DM1

Avidity Biosciences Announces FDA Partial Clinical Hold on New Participant Enrollment in Phase 1/2 MARINA™ Trial

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화요일, 9월 27, 2022
AOCS, Therapy, Antibody-oligonucleotide conjugate, DM1, Safety, COVID-19, Twitter, FDA, Food, Biomarker, AOC, LinkedIn, Facioscapulohumeral muscular dystrophy, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PT, FSHD, Nasdaq, Science, Investigational New Drug, US, RNA, Security (finance), SEC, DMD, Webcast, International, Duchenne muscular dystrophy, Pharmaceutical industry, Avidity, IND

SAN DIEGO, Sept. 27, 2022 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on new participant enrollment in the Phase 1/2 MARINA™ clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1). Close to 40 participants are currently enrolled in the MARINA and MARINA open label extension (MARINA-OLE™) trials.

Key Points: 
  • Close to 40 participants are currently enrolled in the MARINA and MARINA open label extension (MARINA-OLE) trials.
  • The partial clinical hold is in response to a serious adverse event reported in a single participant in the 4mg/kg cohort of the MARINA study.
  • Avidity is working closely with the FDA and the trial investigator to assess the cause of this event.
  • The company is taking all necessary steps to resolve the partial clinical hold on new participant enrollment as quickly as possible.

Dyne Therapeutics Highlights DM1 and DMD Clinical Programs During “Spotlight on the Clinic” Virtual Event

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월요일, 9월 12, 2022
Adult, Therapy, Dystrophin, Investigational New Drug, University of Milan, DM1, Safety, Jude the Apostle, COVID-19, Twitter, DYN, Center, Clinic, Biomarker, Hospital, LinkedIn, Facioscapulohumeral muscular dystrophy, National Institutes of Health Clinical Center, Company, Review, Degenerative disease, U.S. Securities and Exchange Commission, Form 10-Q, Patient, Program, FSHD, Terminology, Nasdaq, MAD, Private Securities Litigation Reform Act, FORCE, Muscle, Security (finance), Pharmacokinetics, GLOBE, DMD, SEC, University, Science, Duchenne muscular dystrophy, Pharmaceutical industry, Medical imaging, Risk management, DRS, Facebook, Dyne, Neurology

WALTHAM, Mass., Sept. 12, 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 hosts a virtual event, “Spotlight on the Clinic," highlighting its clinical programs, DYNE-101 in myotonic dystrophy type 1 (DM1) and DYNE-251 in Duchenne muscular dystrophy (DMD) from 7:30-9:00 a.m. ET. Speakers include neuromuscular disease experts, Valeria Sansone, M.D., Ph.D., Clinical and Scientific Director at Clinical Center NeMO; and Professor of Neurology, University of Milan, Richard Finkel, M.D., Director of the Center for Experimental Neurotherapeutics at St. Jude Children’s Research Hospital, as well as members of Dyne’s leadership team. The live virtual event as well as the replay and slide presentation are available at https://investors.dyne-tx.com/events/event-details/dyne-spotlight-clinic....

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today hosts a virtual event, Spotlight on the Clinic," highlighting its clinical programs, DYNE-101 in myotonic dystrophy type 1 (DM1) and DYNE-251 in Duchenne muscular dystrophy (DMD) from 7:30-9:00 a.m.
  • The live virtual event as well as the replay and slide presentation are available at https://investors.dyne-tx.com/events/event-details/dyne-spotlight-clinic... .
  • With our DYNE-251 and DYNE-101 clinical trials now underway, this is a very exciting time for Dyne and people living with DMD and DM1.
  • Pipeline and Resources: Dyne is prioritizing its focus and resources on its clinical programs, DYNE-101 in DM1 and DYNE-251 in DMD.

