Facioscapulohumeral muscular dystrophy

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

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вторник, мая 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.

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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понедельник, мая 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.

Solid Biosciences Provides First Quarter 2024 Business Update and Financial Results

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среда, мая 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.

Satellos Bioscience Announces Q1 2024 Financial Results and Operational Highlights

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вторник, мая 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 .

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

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четверг, мая 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.

Epic Bio Presents New Data Highlighting Potential of EPI-321 for FSHD and Epigenetic Editing Platform Leadership at ASGCT Annual Meeting

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вторник, апреля 23, 2024
LDLR, Cas9, Facioscapulohumeral muscular dystrophy, CRISPR, Safety, RNA, Cas12a, Cell, Translation, Patient, Gene, FSHD, Society, LDL, Genome, VPR, Annual general meeting, SAN, Vaccine

Across three oral presentations and three poster presentations, Epic Bio will present new data demonstrating the differentiation of the company’s epigenetic editing platform and the promise of lead candidate EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • Across three oral presentations and three poster presentations, Epic Bio will present new data demonstrating the differentiation of the company’s epigenetic editing platform and the promise of lead candidate EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
  • A second study evaluated the use of proprietary epigenetic activators to boost transcription of the LDL Receptor (LDLR) gene.
  • As a leader in the field of epigenetic editing, Epic Bio has done pioneering work to craft a comprehensive off-target assessment platform to ensure the safety of epigenetic editing therapies for human testing.
  • A third oral presentation at ASGCT spotlights a novel, freely available tool recently developed by Epic Bio to assist researchers who wish to use the hypercompact dCasMINI protein for epigenetic editing.

Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy

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среда, марта 20, 2024
CELU, Diagnosis, Shoulder, Biologics license application, Disease, Risk, Patient, Microsoft Exchange Server, Stem cell, IND, Facioscapulohumeral muscular dystrophy, Regenerative medicine, FDA, BLA, FSHD, Muscular dystrophy, Food, Pharmaceutical industry

FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).

Key Points: 
  • FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).
  • Orphan drug designation is a status given to drugs that show the potential to treat, prevent or diagnose serious or life-threatening diseases that affect fewer than 200,000 people in the U.S.
  • Earlier, Celularity received FDA Investigational New Drug Application clearance for PDA-002 in FSHD and plans to commence a phase 1/2 study in the second half of 2024.
  • The trial serves as an important component for submitting a Biologics License Application (BLA) to the FDA in the future as a potential treatment for FSHD and other types of muscular dystrophy.

Fulcrum Therapeutics Appoints Patrick Horn M.D., Ph.D., as Chief Medical Officer

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понедельник, марта 18, 2024
Sickle cell disease, University, Medicine, Doctor of Philosophy, Albireo, New Drug Application, Patient, SVP, Research, Losmapimod, Senior, Homocystinuria, Facioscapulohumeral muscular dystrophy, Therapy, DRS, Hereditary angioedema, Drug development, Interim, Boston, Fulcrum, PFIC, DSM-IV codes, Pharmaceutical industry

CAMBRIDGE, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced the appointment of Patrick Horn, M.D., Ph.D., as chief medical officer, effective immediately. Dr. Horn is a seasoned executive with over 20 years of end-to-end drug development experience spanning multiple therapeutic areas, with an emphasis on rare diseases, across both large pharmaceutical and biotech companies. Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development. Together, Drs. Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.

Key Points: 
  • Interim chief medical officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum’s executive leadership team as SVP of early development.
  • Horn and Fraser will be responsible for leading clinical development and overseeing regulatory strategy and execution.
  • His most recent role was as the Chief Medical Officer at HemoShear Therapeutics, specializing in rare metabolic diseases.
  • Prior to transitioning to industry, Dr. Horn was a practicing pediatrician at major academic institutions in Chicago.

Satellos Bioscience Announces 2023 Year End Financial Results and Operational Highlights

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среда, марта 27, 2024
Biotechnology, Health, Clinical Trials, Research, Conference, TSX Venture Exchange, MSCL, Regeneration, CFO, G&A, Toronto Stock Exchange, Muscular dystrophy, Doctor of Philosophy, MD, Senior, MBA, GMP, IND, Bank statement, TSX, Regenerative medicine, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Investment, Chemistry, Toxicology, Therapy, AAK1, OTCQB, FDA, GLP, Animal, DMD, FSHD, Hand, Episcopal conference, Food, CPA, Pharmaceutical industry

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.

Key Points: 
  • Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.
  • Subsequent to the year end, on March 4, 2024, Satellos announced positive preclinical data presented at the Muscular Dystrophy Association Clinical and Scientific Conference.
  • Satellos also announced that SAT-3247 would be nominated as its lead development candidate with SAT-3153 becoming the backup development candidate.
  • Satellos’ audited financial statements for the year ended December 31, 2023, and the related management’s discussion and analysis (MD&A) will be available on SEDAR+ at www.sedarplus.ca .

Springbok Analytics Named to Fast Company's 2024 List of the World's Most Innovative Companies

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вторник, марта 19, 2024
Diagnosis, Health, Editorial, Culture, Organization, Longevity, MRI, Entrepreneurship, MIC, NCAA, Patient, Premier League, Research, 3D, University, AI, Hope, Athlete, American City Business Journals, NHL, Facioscapulohumeral muscular dystrophy, MLB, Most, Cerebral palsy, CSO, Springbok, NBA, NFL, FSHD, Ageing, Medical imaging

CHARLOTTESVILLE, Va., March 19, 2024 /PRNewswire/ - Springbok Analytics ( www.springbokanalytics.com ), a life sciences muscle analytics company, has been named to Fast Company's prestigious list of the World's Most Innovative Companies in 2024.

Key Points: 
  • CHARLOTTESVILLE, Va., March 19, 2024 /PRNewswire/ - Springbok Analytics ( www.springbokanalytics.com ), a life sciences muscle analytics company, has been named to Fast Company's prestigious list of the World's Most Innovative Companies in 2024.
  • Springbok has created the only technology that comprehensively measures muscle from imaging, producing personalized 3D visualizations and analyses of muscle health.
  • "I have made it my life's work to improve our understanding of muscles and muscle health," said Silvia Blemker, CSO and Co-Founder of Springbok Analytics.
  • The World's Most Innovative Companies stands as Fast Company's hallmark franchise and one of its most anticipated editorial efforts of the year.