Kate Therapeutics Debuts With $51 Million Series A to Develop Next-Generation Genetic Medicines to Treat Muscle and Heart Diseases
Series A financing co-led by Westlake Village BioPartners and Versant Ventures
Company advancing systemically delivered, skeletal and cardiac muscle targeted, liver de-targeted capsid and cargo technology platforms
Initial pipeline addressing myotoni... Regenerative Medicine, Venture Capital Kate Therapeutics, gene therapy, X-linked myotubular myopathy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 8, 2023 Category: Pharmaceuticals Source Type: news
Astellas and Kate Therapeutics Announce Exclusive License Agreement for KT430
KT430 is a preclinical, next-generation investigational gene therapy to treat X-linked myotubular myopathy (XLMTM)
TOKYO and SAN DIEGO, June 8, 2023 -- (Healthcare Sales & Marketing Network) -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki ... Biopharmaceuticals, Regenerative Medicine, Licensing Astellas Pharma, Kate Therapeutics, X-linked myotubular myopathy, gene therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 8, 2023 Category: Pharmaceuticals Source Type: news
What Are Some Initial Evaluations for Suspected Congenital Muscle Diseases?
Discussion
Usually congenital myopathies (CM, e.g. nemaline, core, centronuclear myopathies, etc.) and congenital muscular dystrophy (CMD, e.g. LAMA2-related, collage VI-related, alpha-dystroglycan-related muscular dystrophy, etc.) have been diagnosed based on physical examination and histopathology. Traditionally CMs are due to problems with the muscle contractile apparatus and structures that assist excitation-contraction coupling. CMD are due to problems with the extracellular matrix, muscle membrane and sarcolemmal membrane. Differentiating between CM and CMD have become more blurred as genetic testing and additional r...
Source: PediatricEducation.org - January 17, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
Treatments for Inherited Neuromuscular Diseases of Childhood Treatments for Inherited Neuromuscular Diseases of Childhood
This review highlights recent advances in gene-specific therapies for neuromuscular diseases of childhood, including spinal muscular atrophy, Duchenne muscular dystrophy, and centronuclear myopathy.Seminars in Neurology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 27, 2020 Category: Consumer Health News Tags: Neurology & Neurosurgery Journal Article Source Type: news
New Human Gene Therapy editorial: Concern following gene therapy adverse events
(Mary Ann Liebert, Inc./Genetic Engineering News) Response to the recent report of the deaths of two children receiving high doses of a gene therapy vector (AAV8) in a Phase I trial for X-linked myotubular myopathy (MTM). The news " is a tragic reminder of how difficult it is to predict outcomes in first-in-human studies (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - July 2, 2020 Category: Infectious Diseases Source Type: news
2 boys' deaths halt S.F. company's rare disease gene therapy trial
Two boys died in a critical gene therapy trial against a genetic disease, forcing the company behind the therapy and regulators to halt the study.
Audentes Therapeutics Inc., a San Francisco company that was sold in January to Astellas Pharma of Japan for $3 billion, said both boys with X-linked myotubular myopathy had been given a higher dose of the therapy and had pre-existing liver problems.
The news comes as gene therapies face question s as well as a pivotal moment. BioMarin Pharmaceutical… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 29, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news
2 boys' deaths halt S.F. company's rare disease gene therapy trial
Two boys died in a critical gene therapy trial against a genetic disease, forcing the company behind the therapy and regulators to halt the study.
Audentes Therapeutics Inc., a San Francisco company that was sold in January to Astellas Pharma of Japan for $3 billion, said both boys with X-linked myotubular myopathy had been given a higher dose of the therapy and had pre-existing liver problems.
The news comes as gene therapies face question s as well as a pivotal moment. BioMarin Pharmaceutical… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 29, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news
Gene Therapy Trial Halted After Second Patient Death Gene Therapy Trial Halted After Second Patient Death
A clinical trial of Audentes Therapeutics ' investigational gene therapy for X-linked myotubular myopathy is on hold after two patients died following receipt of a high dose of the drug.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - June 29, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Dynacure Appoints David Garrett as Chief Financial Officer
STRASBOURG, France, Sept. 3, 2019 -- (Healthcare Sales & Marketing Network) -- Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, today announced the appointment of David Garrett,... Biopharmaceuticals, Personnel Dynacure, Centronuclear Myopathies (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - September 3, 2019 Category: Pharmaceuticals Source Type: news
A molecule for fighting muscular paralysis
(Universit é de Gen è ve) Myotubular myopathy is a severe genetic disease that leads to muscle paralysis. Although no treatment currently exists, researchers from the UNIGE-with the University of Strasbourg,- have identified a molecule that not only greatly reduces the progression of the disease but also boosts life expectancy in animal models by a factor of seven. Since the molecule -- known as tamoxifen -- is already used for breast cancer, the researchers hope to soon set up a clinical trial. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 19, 2018 Category: International Medicine & Public Health Source Type: news
Audentes Therapeutics Board of Directors Appoints Co-Founder and Chief Executive Officer Matthew R. Patterson as Chairman
Louis G. Lange, M.D., Ph.D. appointed as Lead Independent Director
SAN FRANCISCO, Nov. 5, 2018 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing in... Biopharmaceuticals, Personnel Audentes Therapeutics, Myotubular Myopathy, Crigler-Najjar syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 5, 2018 Category: Pharmaceuticals Source Type: news
Audentes Announces Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to AT132 for the treatment of X-Linked Myotubular Myopathy
SAN FRANCISCO, June 5, 2018 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-... Biopharmaceuticals, Regulatory Audentes Therapeutics, gene therapy, Myotubular Myopathy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 5, 2018 Category: Pharmaceuticals Source Type: news
Audentes Therapeutics Appoints Mark A. Goldberg, M.D. to its Board of Directors
SAN FRANCISCO, Dec. 13, 2017 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatenin... Biopharmaceuticals, Personnel Audentes Therapeutics, gene therapy, X-Linked Myotubular Myopathy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 13, 2017 Category: Pharmaceuticals Source Type: news
Audentes Therapeutics Announces Publication of Data from RECENSUS, a Retrospective Medical Record Review of Patients With X-Linked Myotubular Myopathy
SAN FRANCISCO, Nov. 21, 2017 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatenin... Biopharmaceuticals Audentes Therapeutics, X-Linked Myotubular Myopathy, RECENSUS Study (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - November 21, 2017 Category: Pharmaceuticals Source Type: news
