Cardiac muscle

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

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Lunedì, Agosto 8, 2022
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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.

Edgewise Therapeutics Reports Second Quarter 2022 Financial Results

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Giovedì, Agosto 4, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, Neuromuscular Disorders, HCM, U.S. Securities and Exchange Commission, ARCH, Cardiac muscle, R, Clinical trial, Security (finance), Research, Marketing, Patient, Securities Act of 1933, Dystrophinopathy, MRI, Securities Exchange Act of 1934, Food, Drug Quality and Security Act, HIV disease progression rates, Industry, Half-life, Safety, CK, Conference, Myopathy, Somnolence, Pharmacokinetics, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Ageing, Drug discovery, Disease, Growth, Therapy, COVID-19, PK, BMD, DMD, Fast Track, Biomarker, Dizziness, PD, Muscular dystrophy, Plasma, Company, Twitter, NASDAQ, Hypertrophic cardiomyopathy, TNNI2, Creatine kinase, Protein, LinkedIn, Administration, Adult, Pharmaceutical industry, Dietary supplement, Video game, Edgewise, Facebook, Biology, IND

Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today reported financial results for the second quarter of 2022 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today reported financial results for the second quarter of 2022 and recent business highlights.
  • Research and development (R&D) expenses were $12.4 million for the second quarter 2022, compared to $11.1 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $4.1 million for the second quarter 2022, compared to $3.7 million for the immediately preceding quarter.
  • Net loss and net loss per share for the second quarter of 2022 was $16.1 million or $0.32 per share, compared to $14.7 million or $0.30 per share for the immediately preceding quarter.

Voyager Therapeutics Appoints Catherine J. Mackey, Ph.D. to its Board of Directors

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Giovedì, Luglio 28, 2022
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CAMBRIDGE, Mass., July 28, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today announced the appointment of Catherine J. Mackey, Ph.D. to its Board of Directors.

Key Points: 
  • CAMBRIDGE, Mass., July 28, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today announced the appointment of Catherine J. Mackey, Ph.D. to its Board of Directors.
  • I am delighted to welcome Catherine to our Board at this pivotal time in Voyagers evolution, said Al Sandrock, M.D., Ph.D., chief executive officer of Voyager.
  • Dr. Mackey currently serves as a board member for Cour Pharmaceuticals, Avid Bioservices, and IDEAYA Biosciences.
  • For example, all statements Voyager makes regarding the timing of Dr. Mackeys election to Voyagers Board of Directors; the suitability ofDr.

Centers for Medicare & Medicaid Services (CMS) Issue New ICD-10-PCS Code for ZOLL Medical's TherOx SSO2 Therapy

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Giovedì, Luglio 14, 2022
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ZOLL Medical Corporation, an Asahi Kasei company that manufactures medical devices and related software solutions, announced today that the Centers for Medicare & Medicaid Services (CMS) ICD-10 Coordination and Maintenance Committee has issued a new ICD-10-PCS code for TherOx SuperSaturated Oxygen (SSO2) Therapy.

Key Points: 
  • ZOLL Medical Corporation, an Asahi Kasei company that manufactures medical devices and related software solutions, announced today that the Centers for Medicare & Medicaid Services (CMS) ICD-10 Coordination and Maintenance Committee has issued a new ICD-10-PCS code for TherOx SuperSaturated Oxygen (SSO2) Therapy.
  • The new code (5A0222C), for cardiac intra-arterial use, is effective for hospital discharges beginning October 1, 2022.
  • The code includes treatment of patients with left anterior descending ST-elevation myocardial infarction (LAD STEMI) heart attacks, which have higher mortality rates than other types of heart attacks.
  • TherOx and ZOLL are trademarks or registered trademarks of ZOLL Medical Corporation in the United States and/or other countries.

Dyne Therapeutics Announces FDA Clearance of IND Application for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy

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Martedì, Luglio 5, 2022
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WALTHAM, Mass., July 05, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and cleared its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. The Company expects to begin dosing patients in a Phase 1/2 clinical trial evaluating DYNE-251 in mid-2022.

Key Points: 
  • The clearance of our first IND is an important achievement for Dyne, and we appreciate the partnership with the FDA throughout this process.
  • We believe we are well-positioned to deliver on our commitment of initiating dosing in both of our DMD and DM1 programs in mid-2022.
  • Dyne plans to evaluate DYNE-251 in a global, randomized, placebo controlled, multiple ascending dose (MAD) clinical trial with a long-term extension study.
  • DYNE-251 is Dynes product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Impulse Dynamics Announces First International Implants of the Optimizer Smart Mini System in Italy

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Martedì, Giugno 14, 2022
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Dr. Stefano Guarracini, Head of Cardiology at the Synergo Casa di Cura Pierangeli Hospital in Pescara, Italy, described his experience with CCM therapy and his outlook for the Optimizer Smart Mini system after the first implant.

