Casma Therapeutics Inc. has nominated its first development candidate, CSM-101, a first-in-class TRPML1 agonist. CSM-101 is being developed for the treatment of Gaucher’s disease patients with Parkinson’s disease, with the potential to expand into GBA-associated Parkinson’s disease and broader Parkinson’s disease populations.
People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.
Yuhan Corp., of Seoul, South Korea, has inked a ₩150 billion (US$108.6 million) deal with Korean biotech Ubix Therapeutics Inc. to gain exclusive global rights to UBX-103, Ubix’s oral small-molecule androgen receptor degrader for prostate cancer. Yuhan also announced July 1 that it gained the U.S. FDA’s nod to start a phase I study of a Gaucher disease drug candidate called YH-35995.
Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Researchers from the Yale University School of Medicine have developed a novel murine model of Gaucher disease type I with the aim to investigate the impact of GBA1 deficiency on hematopoiesis and the immune system, in order to elucidate potential therapeutic targets.
Freeline Therapeutics plc is being taken private by its founding investor and majority shareholder Syncona Ltd, in an all-cash transaction that values the Nasdaq-listed gene therapy specialist at $28.3 million. Syncona also will provide up to $15 million cash to keep Freeline afloat as it awaits the next tranche of data on its lead program FLT-201, which is positioned as a one-off treatment for the lysosomal storage disorder, Gaucher’s disease.
Parkinson’s disease (PD) is one of the most common neurodegenerative diseases, and the role of ?-synuclein accumulation and the subsequent death of dopaminergic neurons in the midbrain have long been recognized as key steps in the disease. Progress in understanding genetic risk factors, meanwhile, has uncovered multiple genetic risk factors. Even though aging is the single biggest risk factor for PD, there are versions of the disorder that affect children.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.