Muscular Dystrophy Association Partners with Gene Therapy Company to Advance Treatment Options for Neuromuscular Disease
Gene Therapy Company Selects Muscular Dystrophy Association as Its Exclusive Canadian Partner for Clinical Trial of Novel Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2D
Breakthrough Collaboration to Provide New Hope for Patients and Families Affected by Devastating Disease
March 8, 2023 - Toronto, ON - Muscular Dystrophy Canada (MDC) is excited to announce a new partnership with GeneTX Biotherapeutics, a leading gene therapy company, to conduct a Phase 2 clinical trial in Canada of a novel gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D).
LGMD2D is a rare and debilitating neuromuscular disease that affects the muscles of the shoulders, arms, and legs. It is caused by mutations in the gene that encodes the protein dysferlin. Symptoms typically appear in childhood and gradually worsen over time, leading to progressive muscle weakness and loss of function.
The gene therapy being developed by GeneTX Biotherapeutics is designed to deliver a functional copy of the dysferlin gene to muscle cells using an adeno-associated virus (AAV) vector. AAV vectors are safe and effective vehicles for gene delivery that have been successfully used in clinical trials for other neuromuscular diseases.
"This partnership with GeneTX Biotherapeutics is a major milestone in our ongoing efforts to find new and effective treatments for neuromuscular diseases," said Abigail Powell, CEO of Muscular Dystrophy Canada. "Gene therapy has the potential to be a transformative treatment for LGMD2D and other neuromuscular diseases, and we are excited to be able to offer this opportunity to our patients and families."
The Phase 2 clinical trial will be conducted at the Children's Hospital of Eastern Ontario (CHEO) in Ottawa and will enroll up to 15 patients with LGMD2D. The trial will evaluate the safety and efficacy of the gene therapy and will assess its impact on muscle function and other outcome measures.
"We are pleased to partner with Muscular Dystrophy Canada to bring our novel gene therapy to patients in Canada," said Dr. David Meeker, CEO of GeneTX Biotherapeutics. "Muscular Dystrophy Canada has a long history of supporting research and providing support to patients and families affected by neuromuscular diseases, and we believe that this partnership will help us to accelerate the development of our gene therapy and bring it to patients as quickly as possible."
The Muscular Dystrophy Association is a national, non-profit organization that provides support and services to individuals and families affected by neuromuscular diseases. The Association funds research, provides advocacy, offers educational programs, and provides support services to help people with neuromuscular diseases live full and productive lives.
GeneTX Biotherapeutics is a clinical-stage gene therapy company focused on developing transformative treatments for rare and devastating CNS diseases. The company's lead product candidate, GTX-102, is a gene therapy for the treatment of LGMD2D.
For more information, please contact:
Muscular Dystrophy Canada
Abigail Powell, CEO
apowell@muscle.ca
(416) 488-0030
GeneTX Biotherapeutics
Dr. David Meeker, CEO
dmeeker@genetxbio.com
(617) 498-7398
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