Treatment for congenital myasthenic syndromes

Amplo Biotechnology is focused on developing innovative gene therapies targeting diseases of the neuromuscular junction (NMJ). Their lead candidate, AMP-101, utilizes AAV (adeno-associated virus) technology to deliver the Dok-7 protein, which is essential for the formation and maintenance of the NMJ. This therapy aims to address Congenital Myasthenic Syndromes and has potential applications for other neuromuscular disorders, including Emery-Dreyfuss Muscular Dystrophy and ALS. Another key product, AMP-201, employs AAV technology to express the Collagen Q protein, which is vital for proper skeletal muscle function, specifically targeting ColQ Congenital Myasthenic Syndrome.

Amplo Biotechnology emphasizes a capital-efficient approach to drug development, focusing solely on AAV therapies for NMJ diseases. By leveraging established delivery technologies and manufacturing methods, the company aims to minimize risks associated with the drug development process. Their commitment to collaboration with leading experts in the field underscores their dedication to providing effective treatments for rare and ultra-rare patient populations suffering from neuromuscular disorders.

United States

Amplo Biotechnology is a private Gene Therapy company focusing on therapy for diseases of the neuromuscular junction. 

Central Nervous System (CNS)

Genetic Diseases

Musculoskeletal Diseases

Nervous System Diseases

Amplo Biotechnology

Amyotrophic lateral sclerosis

Congenital Myasthenic Syndrome

Muscular dystrophy

AMP-101

Biologics

Gene Therapy

AMP-201

Towson, Maryland, United States of America

Maryland

Adeno-associated viral vector (AAV)

Viral Therapies/Virotherapies

Therapeutic

Amplo Biotechnology

Company Details