News

Sharp gearing up to move Gaucher disease treatment ‘901 to trial

Sharp Therapeutics is gearing up to ask the U.S. Food and Drug Administration (FDA) for permission to begin testing its Gaucher disease treatment candidate ‘901 in clinical trials. The developer, a preclinical-stage biotechnology company, announced that it’s engaged the contract research organization Rho to help it put together its…

mRNA treatment shows durable effects in Gaucher disease mice

An experimental treatment that uses messenger RNA (mRNA) to help the body produce the missing enzyme in Gaucher disease reduced a biomarker of disease burden in a mouse model, with longer-lasting effects and fewer side effects than current options. JCXH-301, which was packaged inside tiny spherical particles, was well tolerated…

Abcertin meets Cerezyme biosimilar criteria in Phase 1 study

Abcertin, a newly developed form of imiglucerase, was found to be equivalent to the approved enzyme replacement therapy (ERT) Cerezyme in a Phase 1 study involving healthy adults. The two medications, which contain the same active ingredient, were found to have similar pharmacological properties and safety profiles, suggesting Abcertin…

6 new GBA1 mutations tied to Gaucher type 1: Case series

Researchers identified six previously unknown mutations in the GBA1 gene linked to Gaucher disease, according to a case series report. All clinical signs and symptoms associated with these new mutations were consistent with a diagnosis of Gaucher disease type 1, the most common type of the condition, characterized by…

Casma selects treatment CSM-101 for Gaucher-related Parkinson’s

Casma Therapeutics has selected its first experimental therapy candidate, dubbed CSM-101, which will be developed to treat Gaucher’s disease patients who have Parkinson’s disease. The company is planning to submit an investigational new drug application to the U.S. Food and Drug Administration (FDA) in 2026 asking for permission…

Gaucher disease gene therapy FLT201 shows proof of concept

FLT201, a gene therapy for Gaucher disease type 1 currently in clinical testing, led to sustained increases in glucocerebrosidase (GCase) enzyme activity and reductions in fat molecules in animal models, indicating that it works as designed. The gene therapy provides instructions for the production of an optimized version…