Marisa Wexler, MS,  senior science writer—

Marisa holds a Master of Science in cellular and molecular pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. Her areas of expertise include cancer biology, immunology, and genetics, and she has worked as a science writing and communications intern for the Genetics Society of America.

Articles by Marisa Wexler

Viral vector in LGMD2E gene therapy gets new FDA designation

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E (LGMD2E). “This is one of the first programs to receive platform technology designation and an important recognition by FDA of…

Pepgen pulls plug on exon-skipping therapy for Duchenne MD

Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type…

CureDuchenne invests in new redosable DMD gene therapy

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing gene therapies. “This investment underscores our continued use of venture philanthropy to catalyze progress [toward] transformative treatments…

DMD gene therapy Elevidys wins conditional approval in Japan

Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval. The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The…

FDA set to decide on deramiocel for DMD cardiomyopathy

The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for…

FDA OKs trial of SRP-9005 gene therapy for LGMD type 2C

The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies…