Muscular dystrophy

Kate Therapeutics Unveils Platform and Pipeline Progress at American Society of Gene & Cell Therapy 2024 Annual Meeting

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Giovedì, Maggio 9, 2024
Mortality, FDA, University, Facioscapulohumeral muscular dystrophy, Mouse, RNA interference, Safety, Tissue, Heart, Animal, Death, MD, Primate, Cell, Duchenne, Science, Disease, Rockefeller University, Patient, Liver, Society, Gene, X-linked myotubular myopathy, Pediatrics, Gene expression, Judgement, Duchenne muscular dystrophy, Capsid, Annual general meeting, SAN, Muscular dystrophy, Vaccine

SAN DIEGO, May 9, 2024 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, will present detailed preclinical efficacy and safety results on its pipeline and platform at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting being held May 7-11, 2024, in Baltimore, MD and virtually. The results support the best-in-class potential of KateTx's newest generation of skeletal muscle- and heart-targeted, liver de-targeted engineered MyoAAV capsids and gene regulation technology to treat Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and other devastating genetic skeletal muscle and heart diseases.

Key Points: 
  • KateTx remains on track to select development candidates for additional muscle and cardiac programs in the near term.
  • "KateTx has made significant advances over the past 12 months," said Kevin Forrest, Ph.D., co-founder, president and CEO of KateTx.
  • KateTx's results show the potential to give a one-time gene therapy that potently suppresses DUX4 whenever it is expressed in skeletal muscles.
  • In vitro, the company's MyoAAV-LD mediated RNAi gene therapy candidate showed potent knockdown of DUX4 in FSHD patient myotubes with no off-target effects.

Pliant Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results

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Lunedì, Maggio 6, 2024
Idiopathic pulmonary fibrosis, FVC, Investment, Hospital, Safety, Primary sclerosing cholangitis, Research, IPF, Collagen, Trial of the century, PET, Acceleration, DMD, Tumor microenvironment, PSC, European, Novartis, Week, Therapy, Vital capacity, Muscular dystrophy, Patient, TGF, Fibrosis, Cirrhosis, Integrin, Duchenne muscular dystrophy, Biomarker, Ethics committee, Massachusetts General Hospital, ELF, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., May 06, 2024 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a late-stage clinical biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, today provided a corporate update and reported first quarter 2024 financial results.

Key Points: 
  • As part of the adaptive Phase 2b/3 implementation, the Phase 2b portion of BEACON-IPF was upsized from 267 patients to 360 patients.
  • Enrollment is progressing and we expect to complete enrollment of the upsized Phase 2b study expected in the first quarter of 2025.
  • The increase was due to higher operating expenses coupled with a decrease in collaboration revenues under the Novartis agreement during the quarter.
  • As of March 31, 2024, the Company had cash, cash equivalents and short-term investments of $483.9 million.

Edgewise Therapeutics Reports First Quarter 2024 Financial Results and Recent Business Highlights

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Giovedì, Maggio 9, 2024
Health, Communications, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Public Relations, Investor Relations, TNNI2, Safety, Pharmacokinetics, DUNE, Duchenne, McArdle, Ageing, Exercise, Creatine kinase, Patient, MRI, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Muscular dystrophy

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.
  • The Company expects to report CANYON data in the fourth quarter of 2024.
  • Research and development (R&D) expenses were $27.7 million for both the first quarter of 2024 and for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $7.1 million for the first quarter of 2024, compared to $6.2 million for the immediately preceding quarter.

