Gaucher's disease is a rare hereditary illness that impairs the body's ability to break down a type of fat molecule known as glucocerebroside. This disorder is caused by mutations in the GBA gene ...

Genetic testing can confirm the diagnosis by identifying mutations in the GBA gene,” explained Dr Ankush Golhar. While Gaucher disease does not have any cure yet, it can be treated.

There are no guarantees. Still, he likes his chances. Schlossmacher's lab has found that 10 to 12 per cent of people with Parkinson's have a defect in a gene called GBA.

The clearance allows CAP-003, an intravenously administered gene therapy, to proceed to clinical trials for treating PD associated with glucocerebrosidase (GBA) mutations (PD-GBA).

Capsida is developing a next-generation gene therapy with best-in-class potential, CAP-003, for the treatment of Parkinson's disease (PD) associated with GBA mutations (PD-GBA).

Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of PD patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase ...

For more information about the Phase 1/2 clinical trial, please visit www.clinicaltrials.gov (NCT07011771). About Parkinson’s disease associated with GBA mutations (PD-GBA) ...

THOUSAND OAKS, Calif., June 11, 2025--Capsida Biotherapeutics ("Capsida") today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP ...