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Current Pipeline
ASO

Ionis • ION582

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

Phase 2

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

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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

ION582

Current Clinical Trials

Meet with Genetic Counselor
  • Active, Not Recruiting

    HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

    Genotypes:

    Mutation and Deletion

    Trial Name:

    HALOS

    Learn More

Recent Updates

  • November 2024

    Ionis Announces Pivotal Phase 3 Trial Design for ION582 in Angelman Syndrome.

    Read More
  • July 2024

    Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome

    Read More
  • May 2024

    Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome

    Read More

Media

  • An Update on HALOS Clinical Trial in Individuals Living with 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.