Gene Therapy Pioneer Jerry R. Mendell, M.D., Named to Inaugural TIME100 Health List of the 100 Most Influential People in Global Health

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Venerdì, Maggio 3, 2024
Health, Neurology, Genetics, Research, Science, Pharmaceutical, Biotechnology, Neuromuscular disease, SRPT, National academy, DSM-IV codes, Hospital, Muscular dystrophy, Book, SMA, Cell, Duchenne, Disease, TIME, Patient, Sarepta Therapeutics, Gene, Society, Nationwide, Duchenne muscular dystrophy, Head, Therapy, Global health, Pharmaceutical industry

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that TIME named renown neuromuscular researcher, physician and gene therapy pioneer Jerry R. Mendell, M.D., to the inaugural 2024 TIME100 Health, a list of 100 individuals who most influenced global health this year.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that TIME named renown neuromuscular researcher, physician and gene therapy pioneer Jerry R. Mendell, M.D., to the inaugural 2024 TIME100 Health, a list of 100 individuals who most influenced global health this year.
  • The TIME100 Health list recognizes the impact, innovation, and achievement of the world’s most influential individuals in health.
  • In addition to recognition on the TIME100 Health list, Dr. Mendell is a 2024 recipient of the King Faisal Prize Laureate in Medicine.
  • He is also a published author of more than 400 peer-reviewed articles and books about skeletal muscle disease, peripheral nerve disorders and gene therapy.

Somite Therapeutics to Present at the 27th Annual Meeting of the American Society of Cell and Gene Therapy (ASGCT)

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Mercoledì, Maggio 1, 2024
Doctor of Philosophy, AI, Cell, Poster, Diabetes, MD, Computational biology, B cell, PSC, Differentiable function, Obesity, SVP, Muscular dystrophy, Developmental biology, Gene, Society, MUSCULAR, Cartilage, Therapy, Translation, MIT, Pharmaceutical industry

BOSTON, May 1, 2024 /PRNewswire/ -- Somite Therapeutics, a tech-bio company leveraging big data and AI to introduce novel cell replacement therapies, today announced that the Company will present a poster at the upcoming American Society of Cell and Gene Therapy (ASGCT) Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.

Key Points: 
  • [[To comply with academic institution guidelines, the founders' academic affiliations and roles are listed only at the end of the statement.]]
  • BOSTON, May 1, 2024 /PRNewswire/ -- Somite Therapeutics , a tech-bio company leveraging big data and AI to introduce novel cell replacement therapies, today announced that the Company will present a poster at the upcoming American Society of Cell and Gene Therapy (ASGCT) Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.
  • Founded in October 2023, Somite is building AI foundation models to produce human tissue at scale for cell therapies.
  • These therapies have the potential to treat a wide range of diseases that involve the loss or deficiency of cell populations, such as diabetes, obesity, and muscular dystrophies.

Muscular Dystrophy Association Launches ‘Access The Vote’ Campaign to Amplify Rights of Accessible Voting

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Martedì, Aprile 23, 2024
Public policy, Neuromuscular disease, Social media, Muscular dystrophy, Disability, MDA, Muscular Dystrophy Association, Human, Life, Policy, Federation, Exercise, League, Campaign, Democracy, LWV

Through core messages focusing on the power of an individual’s vote, creating a voting plan, and overcoming barriers to vote, MDA aims to make voting more accessible and inclusive for all.

Key Points: 
  • Through core messages focusing on the power of an individual’s vote, creating a voting plan, and overcoming barriers to vote, MDA aims to make voting more accessible and inclusive for all.
  • The ‘Access The Vote’ Campaign provides people with the power to make an impact through their vote.
  • The ‘Access The Vote’ Campaign addresses frequently asked questions at MDA.org/Vote providing essential information on polling place accessibility, voting options, and voter rights for people living with disabilities.
  • As voting is a cornerstone of democracy, this event will empower attendees to harness the power of their fundamental right to vote.

Revolutionary Scientists Honored for Advancements in Gene Therapy for Neuromuscular Diseases and RNA Discoveries: King Faisal Prize Laureates in Medicine, Professor Jerry Mendell, and in Science, Professor Howard Chang, Awarded

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Lunedì, Aprile 22, 2024
Science (journal), Mortality, FDA, Pediatric Research, Protein, Chromatin, Cell, Limb–girdle muscular dystrophy, Research, American Philosophical Society, National Cancer Institute, Molecular biology, Life, Gene, Quran, Food, LGMD, SMA, RNA, DMD, Spinal muscular atrophy, Infant, Dystrophin, Nationwide, Duchenne muscular dystrophy, Ageing, National academy, Hospital, Cancer research, Literature, DNA, Therapy, Gene expression, Muscular dystrophy, Chromosome, USD, Stanford University, Cancer, Islamic studies, Muscle weakness, Disease, Vilcek Foundation, Patient, Assay, Society, Listeria monocytogenes non-coding RNA, Pharmaceutical industry

Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.

