Duchenne muscular dystrophy

Positive Results from Genethon’s Gene Therapy Trial for Duchenne Muscular Dystrophy, Advances of Its Spinoff Atamyo in the LGMD and Cardiomyopathy Fields, Highlighted in Latest Newsletter

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月曜日, 6月 3, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Newsletter, Interview, Mass, Data monitoring committee, Publication, Therapy, Muscular dystrophy, Duchenne muscular dystrophy, Clinical trial, Gene, Muscular Dystrophy Association, European

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.

Key Points: 
  • Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.
  • Also in an interview, Roger Hajjar, M.D., Director of the Gene and Cell Therapy Institute of Mass General Brigham in Boston, renowned gene therapy researcher and entrepreneur, offered insights on what attracted him to join Atamyo Therapeutics as the newest board member of the Genethon spinout company.
  • “What resonated with me was the fact that Atamyo is a research endeavor by investigators within Genethon whose work is being translated into the clinic,” Dr. Hajjar said.
  • It’s definitely a new model.”
    Genethon CEO Frederic Revah, Ph.D., commenting on gene therapy trials for Duchenne Muscular Dystrophy, observed, “The effective dose in this first phase of the trial is lower than that used in other gene therapy programs using microdystrophin.

Sapreme Appoints CEO, BoD Chair and SAB to Advance Development of Next-Generation RNA Therapeutics for Genetically Driven Diseases

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火曜日, 5月 28, 2024
Health, Other Health, Other Science, Research, Science, Pharmaceutical, Biotechnology, MSD, Medical laboratory, University College Dublin, Self-publishing, Whitehead Institute, Drug development, Molecular medicine, Vaccine, Genetics, RNA, Therapy, Infection, ALS, Free University, Saponin, MIT, MD, MB, Organon, Master of Science, Clinical chemistry, LUMC, Protein, Asos, Duchenne muscular dystrophy, Medicinal chemistry, Psychiatry, Neurology, Complex conjugate, EAN, SAB, Peptide, Damme, Patient, Doctor of Philosophy, Organization, Free University of Berlin, University, Schering-Plough, Leiden University, DMD, Growth, Spacecraft, Cancer, Leiden University Medical Center, Pharmaceutical industry

Sapreme , a biotechnology company focused on developing next-generation RNA therapeutics for patients with genetically driven diseases, today announced the appointment of Marco Timmers, PhD, as Chief Executive Officer and Jeanne Bolger, MD, to Chair of the Sapreme Board of Directors (BoD).

Key Points: 
  • Sapreme , a biotechnology company focused on developing next-generation RNA therapeutics for patients with genetically driven diseases, today announced the appointment of Marco Timmers, PhD, as Chief Executive Officer and Jeanne Bolger, MD, to Chair of the Sapreme Board of Directors (BoD).
  • Before joining Sapreme, he was CEO of Byondis (formerly Synthon Biopharmaceuticals), a clinical-stage biopharmaceutical company developing Antibody-Drug Conjugates (ADCs) targeting intractable cancers and auto-immune diseases.
  • During his time at Byondis, Dr. Timmers also served as Chief Scientific Officer, guiding the company’s clinical strategies and growth.
  • I look forward to working with the team at Sapreme to further advance this innovative platform and accelerate our pipeline of RNA therapeutic candidates addressing a range of genetically driven diseases,” said Marco Timmers, Chief Executive Officer of Sapreme.

Satellos Announces Formation of Clinical Advisory Board to Support Advancing SAT-3247 in Clinical Trial Development for Duchenne Muscular Dystrophy

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火曜日, 5月 28, 2024
Science, Biotechnology, Research, Pharmaceutical, Managed Care, Health, Genetics, Clinical Trials, Patient, FSHD, Hospital for Sick Children, UCLA, MSCL, Tic disorder, FDA, Drug development, FAAN, Myotonic dystrophy, DMD, CAB, CMO, Shirin David, VCU, Professor, Hospital, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, CRISPR, Therapy, TSX, OTCQB, Muscular dystrophy, Pharmaceutical industry

“The formation of this Clinical Advisory Board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” said Frank Gleeson, Co-founder and CEO of Satellos.

