Drug Profile

Rugonersen - Roche

Alternative Names: RG 6091; RO 7248824; UBE3A-LNA

Latest Information Update: 24 Apr 2025

Price :

$50 ^*

Note:

Adis is an information provider. We do not sell or distribute actual drugs.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

Originator Roche
Class Antisense oligonucleotides
Mechanism of Action UBE3A protein expression stimulants

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Phase I Angelman syndrome

Most Recent Events

15 Apr 2025 Rugonersen licensed to Oak Hill Bio worldwide
15 Apr 2025 Oak Hill Bio plans a phase III trial for Angelman syndrome, in early 2026 (Intrathecal)
19 Dec 2023 Phase I development is ongoing the US and United kingdom

You need to be a logged in or subscribed to view this content

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com