Key Points: 
  • Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.
  • Gene therapy offers a solution by addressing this genetic anomaly, allowing the body to produce dystrophin and halt muscle degeneration.
  • Ludwig Professor of Cancer Research at Stanford University, has been awarded King Faisal Prize for Science in Biology.
  • Since 1979, King Faisal Prize in its 5 different categories has awarded 295 laureates who have made distinguished contributions to different sciences and causes.

Lifeward Announces the Appointment of Mike Swinford to its Board of Directors

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Venerdì, Aprile 19, 2024
CMS, DBA, Brain damage, Muscular dystrophy, Disability, General Electric Company, GE HealthCare, Robotics, Amputation, Face, ALS, Rehabilitation, Tetraplegia, Multiple sclerosis, Cerebral palsy

Mr. Swinford held various operational and commercial roles throughout his career leading through various business cycles from start-ups to turnarounds.

Key Points: 
  • Mr. Swinford held various operational and commercial roles throughout his career leading through various business cycles from start-ups to turnarounds.
  • “As someone who is passionate about improving healthcare access and inclusion for people with disabilities, I am excited to join the Board of Directors of Lifeward,” said Mr. Swinford.
  • This is another step to increase the direct commercialization skills and experience within our Board.
  • We are delighted to welcome Mike to the Board.”

Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease

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Mercoledì, Aprile 17, 2024
Pillar, Enzyme, Friedreich's ataxia, Gene editing, CRISPR, GAA, Hope, Muscular dystrophy, Ataxia, FXN, MDA, Diabetes, FARA, Muscular Dystrophy Association, Column, RNA, Doctor of Philosophy, Research, Scoliosis, Parent, Schnucks, University of Massachusetts, Disease, Therapy, Biomedical Research, Nikolaus Friedreich, Fatigue, Movement, FA, Art, Genome, Organization, Pharmaceutical industry

This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).

Key Points: 
  • This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).
  • The grant, Paired Prime Editors to treat Friedreich’s Ataxia, involves prime editing (PE), a next-generation CRISPR gene editing tool that can precisely target the removal of the GAA expansions in the frataxin (FXN) gene.
  • “Our team of investigators is excited to bring multi-disciplinary expertise to the unique challenges of developing PE for FA.
  • This treatment method being targeted – prime editing – aims to directly address the cause of the disease, which is the GAA expansion in the FXN gene.

International Association of Fire Fighters & Affiliates Launch ‘Fill the Boot’ Fundraisers Nationwide for Muscular Dystrophy Association

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Mercoledì, Aprile 10, 2024
Online, FDA, Social media, Firefighter, Muscular dystrophy, MDA, Heart, Muscular Dystrophy Association, Partnership, Research, Growth, International Association, IAFF, Boot, ALS, Pedestrian, Pharmaceutical industry

Funds raised from IAFF’s Fill the Boot events for MDA accelerate research, advance care, and empower people living with muscular dystrophy, ALS, and related neuromuscular diseases to live stronger and love longer.

Key Points: 
  • Funds raised from IAFF’s Fill the Boot events for MDA accelerate research, advance care, and empower people living with muscular dystrophy, ALS, and related neuromuscular diseases to live stronger and love longer.
  • Online donations will continue, even as fire fighters take to the streets in communities across the country with boots in hand asking pedestrians, motorists, customers, and other passersby to support MDA’s mission.
  • Since 1954, our partnership with Muscular Dystrophy Association has advanced breakthroughs in treatments and access to care for families living with neuromuscular diseases.
  • "In our journey to end neuromuscular disease, fire fighters remain steadfast allies in our mission," said Donald S. Wood, Ph.D., President and CEO of MDA, reflecting on the 70-year collaboration between Muscular Dystrophy Association and International Association of Fire Fighters.