Key Points: 
  • “The formation of this Clinical Advisory Board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” said Frank Gleeson, Co-founder and CEO of Satellos.
  • Research in his laboratory focuses on utilizing genome editing technologies like CRISPR for the development of therapeutic approaches for neurogenetic disorders.
  • Francesco Muntoni, M.D., Director, Dubowitz Neuromuscular Centre, Great Ormond Street UCL Institute of Child Health, is focused on advancing novel therapeutics in translational and clinical research for pediatric neuromuscular disorders, especially Duchenne muscular dystrophy.
  • Dr. Shieh’s principal clinical interests include Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and myotonic dystrophy.

hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data from tRNA-based Protein Editing Platform

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金曜日, 5月 24, 2024
Duchenne muscular dystrophy, DMD, Haemophilia, Cancer, Conference, Telescopic sight

Boston, Massachusetts--(Newsfile Corp. - May 24, 2024) - hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced preclinical data supporting its program in Duchenne muscular dystrophy (DMD) at the CureDuchenne FUTURES National Conference in Orlando, Fla.

Key Points: 
  • Boston, Massachusetts--(Newsfile Corp. - May 24, 2024) - hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced preclinical data supporting its program in Duchenne muscular dystrophy (DMD) at the CureDuchenne FUTURES National Conference in Orlando, Fla.
    To view the full announcement, including downloadable images, bios, and more, click here .
  • DMD program is currently in the early lead identification stage and joins hC Bioscience's growing pipeline, which also includes programs for severe hemophilia A and cancer
    Click image above to view full announcement.

Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints

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月曜日, 5月 20, 2024
CDER, PMO, FSHD, FDA, Dyne, Therapy, Eteplirsen, Facial muscles, Duchenne muscular dystrophy, Dystrophin, MPH, Food, DYN, Clinical trial, Disease, Safety, Event, Patient, Facioscapulohumeral muscular dystrophy, DM1, Male, Update, DMD, PMOS, Biomarker, QMT, Fatigue, Central nervous system, Pharmaceutical industry

New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles.

Key Points: 
  • New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles.
  • “We are excited to report new clinical data from both our ACHIEVE and DELIVER trials demonstrating meaningful impact on key biomarkers and functional improvement in multiple clinical endpoints that matter to patients.
  • Our robust preclinical work is translating into clinical benefit along with favorable safety profiles for both DYNE-101 and DYNE-251.
  • In the ACHIEVE trial, DYNE-101 demonstrated robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in multiple functional endpoints and patient reported outcomes.

Dyne Therapeutics to Host Virtual Investor Event to Review New Clinical Data from the ACHIEVE and DELIVER Trials Tomorrow, May 20 at 8:00 a.m. ET

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日曜日, 5月 19, 2024
Patient, PMO, Facial muscles, DYN, DM1, OLE, Therapy, Safety, DMD, Dyne, Event, Duchenne muscular dystrophy, LTE, Webcast, Dystrophin, Vaccine

The company intends to issue a press release prior to the start of the event.

Key Points: 
  • The company intends to issue a press release prior to the start of the event.
  • ACHIEVE is a Phase 1/2 global clinical trial evaluating DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1) who are 18 to 49 years of age.
  • In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts.
  • In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts.

Solid Biosciences Provides First Quarter 2024 Business Update and Financial Results

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水曜日, 5月 15, 2024
Patient, TNNT2, FSHD, Investment, NHP, FDA, IND, Safety, Sale, Duchenne, Research, CMC, RNA interference, Solid, Duchenne muscular dystrophy, BLA, Facioscapulohumeral muscular dystrophy, Therapy, CPVT, Pharmaceutical industry

CHARLESTOWN, Mass., May 15, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2024, and provided a business update.

