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

AOC 1044 receives FDA orphan drug designation for DMD

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer…

Phase 1/2 trial of AB-1003 gene therapy doses 1st LGMD patient

The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003 in a clinical trial, according to an announcement from the therapy’s developer Asklepios BioPharmaceutical (AskBio). “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is…

New cell models available to help advance DMD research

Synthetic biology company bit.bio has launched two new human muscle cell models to advance research into new treatment strategies for Duchenne muscular dystrophy (DMD). The two models, dubbed ioSkeletal Myocytes DMD Exon 44 Deletion, and ioSkeletal Myocytes DMD Exon 52 Deletion, are the eighth and ninth products that…

Catalyst acquires North American rights to DMD therapy vamorolone

Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review in the U.S. and Europe for Duchenne muscular dystrophy (DMD). Under the deal, Catalyst will give vamorolone’s developer Santhera Pharmaceuticals an upfront cash payment of $75 million,…

FDA panel favors accelerated approval of SRP-9001 for DMD

An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). While the FDA is not obligated to abide by the committee’s vote, the agency will consider…