Clinical trial of groundbreaking Angelman Syndrome gene treatment to commence in 2021
The U.S. Food and Drug Administration (FDA) has given the green light for Mavrix Bio to begin clinical trials for MVX-220, an experimental gene therapy designed to treat Angelman syndrome. This milestone marks a significant step forward in the quest for a potential single treatment therapy for the Angelman community.
Mavrix Bio, a biotechnology company launched by the Foundation for Angelman Syndrome Therapeutics, will be leading the clinical development of MVX-220 in collaboration with Gemmabio, a company specialising in rare disease therapies. MVX-220 is a gene replacement therapy that delivers a healthy copy of the UBE3A gene, which is mutated in individuals with Angelman syndrome, causing the abnormal behaviour and motor development symptoms associated with the condition.
The upcoming clinical trial will evaluate the safety, tolerability, and efficacy of MVX-220 in children and adults with Angelman syndrome who have various genetic anomalies. The study design and expected timeline will be announced in the coming months. The trial will be conducted for individuals living with Angelman syndrome.
Jim Wilson, MD, PhD, President and CEO of Gemmabio, expressed his excitement about supporting MavriX Bio in advancing MVX-220 into clinical evaluation. He noted that this collaboration offers renewed hope for families affected by Angelman syndrome. Allyson Berent, Mavrix Bio's chief development officer, echoed this sentiment, stating that the approval brings them one step closer to delivering the first gene therapy for Angelman syndrome.
MVX-220 uses an adeno-associated virus (AAV) vector for delivery, a harmless, engineered virus commonly used in gene therapy. This gene therapy was developed at the University of Pennsylvania with funding from the Foundation for Angelman Syndrome Therapeutics.
Mavrix Bio's investigational new drug application (IND) was approved by the FDA, paving the way for the first-in-human trial for MVX-220. The company is committed to advancing novel therapeutic options for those living with Angelman syndrome.
With the FDA's approval, the clinical trial is set to commence later this year. The trial will be a crucial step in determining the potential of MVX-220 as a transformative treatment for Angelman syndrome. Stay tuned for more updates on this promising development.
