Skip to main content
Donate
Current Pipeline
FAST Logo Funded
ASO

UGX • GTX-102

Ultragenyx Pharmaceuticals (UGX) is exploring an investigational Antisense Oligonucleotide (ASO) approach for paternal gene activation as a potential treatment for Angelman syndrome.

Phase 3

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

  • progress mobile progress mobile progress mobile progress mobile progress mobile progress mobile

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

A therapeutic approach that uses modified RNA or DNA molecules that bind to the RNA of the UBE3A-ATS (UBE3A antisense transcript).

Investigational Drug Name

GTX-102

Current Clinical Trials

Meet with Genetic Counselor
  • Recruiting

    Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire)

    Genotypes:

    Deletion

    Trial Name:

    Aspire

    Learn More
  • Enrolling by Invitation

    Long-term Extension of GTX-102 in Angelman Syndrome

    Genotypes:

    Deletion

    Trial Name:

    Long-term Extension (LTE)

    Learn More
  • Active, Not Recruiting

    A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

    Genotypes:

    Deletion

    Trial Name:

    KIK-AS

    Learn More

Program Overview

In early 2012, FAST began funding Scott Dindot’s laboratory at Texas A&M University to understand the mechanism by which the UBE3A-antisense transcript (UBE3A-AS) silences the paternal UBE3A allele, why it is only silenced in neurons, and how that process may be leveraged as a potential therapy for AS. In late 2017, the Dindot laboratory identified an investigational ASO that, through preliminary studies, was shown to interfere with the imprinting mechanism of the paternal UBE3A allele in neurons, and unsilenced the gene. FAST wanted to ensure that the development of this program moved forward in the most expeditious, safe, and detailed manner possible, with the primary goal of moving into a human clinical trial if preclinical studies supported further investigation. To do this, FAST formed GeneTx Biotherapeutics LLC (GeneTx), a for-profit limited liability company, singularly focused to develop an investigational ASO for potential future use in a Phase 1/2 clinical trial for individuals living with AS.

In July 2022, GeneTX was sold to Ultragenyx Pharmaceuticals. Ultragenyx is now the sole Sponsor of the development program.

Read more

Recent Updates

  • December 2024

    Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

    Read more
  • October 2024
  • July 2024

    Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program

    Read More
  • April 2024

    Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102

    Read More
  • January 2024

    Ultragenyx Pharmaceutical Ultragenyx announces completion of enrollment in Global Phase 1/2 Trial of GTX-102

    Read more

Media

  • Update on Ultragenyx's Angelman Syndrome Aspire Trial

  • Update on the GTX-102 program for Angelman Syndrome

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.