On February 14 and 15, 2025, FAST Italy made history by organizing the very first Scientific Symposium on Angelman syndrome in Monza, Italy. With over 100 attendees, including scientists, clinicians, and families, the conference served as a crucial platform for education, collaboration, and hope.
The symposium coincided with International Angelman Day (February 15), reinforcing the global commitment to accelerating research and treatments for Angelman syndrome. Attendees had the opportunity to hear from leading experts from both Italy and the United States, sharing the latest advancements in the field.
Major Scientific Highlights
Dr. Allyson Berent presented FAST’s Roadmap to a Cure, outlining the multi-pronged strategy to bring effective treatments to individuals with Angelman Syndrome.
Jennifer Panagoulias provided an exciting update on the gene therapy programs supported by AS2Bio, the FAST-initiated accelerator driving promising therapeutic approaches forward.
A major highlight of the conference was the announcement of 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.
Read the full announcement here.
A Milestone for the Italian AS Community
The success of FAST Italy’s inaugural symposium is a testament to the power of knowledge-sharing and international collaboration. By providing families and professionals with direct access to the latest research and therapeutic advancements, FAST Italy has taken a significant step toward empowering the Italian Angelman community with education and hope.
We extend a special thanks to Benedetta Sirtori for her dedication and hard work in organizing this impactful event. Her efforts have helped create a space for learning, connection, and progress.
Congratulations to FAST Italy on this incredible achievement! We look forward to many more conferences that will continue to push the boundaries of science and bring us closer to a cure.
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