Marisa Wexler MS,  —

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Articles by Marisa Wexler

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…

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…

FDA Grants Orphan Drug Status to M6P Gene Therapy

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…