Key Points: 
  • CHARLESTOWN, Mass., May 15, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2024, and provided a business update.
  • We anticipate providing a topline readout of safety, microdystrophin expression and functional benefit by year end,” said Bo Cumbo, President and CEO, Solid Biosciences.
  • Initial safety, microdystrophin expression and functional benefit data from the first 3-4 patients enrolled in the trial is expected by the fourth quarter of 2024.
  • Net loss for the first quarter of 2024 was $24.3 million, compared to $30.1 million for the first quarter of 2023.

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights

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火曜日, 5月 14, 2024
CTA, Patient, Health care, Research and development, SAD, PGN, Food, MBNL1, Treatment, Clinical trial, Episcopal conference, News, DM1, Medication, IND, Safety, Male, Dystrophin, Conference, DMD, Duchenne, Efficacy, Event, MHRA, RNA, Development, Duchenne muscular dystrophy, Research, Administration, MDA, Potential, Three Months, Pharmaceutical industry

BOSTON, May 14, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results and recent corporate highlights for the quarter ended March 31, 2024.

Key Points: 
  • BOSTON, May 14, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results and recent corporate highlights for the quarter ended March 31, 2024.
  • Phase 2 CONNECT1-EDO51 Clinical Trial of PGN-EDO51: In March 2024, PepGen announced that the 5 mg/kg PGN-EDO51 dose cohort was fully enrolled.
  • In March 2024, PepGen presented two posters on the PGN-EDO51 program at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
  • In April 2024, PepGen presented a poster on the PGN-EDODM1 program at The 14th International Myotonic Dystrophy Consortium (2024 IDMC-14) Meeting.

hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data From tRNA-based Protein Editing Platform

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金曜日, 5月 24, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Oncology, B cell, PTC, ACE, Conference, DMD, Genome, Duchenne muscular dystrophy, Physician

In DMD, a rare, fatal, progressive neuromuscular disease caused by mutations to dystrophin that affect about 300,000 males worldwide, PTCs account for approximately 26 percent of cases.

Key Points: 
  • In DMD, a rare, fatal, progressive neuromuscular disease caused by mutations to dystrophin that affect about 300,000 males worldwide, PTCs account for approximately 26 percent of cases.
  • “We are grateful for the opportunity to share these results with the DMD community as we explore the potential of tRNA-based protein editing.
  • No other treatment, approved or investigational, restores production of full-length dystrophin, and tRNA-based protein editing achieves that goal without altering the genome,” said Leslie Williams, CEO of hC Bioscience.
  • “This rapidly emerging modality has the potential to be a new breakthrough therapeutic for about one in four people affected by DMD.

Satellos Bioscience Announces Q1 2024 Financial Results and Operational Highlights

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火曜日, 5月 14, 2024
Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Patient, PPMD, Human, FSHD, Food, Investment, MSCL, Muscular Dystrophy Association, Episcopal conference, FDA, Trial of the century, Travel, Chemistry, Bank statement, Safety, Conference, DMD, Phase 1, MD, Animal, GLP, Duchenne muscular dystrophy, Tablet, Volunteering, Facioscapulohumeral muscular dystrophy, Toxicology, G&A, TSX, OTCQB, MDA, GMP, Pharmaceutical industry

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today its financial results and operational highlights for the three months ended March 31, 2024.

Key Points: 
  • Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today its financial results and operational highlights for the three months ended March 31, 2024.
  • On March 4, 2024, Satellos announced positive preclinical data presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
  • Satellos had cash and cash equivalents and short-term investments of $33.2 million as of March 31, 2024, compared with $39.6 million at December 31, 2023.
  • Satellos’ condensed consolidated interim financial statements for the three months ended March 31, 2024, and the related management’s discussion and analysis (MD&A) will be available on SEDAR+ at www.sedarplus.ca .