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AT845, Pompe experimental treatments | Pompe Disease News | illustration of patient getting IV medicine

AT845 for Pompe Disease

Last updated July 21, 2022, by Lindsey Shapiro, PhD

✅ Fact-checked by José Lopes, PhD

FAQs about AT845

AT845 is an experimental gene therapy that is not yet approved for the treatment of Pompe disease. It is designed to deliver to muscle cells a working copy of the GAA gene — mutations which cause Pompe. Data from animal models suggest the therapy may be able to prevent the toxic buildup of glycogen, a sugar molecule, that’s characteristic of the disease. The therapy also is expected to improve muscle function.

AT845 is in the early stages of clinical testing, with a first Phase 1/2 trial in Pompe patients on clinical hold. As AT845’s safety and efficacy need to be established, it is too early to say if or when the therapy might be approved in the U.S.

It is not known whether AT845 is safe for people who are pregnant or breastfeeding.

The efficacy of AT845 has not been established in clinical trials. The time course on which AT845 may lead to clinical benefit also is not yet known.

Hair loss and weight gain have not been reported as side effects of AT845. Patients who experience any unexpected side effects after starting a new treatment should speak with their healthcare providers.

Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.


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