The Foundation for Angelman Syndrome Therapeutics (FAST) is proud to announce the launch of two biotechnology companies, MavriX Bio (pronounced ‘mavericks’) and CourageAS Bio (pronounced ‘courageous’), each dedicated to advancing potentially transformative therapies for Angelman syndrome (AS). These companies plan to develop gene replacement and gene-editing therapies, respectively, under the management of AS²Bio, the therapeutic accelerator launched by FAST.
MavriX Bio will advance the first AAV-delivered, investigational gene replacement therapy for AS, building on cutting-edge research from Dr. Jim Wilson’s laboratory at the University of Pennsylvania, now Gemma Bio. This program, initially funded by FAST, represents a major step toward clinical translation for a disorder with no approved disease-modifying treatments.
CourageAS Bio, the second newly launched company, will focus on advancing an investigational non-viral delivered gene-editing program for AS. Gene editing holds the potential to target and restore endogenous UBE3A gene function, offering an innovative approach to treating the disorder.
“These launches mark another milestone in our mission to accelerate the drug development ecosystem for Angelman syndrome,” said Alana Newhouse, president of FAST. “By establishing MavriX Bio and CourageAS Bio, and managing these programs through AS²Bio, we are ensuring that the most promising scientific breakthroughs rapidly move from research into the clinic with the ultimate goal of reaching those who need them most.”
FAST has long been a leader in funding and driving translational research, supporting both gene therapy, gene-editing, and other disease modifying strategies to tackle Angelman syndrome from multiple angles. The establishment of these companies under the AS²Bio accelerator reflects a strategic vision to fast-track therapeutic development, fostering industry partnerships and regulatory progress to bring potentially transformative treatments to clinical trials as quickly as possible.
“We are witnessing an extraordinary moment in Angelman syndrome drug development,” said Jennifer Panagoulias, COO of MavriX Bio. “With the support of FAST and the foundation laid by Dr. Wilson’s research, MavriX Bio is committed to delivering the first clinical trial of an investigational gene replacement therapy for AS.”
Similarly, CourageAS Bio will drive forward the promise of gene editing as an alternative therapeutic strategy. “We believe that gene editing holds tremendous potential to create precise, durable solutions for Angelman syndrome and other neurogenetic disorders,” Jiangbing Zhou, Chief Technology Advisor of CourageAS Bio, added. “We are very excited to bring this program into clinical development for our lead program in AS.”
Both companies will work closely with scientific leaders, regulatory agencies, and the broader Angelman community to ensure rigorous clinical translation and maximize the potential for patients.
For more information about AS²Bio and its programs, including MavriX Bio and CourageAS Bio, please visit https://www.AS²Bio.com/.
About the Foundation for Angelman Syndrome Therapeutics (FAST)
FAST is the largest non-governmental funder of Angelman syndrome research, dedicated to finding a cure through scientific advancements and therapeutic development. FAST collaborates with researchers, clinicians, and industry leaders to accelerate the path from discovery to treatment.
About AS²Bio
AS²Bio is a therapeutic accelerator announced by FAST in 2024 to support the development of multiple treatment strategies for Angelman syndrome. By fostering and managing biotech companies focused on gene therapy, gene editing, and other innovative approaches, AS²Bio is driving the rapid translation of scientific breakthroughs into clinical treatments.