Angelman Syndrome (AS) Week: Day 2 Takeaways

研报英文原文证据摘录

Angelman Syndrome (AS) Week: Day 2 Takeaways

Biotechnology

EQUITY RESEARCH Industry Report

May 12, 2026

Companies mentioned

Ticker Price Rating Target Who Did We Host Today? RARE $26.84 Overweight $84.00

●We continued our Virtual Angelman Syndrome Week today, which Research Analysts:

included discussions with the Foundation for Angelman Syndrome Kristen Kluska

212-915-1927 Therapeutics (FAST) and Oak Hill Bio (private).

Kristen.Kluska@cantor.com

●Reach out if you'd like any replays! Eric Schmidt

212-294-7724

Eric.Schmidt@cantor.com What's On Deck Tomorrow?

Ross Fladeland

212-915-1803 ●Please see our recent deep dive HERE and join us tomorrow, 5/13, for

ross.fladeland@cantor.com Day 3 with discussions with the Angelman Syndrome Foundation (ASF),

Ayan Hussein, PhD and KOL, Dr. Robert Carson.

212-915-1122

Ayan.Hussein@cantor.com

Key Takeaways From Our Discussion with FAST:

●FAST is a patient advocacy organization working to cure Angelman

syndrome through funding AS research.

●Notably, FAST established AS²Bio, an AS therapeutic accelerator

designed to create a collaborative drug development ecosystem that

enables companies to leverage insights from other AS programs and

streamline therapeutic development.

●Through a science lens, FAST looks at all modalities for AS, including

ASOs, CRISPR, gene therapy, and hematopoietic stem cells.

●Our discussion also focused on misconceptions about AS and

highlighted how debilitating the disease is for patients and families,

making even small clinical improvements meaningful and valuable.

●Interestingly, this also makes selecting a singular clinical endpoint

difficult because improvements in any domain, even if not statistically

significant, can greatly improve a patient's quality of life.