Drug intelligence / Profile preview

Myo-P5

Development stage
Preclinical
Lead developer
Release Therapeutics
Modality
Cell Therapies
Administration
Implant
01

Overview

Myo-P5 is an encapsulated cell therapy candidate being developed by Release Therapeutics for the treatment of Sanfilippo disease Type A (Mucopolysaccharidosis type IIIA). The therapy utilizes the company's proprietary Encapsulated Cell Technology (ECT), which consists of a semi-permeable polymer capsule containing genetically engineered cells that continuously produce and secrete the enzyme N-sulfoglucosamine sulfohydrolase (SGSH). By implanting the device, typically into the central nervous system, the therapy provides a sustained, long-term supply of the missing enzyme directly to the affected tissues, bypassing the blood-brain barrier. The encapsulation membrane protects the producer cells from the host's immune system, eliminating the need for systemic immunosuppression and reducing the frequency of invasive administrations compared to traditional enzyme replacement therapies.

Brand names
Myo-P5Myo-P-5Myo-P 5

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