Cardiac muscle

Voyager Therapeutics Announces Pfizer License of Next-Generation AAV Capsid for Rare Neurologic Disease Target

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Tuesday, October 4, 2022
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CAMBRIDGE, Mass., Oct. 04, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy and neuroscience company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today announced that Pfizer, Inc. (NYSE: PFE) has exercised its option to license a novel capsid generated from Voyager’s TRACERTM capsid discovery platform to help enable a potential gene therapy program against an undisclosed rare neurologic disease target.

Key Points: 
  • The target under agreement with Pfizer is distinct from those utilized in Voyagers internal pipeline programs.
  • Pfizer has elected not to exercise its option to license a capsid for the cardiac target under the original agreement, and all capsid rights for that target are returned to Voyager.
  • Voyager Therapeutics(Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the modality to treat devastating diseases.
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    Voyager Therapeutics is a registered trademark, and TRACER is a trademark, ofVoyager Therapeutics, Inc.

Cytokinetics Presents New Data From REDWOOD-HCM OLE in Late Breaking Clinical Trial Session at the HCM Society Scientific Sessions

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Friday, September 30, 2022
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Cytokinetics is also developing aficamten, a next-generation cardiac myosin inhibitor, for the potential treatment of hypertrophic cardiomyopathies (HCM).

Key Points: 
  • Cytokinetics is also developing aficamten, a next-generation cardiac myosin inhibitor, for the potential treatment of hypertrophic cardiomyopathies (HCM).
  • Cytokinetics expects to start a Phase 3 clinical trial of aficamten in patients with obstructive HCM in Q4 2021.
  • For further information regarding these and other risks related to Cytokinetics' business, investors should consult Cytokinetics' filings with the Securities and Exchange Commission.
  • CYTOKINETICS and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries.

 Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial for RP-A501 in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022

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Friday, September 30, 2022
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Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.

Key Points: 
  • Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for Duchenne Muscular Dystrophy Demonstrates Clinically Significant Functional and Biomarker Improvements

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Monday, September 26, 2022
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Patient #2. (15-year-old non-ambulatory with deletion of Exon 48-50).  Improved duration and amplitude of motor unit potentials (up to 287% of baseline) on EMG when compared to pretreatment baseline. Improved grip strength (8kg-->9.7kg); improved PUL (20-->23). Statistically and clinically significant increase in activity via activity tracker.

Key Points: 
  • Results include 6-month safety, functional, and biomarker data from three clinical trial participants in the first, low-dose cohort.
  • All patients, independent of their genetic mutation, demonstrated both clinical and biomarker improvements when compared to baseline.
  • Dystrogen Therapeutics is a clinical-stage life sciences company committed to developing therapies for rare genetic diseases and disorders associated with aging.
  • Dystrogen has developed a patented cell engineering technology platform, which has been shown to improve function in both clinical and pre-clinical trials.

NEW LONGEVITY APP TURNS BACK THE CLOCK-- REBOOT YOUR AGE LETS YOU DO WHAT YOU LOVE FOR LONGER

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Tuesday, September 13, 2022
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CLEVELAND, Sept. 13, 2022 /PRNewswire/ -- Great Age Reboot, Inc., a new digital health company, today announced the launch of its doctor-approved lifestyle program and app, Reboot Your Age. The new app informs and inspires users to build healthy habits that make their physiological age younger than their calendar age. Led by founder Michael Roizen, M.D., Chief Wellness Officer Emeritus at the Cleveland Clinic and creator of the best-selling RealAge books and concept, the Great Age Reboot team has analyzed more than 50,000 scientific studies, and counting, to generate the guidance and algorithms that power the Reboot Your Age program. The app guides users through their own personalized Reboot journey using intriguing content (such as short videos and articles), compelling gamification features (including brain games, leaderboards, and quizzes), and fun activities that promote healthy habit-building.

Key Points: 
  • , a new digital health company, today announced the launch of its doctor-approved lifestyle program and app, Reboot Your Age.
  • The new app informs and inspires users to build healthy habits that make their physiological age younger than their calendar age.
  • "The underlying Reboot Platform that powers the Reboot Your Age app is built on extensive and rigorous scientific data.
  • Along with the Reboot Your Age app, users can reference Dr. Roizen's new book, "The Great Age Reboot: Cracking the Longevity Code for a Younger Tomorrow."

Avidity Biosciences Engages with Patient Communities During National Muscular Dystrophy Awareness Month as part of Commitment to Developing Muscular Dystrophy Programs

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Wednesday, September 7, 2022
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SAN DIEGO, Sept. 7, 2022 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), honors National Muscular Dystrophy Awareness Month, an annual observance that raises awareness for families across the nation who are living with neuromuscular diseases. This month Avidity will also raise awareness and support patient-focused activities for World Duchenne Awareness Day on September 7 and International Myotonic Dystrophy Awareness Day on September 15. Avidity is on track to have three rare muscular dystrophy programs in the clinic by the end of this year for myotonic dystrophy type 1 (DM1), Facioscapulohumeral muscular dystrophy (FSHD) and Duchenne muscular dystrophy (DMD).

Key Points: 
  • This month Avidity will also raise awareness and support patient-focused activities for World Duchenne Awareness Day on September 7 and International Myotonic Dystrophy Awareness Day on September 15.
  • Avidity is on track to have three rare muscular dystrophy programs in the clinic by the end of this yearfor myotonic dystrophy type 1 (DM1), Facioscapulohumeral muscular dystrophy (FSHD) and Duchenne muscular dystrophy (DMD).
  • "We look forward to engaging with patient communities and their families this month to better understand their unique journeys and experiences.
  • We are on track to have three rare muscular dystrophy programs in the clinic by the end of this year."

Dyne Therapeutics Announces First Patient Dosed in Phase 1/2 DELIVER Clinical Trial of DYNE-251 for the Treatment of Duchenne Muscular Dystrophy

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Tuesday, September 6, 2022
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WALTHAM, Mass., Sept. 06, 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 first patient has been dosed in its Phase 1/2 clinical trial, DELIVER, evaluating DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping.

Key Points: 
  • We are excited to bring our FORCE platform to the clinic for the first time with the initiation of patient dosing in the DELIVER trial.
  • This is a significant milestone for Dyne and our efforts to build a global franchise of DMD exon skipping therapies.
  • The DELIVER trial is a Phase 1/2 global clinical trial evaluating DYNE-251, consisting of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension.
  • Dyne anticipates reporting data from the MAD placebo-controlled portion of the DELIVER trial on safety, tolerability and dystrophin in the second half of 2023.

Maze Therapeutics Announces FDA Orphan Drug Designation Granted to MZE001 for the Treatment of Pompe Disease

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Wednesday, August 31, 2022
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Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MZE001, the companys investigational product for the treatment of Pompe disease.

Key Points: 
  • Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MZE001, the companys investigational product for the treatment of Pompe disease.
  • MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup through substrate reduction therapy (SRT).
  • Following the Phase 1 trial readout, which is expected by the end of 2022, Maze plans to initiate a Phase 2 trial in Pompe disease patients in the first half of 2023.
  • We are pleased to have received this designation from the FDA for MZE001, which underscores the need for new, innovative treatments for Pompe disease.

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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Monday, August 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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Thursday, August 4, 2022
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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.