AMP-101

DB18253InvestigationalGene Therapies

AMP-101 is a gene therapy comprising an adeno-associated virus type 9 (AAV9) vector encoding the human Dok-7 protein. It is under investigation for the treatment of Dok-7-associated myasthenia.

Also known as
Adeno-associated virus serotype 9 vector encoding the Dok7 protein
Code names
AMP-101

What you can answer from here — as of September 28, 2026

1Clinical trialPhase, status and sponsor resolved per trial
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Which companies are running trials of AMP-101, in which indications and phases, and which of those programs are still active?

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