Angelman Syndrome (AS) Week: Day 4 Takeaways

研报英文原文证据摘录

Angelman Syndrome (AS) Week: Day 4 Takeaways

Biotechnology

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May 14, 2026

Companies mentioned

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●Our virtual Angelman Syndrome Week continued today with a Research Analysts:

discussion with a KOL, Dr. Cesar Ochoa-Lubnioff. Kristen Kluska

212-915-1927

Kristen.Kluska@cantor.com What's On Deck Tomorrow?

Eric Schmidt

212-294-7724 ●Please see our recent deep dive HERE and join us tomorrow for the Day

Eric.Schmidt@cantor.com 5 of Angelman Syndrome Week and a discussion with Ionis (NC).

Ross Fladeland

212-915-1803 Key Takeaways From Our Discussion with Dr. Cesar Ochoa-Lubinoff.

ross.fladeland@cantor.com

Ayan Hussein, PhD ●Dr. Cesar Ochoa-Lubinoff is the Director of the Angelman Syndrome

212-915-1122 Clinic and the Director of Developmental-Behavioral Pediatrics at Ayan.Hussein@cantor.com

Cedars-Sinai Medical Center.

●Dr. Ochoa-Lubinoff was an investigator for Ultragenyx's Phase 1/2,

Ionis' Phase 1/2a, and Roche's Phase 1 antisense oligonucleotide

(ASO) programs for AS.

●Currently, he is also a primary investigator for Ultragenyx's Phase 3,

Ionis' Phase 3 and will be a primary investigator for Oak Hill Bio's Phase

3.

●Dr. Ochoa-Lubinoff highlighted to us the debilitating nature of the

disease which included developmental issues, lack of communication,

hyperactivity and lack of sleep.

●Interestingly, patients tend to plateau between the ages of four and

five, with developmental progress becoming stagnant.

●Just like other experts we've talked to this week, Dr. Ochoa-Lubinoff

highlighted communication as the area of highest unmet need in AS

and believes this is why therapeutic interventions must be developed.

●Dr.