The Foundation for Angelman Syndrome Therapeutics (FAST) is proud to announce the launch of two pioneering biotechnology companies, MavriX Bio and CourageAS Bio, each dedicated to advancing potentially transformative therapies for Angelman syndrome (AS). These companies will develop gene replacement and gene-editing therapies 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 towards clinical translation for a disorder with no approved disease-modifying treatments.
CourageAS Bio will focus on advancing an investigational non-viral delivered gene-editing program for AS. Gene editing has the potential to restore endogenous UBE3A gene function, offering an innovative approach to treatment.
“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 been a leader in driving translational research, supporting gene therapy, gene-editing, and other disease-modifying strategies. The establishment of these companies under AS²Bio reflects a strategic vision to fast-track therapeutic development, foster industry partnerships, and accelerate progress towards clinical trials.
“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 is focused on the potential of gene editing.
“Gene editing holds tremendous potential to create precise, durable solutions for Angelman syndrome and other neurogenetic disorders,” said Jiangbing Zhou, Chief Technology Advisor of CourageAS Bio. “We are excited to bring this program into clinical development for our lead program in AS.”
Both companies will collaborate with scientific leaders, regulatory agencies, and the broader Angelman community to ensure rigorous clinical translation, bringing us closer to life-changing treatments.
🔗 For more information about AS²Bio and its programs, visit: AS²Bio Website
