X-linked myotubular myopathy

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

Retrieved on: 
Friday, November 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.

Kate Therapeutics Debuts With $51 Million Series A to Develop Next-Generation Genetic Medicines to Treat Muscle and Heart Diseases

Retrieved on: 
Thursday, June 8, 2023
Tissue, Heart, Versant, Capsid, Broad Institute, University, DM1, Pardis Sabeti, Cardiac muscle, Research, MIT, FSHD, RNA, Harvard University, AAV, Facioscapulohumeral muscular dystrophy, Patient, Machine learning, SAN, Cellular Dynamics International, Therapy, X-linked myotubular myopathy, Safety, Pharmaceutical industry, Vaccine, Cell

SAN DIEGO, June 8, 2023 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, today emerged from stealth mode with a $51 million Series A financing co-led by founding investor Westlake Village BioPartners and Versant Ventures, with participation from Osage University Partners and UF Innovate | Ventures. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM), the details of which are described in a separate press release issued today.

Key Points: 
  • "We are excited to announce KateTx's launch and what this means for patients suffering from muscle and heart diseases," said Kevin Forrest, Ph.D., president, CEO and a director of KateTx.
  • "KateTx is applying novel capsid and cargo technology platforms to enable skeletal and cardiac muscle targeting and liver de-targeting.
  • KateTx's current focus is identifying and advancing clinical candidates for DM1 and FSHD, as well as for other genetic muscle and heart diseases.
  • "With this Series A financing and licensing agreement, KateTx will be able to progress its deep pipeline of internal programs."

Astellas and Kate Therapeutics Announce Exclusive License Agreement for KT430

Retrieved on: 
Thursday, June 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.

Sarepta Therapeutics Announces Progress on the MyoAAV Program and Exclusive Licensing Agreement with The Broad Institute for MyoAAV Next-generation Capsids for Rare Genetic Diseases

Retrieved on: 
Monday, August 8, 2022
Liver, U.S. Securities and Exchange Commission, Survival, Cardiac muscle, Risk, Re Recher's Will Trusts, Cell, GLOBE, Broad Institute, Duchenne muscular dystrophy, Gene expression, SEC, Patent, Sale, Harvard University, Sarepta Therapeutics, Security (finance), Research, Gene editing, Annual report, Investor Network on Climate Risk, Capsid, United States Patent and Trademark Office, Review, X-linked myotubular myopathy, Instagram, Therapy, SRPT, Company, COVID-19, Twitter, Degenerative disease, RNA, MIT, Viral load, NASDAQ, Science, Goal, DMD, LinkedIn, Sarepta, Muscular dystrophy, Lists of diseases, Efficiency, AAV, Vaccine, Pharmaceutical industry, Facebook, Duchenne

CAMBRIDGE, Mass., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that following progress on its sponsored research agreement on the MyoAAV program, it has executed a license agreement with the Broad Institute of MIT and Harvard (Broad Institute) for MyoAAV in Duchenne muscular dystrophy and certain other neuromuscular and cardiac indications. The announcement follows confirmation by Sarepta’s internal research and manufacturing teams of earlier published research from Broad Institute, which was conducted under a research agreement that began in 2020.

Key Points: 
  • The announcement follows confirmation by Sareptas internal research and manufacturing teams of earlier published research from Broad Institute, which was conducted under a research agreement that began in 2020.
  • In addition to an upfront payment, Broad Institute is entitled to future royalties and milestone payments, details of which were not disclosed.
  • Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.
  • For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta.

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

Retrieved on: 
Thursday, January 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 to Present at 40th Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 3, 2022
Degenerative disease, Ionis Pharmaceuticals, Conference, Multimedia, CNM, Patient, Drug development, Centronuclear myopathy, Pharmaceutical industry, X-linked myotubular myopathy

The presentation will take place January 11, 2022, at 9:00 AM Eastern Standard Time.Company management will also be available to participate in one-on-one meetings with investors.

