Drug intelligence / Profile preview

Myo-P4

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

Overview

Myo-P4 is an encapsulated cell therapy product candidate being developed by Release Therapeutics for the treatment of Metachromatic Leukodystrophy (MLD). The therapy utilizes the company's proprietary Encapsulated Cell Technology (ECT), which consists of genetically engineered human cells enclosed within a semi-permeable, immunoisolatory polymer membrane. These cells are designed to continuously produce and secrete Arylsulfatase A (ARSA), the enzyme that is deficient in patients with MLD. By providing a sustained, localized source of the enzyme, Myo-P4 aims to prevent the accumulation of sulfatides in the central nervous system, thereby halting or slowing the progressive demyelination and neurodegeneration associated with the disease. The encapsulation device allows the therapeutic protein to diffuse out while protecting the engineered cells from host immune rejection, potentially eliminating the need for systemic immunosuppression.

Brand names
Myo-P4Myo-P-4Myo-P 4
02

Targets

Sulfatide

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