Key Points: 
  • Dr. Stefano Guarracini, Head of Cardiology at the Synergo Casa di Cura Pierangeli Hospital in Pescara, Italy, described his experience with CCM therapy and his outlook for the Optimizer Smart Mini system after the first implant.
  • Starting in 2019, we had the opportunity to implant Optimizer devices in several patients with heart failure.
  • We believe in the technology and consider CCM therapy to be the only option for many patients suffering from heart failure.
  • We believe that the launch of the Optimizer Smart Mini system is an example of the importance of a global infrastructure to support ongoing technology innovation and access.

Voyager Therapeutics’ TRACER™ Capsids Demonstrate Enhanced CNS Transduction Across Species; New Preclinical Results Bolster GBA1, Tau, SOD1 ALS Gene Therapy Programs

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Giovedì, Maggio 19, 2022
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CAMBRIDGE, Mass., May 19, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today is scheduled to present new preclinical data on a family of AAV9-derived TRACER capsids demonstrating cross-species central nervous system (CNS) transduction. The Company is also scheduled to report updated results from preclinical gene therapy programs in GBA1, tauopathies, and SOD1 ALS. These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting in Washington, D.C.

Key Points: 
  • The Company is also scheduled to report updated results from preclinical gene therapy programs in GBA1, tauopathies, and SOD1 ALS.
  • While other TRACER capsids have shown strong neuronal delivery, preclinical data in this study also demonstrated that VCAP-102 and other TRACER capsids showed preferential tropism for glial cells in mice.
  • Furthermore, the glial tropism observed in this AAV9-derived capsid family may allow us to address CNS indications that would benefit from non-neuronal cell transduction.
  • Voyager is scheduled to present preclinical data demonstrating improvements in physiologic parameters across pipeline programs involving CNS targets of interest: GBA1, tauopathies, and SOD1 ALS.

Dyne Therapeutics Presents New In Vivo Data from DYNE-101 at ASGCT Annual Meeting Demonstrating Low Monthly Dosing Leads to Robust DMPK RNA Knockdown

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Lunedì, Maggio 16, 2022
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WALTHAM, Mass., May 16, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is presenting new in vivo data from DYNE-101, its myotonic dystrophy type 1 (DM1) candidate, at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting today demonstrating robust knockdown of DMPK RNA in multiple muscles with low monthly dosing.

Key Points: 
  • These findings combined with previous data demonstrating correction of splicing, support the potential for DYNE-101 to be a disease-modifying therapy with low, infrequent dosing.
  • We look forward to evaluating DYNE-101 in our planned global multiple ascending dose clinical trial in patients with DM1.
  • As a result of this altered splicing, people living with DM1 typically experience progressive weakness of skeletal, cardiac and smooth muscle.
  • The new data being presented during ASGCT evaluated monthly repeat doses of DYNE-101 in an innovative hTfR1/DMSXL mouse model developed by Dyne and in non-human primates (NHPs).

Voyager Therapeutics to Present Preclinical Data from its Vectorized anti-HER2 Antibody Program and a Novel AAV5-Derived TRACER™ Capsid at the 25th American Society of Gene and Cell Therapy Annual Meeting

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Lunedì, Maggio 16, 2022
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CAMBRIDGE, Mass., May 16, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today is scheduled to present findings demonstrating preclinical proof-of-concept for its vectorized anti-HER2 antibody program and cross-species translatability for its novel AAV5-derived capsid at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

Key Points: 
  • Delivery of an anti-HER2 antibody vectorized with the TRACER capsid VCAP-102 attenuated HER2+ metastatic brain tumors in three independent mouse models.
  • The vectorized anti-HER2 antibody gene therapy conferred a survival benefit compared to a control antibody, with a median survival of 129 days for mice receiving the antibody versus 94 days for control mice.
  • Vectorized HER2-directed antibodies penetrated brain tumors and elicited an innate immune response in mouse models.
  • Additional results are scheduled to be presented today at the ASGCT Annual Meeting at 5:15 p.m.

Voyager Therapeutics Reports First Quarter 2022 Financial and Operating Results

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Mercoledì, Maggio 4, 2022
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Collaboration Revenues: Voyager had collaboration revenue of $0.7 million for the first quarter of 2022, compared to $6.5 million for the same period in 2021.

Key Points: 
  • Collaboration Revenues: Voyager had collaboration revenue of $0.7 million for the first quarter of 2022, compared to $6.5 million for the same period in 2021.
  • Voyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the modality to treat devastating diseases.
  • voyagertherapeutics.com LinkedIn Twitter
    Voyager Therapeutics is a registered trademark, and TRACER is a trademark, ofVoyager Therapeutics, Inc.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.