
Researchers from Arkuda Therapeutics reported the discovery and preclinical evaluation of novel oxazoline enhancers of cellular progranulin (PGRN) secretion as candidates for cognition disorders therapy. Phenotypic screening of a diverse library of compounds was applied with the aim of identifying structures that effectively enhanced PGRN release. The lead hits were optimized and assessed in an immortalized murine microglial cell line (BV-2).
Arkuda Therapeutics Inc., a company targeting lysosomal dysfunction to address neurodegeneration, has completed a $64 million series B financing co-led by Cormorant Asset Management and Pivotal Bioventure Partners. The funds will help advance its lead program, a small-molecule progranulin enhancer for frontotemporal dementia in people with an autosomal dominant mutation in the GRN gene, which codes for the protein.
Arkuda Therapeutics, a new company targeting progranulin and lysosomal biology to treat neurodegenerative diseases, has completed a $44 million series A financing led by Atlas Venture, its co-creator, and Pfizer Ventures. Funds managed by Tekla Capital Management LLC and Bioinnovation Capital also participated.