Key Points: 
  • The presentation will take place January 11, 2022, at 9:00 AM Eastern Standard Time.Company management will also be available to participate in one-on-one meetings with investors.
  • Dynacure is a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options.
  • The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drug candidates.
  • Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.

Audentes Therapeutics Announces FDA Lifts Hold on ASPIRO Clinical Trial of AT132 for Treatment of X-Linked Myotubular Myopathy (XLMTM)

Retrieved on: 
Thursday, December 24, 2020
FDA, Health, Genetics, Clinical trials, Pharmaceutical, Biotechnology, Astellas Pharma, Mitsubishi UFJ Financial Group, Centronuclear myopathy, Rare diseases, X-linked myotubular myopathy, Companies, Myotubularin 1, Myopathy, Branches of biology, Life sciences

Audentes Therapeutics , an Astellas genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold for the ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy (XLMTM).

Key Points: 
  • Audentes Therapeutics , an Astellas genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold for the ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy (XLMTM).
  • XLMTM is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure and early death.
  • Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of XLMTM.
  • Audentes Therapeutics, an Astellas company, is developing genetic medicines with the potential to deliver transformative value for patients.

Audentes Announces Upcoming Presentations at the 24th International Annual Congress of the World Muscle Society, Including New Data From ASPIRO, the Clinical Trial Evaluating AT132 in Patients With X-Linked Myotubular Myopathy (XLMTM)

Retrieved on: 
Monday, September 30, 2019
Health, Genetics, Clinical trials, Pharmaceutical, Biotechnology, Rare diseases, Centronuclear myopathy, Myotubularin 1, Jay-Z, X-linked myotubular myopathy, Myopathy, Aspiro, X-linked Myotubular Myopathy, AT132 for the treatment of XLMTM, Audentes Therapeutics, Inc.

Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls).

Key Points: 
  • Dr. Dowling will present new efficacy and safety data from the ASPIRO Phase 1/2 dose escalation cohorts (10 treated patients and 2 controls).
  • We are excited to share new efficacy and safety data from our ASPIRO study, stated Natalie Holles, President and Chief Operating Officer.
  • Audentes is planning several additional presentations during the conference, including a company-sponsored symposium that will provide an in-depth review of XLMTM and the ASPIRO study results.
  • Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM).

Audentes Therapeutics Announces Upcoming Presentations at 22nd Annual Meeting of the American Society of Gene and Cell Therapy Including New Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy

Retrieved on: 
Monday, April 15, 2019
Enteral feeding, Medicine, Organ systems, Medical specialties, Centronuclear myopathy, Rare diseases, Myotubularin 1, X-linked myotubular myopathy, Gastrostomy, Myopathy, Feeding tube

Audentes has a range of additional presentations planned during the conference, including a sponsored symposium that will provide an in-depth review of the new data from ASPIRO.

Key Points: 
  • Audentes has a range of additional presentations planned during the conference, including a sponsored symposium that will provide an in-depth review of the new data from ASPIRO.
  • More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support.
  • Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of X-linked Myotubular Myopathy (XLMTM).
  • Audentes is a focused, experienced and passionate team driven by the goal of improving the lives of patients.

Audentes Therapeutics to Provide Update on New Positive Interim Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy, from the 23rd International Annual Congress of the World Muscle Society

Retrieved on: 
Friday, October 5, 2018
Rare diseases, Centronuclear myopathy, Myopathy, X-linked myotubular myopathy

The call will focus on new positive interim data from ASPIRO, the Phase 1/2 clinical trial of AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).

Key Points: 
  • The call will focus on new positive interim data from ASPIRO, the Phase 1/2 clinical trial of AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).
  • All treated patients continue to show meaningful improvements in neuromuscular and respiratory function, with no new treatment-related SAEs reported since the last scientific update in May 2018.
  • To access a live webcast of the conference call, please visit the Investor and Media page of the Audentes website at www.audentestx.com .
  • We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.