AMP-201
ColQ Congenital Myasthenic Syndrome (CMS)
PreclinicalActive
Key Facts
Indication
ColQ Congenital Myasthenic Syndrome (CMS)
Phase
Preclinical
Status
Active
Company
About Amplo Biotechnology
Amplo Biotechnology is a focused, capital-efficient gene therapy company advancing a pipeline of AAV-based therapies for neuromuscular junction disorders. Its lead assets, AMP-101 (AAV-Dok7) and AMP-201 (AAV-ColQ), are in preclinical development for specific Congenital Myasthenic Syndromes, with broader potential applications in conditions like ALS and muscular dystrophy. The company employs a de-risked development approach using known technologies and has secured non-dilutive STTR funding, positioning it 10-12 months from a clinical trial application for its lead candidate.
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