Grants and Contributions:
Title:
Non-Viral Gene Therapy for Myotubular Myopathy: Skeletal Muscle Targeted
Lipid Nanoparticle-Mediated Delivery of the MTM1 Gene to Restore Muscle
Function.
Agreement Number:
1025235
Agreement Value:
$204,387.00
Agreement Date:
Sep 5, 2024 - Feb 5, 2026
Description:
Myotubular myopathy is a severe congenital disorder marked by muscle
weakness and hypotonia due to mutations in the MTM1 gene, which encodes
myotubularin. This protein is crucial for muscle cell maintenance and repair, and
its deficiency disrupts these processes, causing significant muscle dysfunction.
Current treatments are symptomatic and fail to address the genetic cause,
necessitating therapies that target the root of the disease. Gene therapy,
particularly with viral vectors, shows promise but has limitations such as immune
responses and high costs. Recent safety setbacks in AAV8-MTM1 gene therapy
trials underscore the need for alternative delivery methods. Lipid nanoparticles
(LNPs), successful in mRNA vaccine delivery, offer a promising non-viral
alternative. NRC’s proprietary LNPs, targeted to skeletal muscle cells, present an
innovative solution. This project hypothesizes that LNP-mediated delivery of a
functional MTM1 gene can restore myotubularin function, enhancing muscle
strength and function in myotubular myopathy patients without the risks
associated with viral vectors.
Organization:
National Research Council Canada
Expected Results:
In the short term, anticipated outcomes will be strengthened collaborations across industry, academia, and government to support research excellence. In the medium term, anticipated outcomes will be the development of new and potentially disruptive technologies with collaborators. In the long term, find collaborative solutions to public policy challenges and create stronger innovation systems.
Location:
Toronto, Ontario, CA M5G 1X8
Reference Number:
172-2024-2025-Q3-1025235
Agreement Type:
Grant
Report Type:
Grants and Contributions
Recipient Business Number:
108162330
Recipient Type:
Not-for-profit organization or charity
Recipient's Legal Name:
The Hospital for Sick Children
Federal Riding Name:
University--Rosedale
Federal Riding Number:
35112
Program:
Collaborative Science, Technology and Innovation Program - Collaborative R&D Initiatives
Program Purpose:
Collaborate on multiparty research and development programs to catalyze transformative, high-risk, high-reward research with the potential for game-changing scientific discoveries and technological breakthroughs in priority areas.
NAICS Code:
541710