---
title: "After Ultragenyx Angelman drug failure, experts say other antisense therapies still hold promise"
id: "stat-news-1-stat-after-failure-of-angelman-syndrome-drug-experts-see-hope-in-other"
canonical_url: "https://medichelpline.com/clinical-feed/stat-news-1-stat-after-failure-of-angelman-syndrome-drug-experts-see-hope-in-other"
content_type: "clinical_feed_article"
specialty: "Neurology"
source_name: "STAT News"
source_url: "https://www.statnews.com/2026/09/04/angelman-syndrome-drug-ultragenyx-oak-hill-ionis/?utm_campaign=rss"
published_at: "2026-09-04T15:34:42.000Z"
evidence_level: "Verified Feed"
license: "CC-BY-NC-4.0 / Informational Use"
---
# After Ultragenyx Angelman drug failure, experts say other antisense therapies still hold promise
## Provenance & Clinical Metadata
- **Canonical URL:** https://medichelpline.com/clinical-feed/stat-news-1-stat-after-failure-of-angelman-syndrome-drug-experts-see-hope-in-other
- **Specialty:** [Neurology](https://medichelpline.com/clinical-feed/neurology.md)
- **Primary Source:** STAT News
- **Source URL:** [Original Journal Publication](https://www.statnews.com/2026/09/04/angelman-syndrome-drug-ultragenyx-oak-hill-ionis/?utm_campaign=rss)
- **Published At:** 2026-09-04T15:34:42.000Z
- **Evidence Rating:** Verified Feed
## Executive GIST (TL;DR)
- Ultragenyx announced that its experimental drug for **Angelman syndrome** failed in a late-stage clinical trial, a setback for patients and the company. - The failed trial was described in STAT News coverage; specific trial results, endpoints, and numerical data were not reported in the source article. - Experts quoted in the article emphasized that a single unsuccessful trial does not necessarily invalidate the therapeutic approach under study. - Mark Zylka, an Angelman researcher at the University of North Carolina, is quoted saying the failed trial does not mean the mechanism is inherently flawed. - The article notes that other companies — including **Ionis** and **Oak Hill Bio** — are developing their own **antisense** treatments for Angelman syndrome and that those programs remain ongoing. - Observers in the piece suggested continued reason for cautious optimism that similar experimental medicines could succeed in restoring functions such as cognition and communication in patients with intellectual disabilities. - The STAT article is a STAT+ exclusive; the published item referenced another STAT story reporting Ultragenyx’s failed trial but withheld many trial-specific details behind the subscription. - The source emphasized broader implications: success or failure in Angelman trials could influence development of genetic medicines for other neurological conditions, but the article did not provide definitive conclusions or new data. - Multiple contextual items in the source (subscription prompts, author contact information, and site navigation) are present but do not add clinical detail about the trials. - Several important details — including the specific therapeutic modality used by Ultragenyx, trial design, patient numbers, safety findings, and timelines for rival programs — were not reported in the source article and therefore are not available here.
## Clinical Analysis & Structured Key Points
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[](https://x.com/JasonMMast "X") jason.mast@statnews.com Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. The [failure of an experimental drug for Angelman syndrome](https://www.statnews.com/2026/09/02/ultragenyx-drug-angelman-syndrome-clinical-trial/), a rare genetic disease that causes severe developmental delays, was a major blow to patients and the drug’s maker, Ultragenyx, which announced the outcome of its late-stage trial earlier this week. But experts cautioned that there is still reason to think similar experimental medicines might yet succeed. That could potentially pave the way for genetic medicines in other neurological conditions that could restore cognition, communication, and other skills in patients with intellectual disabilities. Advertisement “I don’t think this really says anything about the other trials that are ongoing,” said Mark Zylka, an Angelman researcher at the University of North Carolina. “I wouldn’t say that just because this one trial fails, that means [this is a bad mechanism].” STAT+ Exclusive Story Already have an account? [Log in](https://www.statnews.com/login/) ![STAT+](https://www.statnews.com/wp-content/themes/stat/images/stat-plus-breaker-bg.png) [ ![STAT+](https://www.statnews.com/wp-content/themes/stat/images/stat-plus-logo-white.svg) ](https://www.statnews.com/stat-plus/) ## This article is exclusive to STAT+ subscribers ### Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? 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