MavriX Bio’s AAV Gene Therapy for Angelman Syndrome Receives FDA Fast Track Designation

Sep 28 , 2025
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MIDDLETON, Mass.—September 28, 2025—MavriX Bio announced that its investigational adeno-associated virus (AAV) vector-based gene therapy, MVX-220, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome (AS).

The designation accelerates the development path for the one-time therapy, which is intended to deliver a functional copy of the disease-targeted gene, UBE3A, to neurons.

The initial development of MVX-220 was supported by the Foundation for Angelman Syndrome Therapeutics (FAST) and originated at the University of Pennsylvania. MavriX Bio, a portfolio company of the FAST drug development accelerator AS2Bio, is now leading the clinical program in conjunction with its partner, GEMMA Biotherapeutics (GEMMABio).

Accelerated Path to the Clinic

MavriX Bio received FDA clearance for its investigational new drug (IND) application for MVX-220 in May of this year, paving the way for the planned Phase 1/2 clinical trial, ASCEND-AS (NCT07181837).

  • The ASCEND-AS study is expected to launch in October 2025.
  • It will enroll approximately 12 patients with AS, aged 4 to 50 years, and will include individuals with various underlying genetic causes, such as deletion and uniparental disomy.

“The FDA’s decision… reflects the urgent need for therapies for individuals living with AS,” said Allyson Berent, DVM, DACVIM, Chief Development Officer of MavriX Bio. “This designation highlights the promise MVX-220 holds… and importantly, enables closer collaboration with the FDA and an accelerated path forward.”

James M. Wilson, MD, PhD, CEO at GEMMABio, added that the Fast Track status “means the team can engage more closely with regulators and accelerate development milestones. For the AS community, Fast Track Designation opens up a path toward bringing gene-targeted therapies to patients sooner.”

Competitive Landscape in AS

MVX-220 is one of several advanced therapeutics currently in development for Angelman syndrome. Ultragenyx is also developing an antisense oligonucleotide product, GTX-102 (apazunersen), which is currently being evaluated in a Phase 3 clinical trial (NCT06415344). Interim data from earlier studies of GTX-102 have shown evidence of a therapeutic effect and reported improvements in quality of life for families.

Source:

https://www.cgtlive.com/view/mavrix-bio-angelman-syndrome-gene-therapy-mvx-220-garners-fda-fast-track-designation

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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