MTM1

Genethon Announces Publication in The Lancet Neurology of Clinical Trial Results of a Gene Therapy for Myotubular Myopathy, a Severe Muscle Disease

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星期五, 十一月 17, 2023
Research, Technology, Genetics, Clinical Trials, Nanotechnology, Biotechnology, Pharmaceutical, Health, Science, Other Science, The Lancet, X-linked myotubular myopathy, Acquisition, Therapy, Gene, Pathology, Head, Clinical trial, MTM1, Death, Muscle weakness, Periodic breathing, Muscular Dystrophy Association, Astellas Pharma, Neurology, Myopathy, Knowledge, Lancet Neurology, Pharmaceutical industry, Audentes/Noortekoondis, Inserm

Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.

Key Points: 
  • Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.
  • Characterized by extreme muscle weakness and severe respiratory distress, 50% of affected children die before age 18 months and 75% die before age 10.
  • The gene therapy uses an adeno-associated viral vector (AAV8) to deliver a copy of the MTM1 gene.
  • It took years of research to imagine, design and demonstrate the efficacy of the gene therapy for this very severe and complex disease.

Astellas and Kate Therapeutics Announce Exclusive License Agreement for KT430

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星期四, 六月 8, 2023
Astellas Pharma, MTM1, Kate, DSM-IV codes, AT132, Muscle weakness, Neuromuscular disease, Patient, SAN, X-linked myotubular myopathy, Safety, Respiratory failure, TSE, Pharmaceutical industry

TOKYO and SAN DIEGO, June 8, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas"), and Kate Therapeutics ("KateTx") today announced an exclusive license agreement to develop and commercialize KT430. KT430 is a preclinical next-generation investigational gene therapy that delivers a functional copy of the MTM1 gene via a novel MyoAAV capsid to treat X-linked myotubular myopathy (XLMTM), a serious, life-threatening, rare neuromuscular disease characterized by extreme muscle weakness, respiratory failure and early death.

Key Points: 
  • TOKYO and SAN DIEGO, June 8, 2023 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas"), and Kate Therapeutics ("KateTx") today announced an exclusive license agreement to develop and commercialize KT430.
  • Under the terms of the agreement, Astellas will make an undisclosed upfront payment to KateTx, which is also eligible to receive development, regulatory and commercial milestone payments, plus royalties on worldwide sales.
  • Astellas will receive an exclusive worldwide license to develop, manufacture and commercialize KT430.
  • "This agreement brings together Astellas and KateTx's collective patient-focused missions, allowing us to evaluate how to advance this new potential therapy for people diagnosed with XLMTM," stated Adam Pearson, Chief Strategy Officer at Astellas.

Dynacure Receives Fast Track Designation for DYN101, an Investigational Antisense Oligonucleotide for the Treatment of Myotubular and Centronuclear Myopathies

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星期四, 一月 6, 2022
People, View, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Degenerative disease, Review, Patient, Ventilation, Life, MTM1, Ionis Pharmaceuticals, Disease, Centronuclear myopathy, FDA, BIN1, Union, Drug development, Muscle weakness, Multimedia, Autosome, CNM, Pharmaceutical industry, X-linked myotubular myopathy, DNM2

Receiving Fast Track Designation will provide us with greater access to FDA and guidance on regulatory pathways," said Leen Thielemans, Chief Development Officer of Dynacure.

Key Points: 
  • Receiving Fast Track Designation will provide us with greater access to FDA and guidance on regulatory pathways," said Leen Thielemans, Chief Development Officer of Dynacure.
  • Myotubular and Centronuclear Myopathies (CNM) are serious, rare, life-threatening disorders that affect skeletal muscles from birth.
  • Myotubular and Centronuclear Myopathies affect an estimated 4,000 to 5,000 patients in the European Union, United States, Japan and Australia1.
  • Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals.

Dynacure Announces Strategic Collaboration and Option Agreement with Nippon Shinyaku

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星期三, 十一月 10, 2021
People, Hematology, Autosome, View, CNM, HQ-9, Orphan drug, BIN1, European Union, Disease, EMA, Food, Medicines and Healthcare products Regulatory Agency, European Medicines Agency, Lists of diseases, Union, Degenerative disease, Centronuclear myopathy, Multimedia, MTM1, Research, US Foods, Medication, Program, Ventilation, Gynaecology, Ionis Pharmaceuticals, Patient, PMDA, Urology, Drug discovery, Mouse, Muscle weakness, Survival, Pharmaceuticals and Medical Devices Agency, Marketing, Life, FDA, Code of Federal Regulations, Drug development, Adult, Pharmaceutical industry

Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the Phase 1/2 study, UNITE-CNM.

Key Points: 
  • Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the Phase 1/2 study, UNITE-CNM.
  • "We look forward to leveraging the skills and network Nippon Shinyaku brings and advancing this important clinical program toward commercialization."
  • Nippon Shinyaku, established in 1919, has a long-history as a research-oriented pharmaceutical company in Japan.
  • Areas of specialty for both R&D and marketing by Nippon Shinyaku include hematology, intractable and rare diseases, urology and gynecology.