# Translational development of recombinant protein therapeutic for LGMD2B

> **NIH NIH R41** · MYOFINITY BIOSCIENCES INC · 2022 · $259,487

## Abstract

PROJECT ABSTRACT
 This Phase I STTR project will accomplish key milestones in commercializing a protein therapeutic for
dysferlinopathies that will enhance the repair capacity of muscle cell membranes compromised by mutations in
the dysferlin gene. The dysferlinopathies include Limb Girdle Muscular Dystrophy Type 2B (LGMD2B), Miyoshi
Myopathy (MMD1) and other, rarer myopathies that all present as adult-onset debilitating muscle diseases
characterized by extensive muscle damage and progressive weakness. All these myopathies arise from
mutations in the gene encoding an essential muscle membrane repair protein, dysferlin. Progress in treatment
of dysferlinopathies has been hampered by the large size of the protein, which complicates gene therapy
approaches, and the complex function of the native dysferlin protein. Myos proposes to develop a treatment for
dysferlinopathies through protein supplementation therapy using a key binding partner of dysferlin, the tripartite
motif protein 72/mitsugumin 53 protein (MG53). MG53 is an essential regulator of membrane repair in skeletal
and cardiac muscle that binds dysferlin and can compensate for the loss of dysferlin in membrane repair. To
provide protein supplementation therapy for dysferlinopathies, we will use recombinant human MG53 (rhMG53)
protein. rhMG53 binds membrane damage sites to enhance membrane repair capacity in cultured cells and
dystrophic animal models when applied outside the cell. Based on these studies, Myos seeks to develop
MyoTRIM, novel engineered version of rhMG53, to treat dysferlinopathy. MyoTRIM is designed to enhance repair
and restore the compromised membrane repair in dysferlinopathy muscle, providing a complementary treatment
approach to other dysferlinopathy therapies in development. The objective of this project is to develop Chemistry,
Manufacturing, and Control (CMC) methods to produce MyoTRIM,protein and to test whether MyoTRIM can
rescue pathology in a dysferlinopathy mouse model using two specific aims. Aim 1 will develop initial CMC
procedures for MyoTRIM. Aim 2 will complete pre-clinical trial for MyoTRIM efficacy in the Bla/J mouse model of
dysferlinopathy. Successful completion of this Phase I project will advance the commercialization MyoTRIM and
provide a significant impact on public health by improving muscle membrane repair to treat muscular dystrophies,
independent of gene or mutation. MyoTRIM will provide a platform technology to target other diseases involving
necrotic cell death.
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## Key facts

- **NIH application ID:** 10483343
- **Project number:** 1R41AR081205-01
- **Recipient organization:** MYOFINITY BIOSCIENCES INC
- **Principal Investigator:** Noah Weisleder
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $259,487
- **Award type:** 1
- **Project period:** 2022-08-01 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10483343

## Citation

> US National Institutes of Health, RePORTER application 10483343, Translational development of recombinant protein therapeutic for LGMD2B (1R41AR081205-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10483343. Licensed CC0.

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