Seven previously unreported mutations linked to type 1 Gaucher disease were identified in a study analyzing the genetic code of dozens of people with the condition in Egypt. “All patients carrying these novel variants exhibited reduced enzyme levels and clinical symptoms characteristic of type 1 [Gaucher disease], without neurological…
One-time treatment with the gene therapy FLT201 (avigbagene parvec) may help control Gaucher disease without the need for additional therapies, according to new data from four patients in an early clinical trial. Spur Therapeutics, the company developing FLT201, presented the data at the European Society of…
People with Gaucher disease are not at a higher risk of developing cancer, but their risk of blood cancers is higher than in the general population, according to a new analysis. “We demonstrated that patients with GD [Gaucher disease] do not have an increased overall risk of developing cancer.
In Gaucher disease, the toxic buildup of a fatty molecule called glucosylsphingosine (lyso-Gb1) leads to issues with how nerve cells generate energy and other cellular problems, according to a new study. Lyso-Gb1 is a biomarker of disease burden and response to treatment. The findings help to understand how the…
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…
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…
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…
Treatment with the experimental gene therapy FLT201 led to sustained clinical benefits that lasted up to 21 months for people with Gaucher disease type 1, according to new clinical trial data. Spur Therapeutics, the therapy’s developer, presented the findings at the 28th annual meeting of the…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to two gene therapies that M6P Therapeutics is developing — one intended for Gaucher disease, the other aiming to treat the inherited metabolic disorder mucolipidosis. The regulatory agency also awarded six rare pediatric disease designations to…
Cerdelga (eliglustat) safely and effectively treated three adults with Gaucher disease type 1 (GD1), who switched from long-term use of enzyme replacement therapy (ERT) to this substrate reduction therapy (SRT), a study from Korea reports. The study, “One-year experience of oral substrate reduction therapy in three…