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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수요일, 9월 7, 2022
People, Muscle weakness, DMPK, COVID-19, Fatigue, Muscle, SAN, Awareness, AOC, Science, U.S. Securities and Exchange Commission, Twitter, MDF, RNA transfection, FSHD, Patient, Global Genes, Caregiver, Cardiac muscle, Degenerative disease, Protein, SEC, Myotonic dystrophy, Nasdaq, Tears, Myotonia, Security (finance), RNA, Antibody-oligonucleotide conjugate, Disease, Avidity, Duchenne, Muscular dystrophy, LinkedIn, DUX4, Lists of diseases, DMD, Facioscapulohumeral muscular dystrophy, Private Securities Litigation Reform Act, Mortality, AOCS, Weakness, Life expectancy, Tissue, IND World's Fair Line, Learning, Duchenne muscular dystrophy, Hypersomnia, Conference, DM1, Therapy, Face, Vaccine, Pharmaceutical industry, Medical device, Dietary supplement

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 to Present at Morgan Stanley 20th Annual Global Healthcare Conference

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화요일, 9월 6, 2022
DYN, Degenerative disease, Therapy, Morgan, Facioscapulohumeral muscular dystrophy, LinkedIn, Duchenne muscular dystrophy, DMD, GLOBE, DM1, Nasdaq, Dyne, Twitter, Investor, FSHD, Pharmaceutical industry, Facebook

WALTHAM, Mass., Sept. 06, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc .

Key Points: 
  • 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 management is scheduled to participate in a fireside chat at the Morgan Stanley 20th Annual Global Healthcare Conferencebeing held in New York, NY on Monday, September 12, 2022 at 2:15 p.m.
  • A live webcast will be available in the Investors & Media section of Dynes website at https://investors.dyne-tx.com/news-and-events/events-and-presentations and a replay will be accessible for 90 days following the presentation.
  • For more information, please visit https://www.dyne-tx.com/ , and follow us on Twitter , LinkedIn and Facebook .

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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화요일, 9월 6, 2022
COVID-19, DYN, Muscle, Safety, Pharmacokinetics, Degenerative disease, University of Milan, PMO, U.S. Securities and Exchange Commission, GLOBE, Company, Twitter, Investor, Event, FSHD, Patient, Research institute, Cardiac muscle, Time, Private Securities Litigation Reform Act, SEC, Center, Nasdaq, National Institutes of Health Clinical Center, Terminology, MPH, Partnership, Security (finance), Dystrophin, Gene, Therapy, Disease, FORCE, Form 10-Q, Transferrin, Facioscapulohumeral muscular dystrophy, LinkedIn, DMD, Exon, Science, Q&A, Myopathy, Nationwide, Mortality, University, Diaphragm, Heart, Duchenne muscular dystrophy, DM1, MAD, Virtual event, Pharmaceutical industry, Online shopping, Neurology, Facebook, Dyne, Duchenne

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.

Dyne Therapeutics Announces Initiation of Phase 1/2 ACHIEVE Clinical Trial of DYNE-101 for the Treatment of Myotonic Dystrophy Type 1

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화요일, 9월 6, 2022
People, DMPK, COVID-19, DYN, Safety, Pharmacokinetics, Degenerative disease, University of Milan, CTG, U.S. Securities and Exchange Commission, GLOBE, Company, Twitter, Adult, Investor, RNA transfection, Event, FSHD, Patient, Cognition, Private Securities Litigation Reform Act, SEC, Muscle, Nasdaq, National Institutes of Health Clinical Center, Terminology, MPH, Partnership, Security (finance), RNA, Therapy, Disease, FORCE, Form 10-Q, Facioscapulohumeral muscular dystrophy, LinkedIn, Speech, Lists of diseases, DMD, Myotonia, Science, Q&A, ASO, University, Duchenne muscular dystrophy, University of Auckland, DM1, MAD, Clinical trial, Virtual event, Digestion, Pharmaceutical industry, Medical imaging, Online shopping, Neurology, Facebook, Dyne

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 the initiation of its ACHIEVE Phase 1/2 clinical trial evaluating DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1).

Key Points: 
  • The ACHIEVE trial is a Phase 1/2 global clinical trial evaluating DYNE-101, 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 ACHIEVE trial on safety, tolerability and splicing in the second half of 2023.
  • Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases.
  • Dyne has a broad portfolio of programs for serious muscle diseases, including candidates for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).

Avidity Biosciences to Participate in Upcoming Investor Conferences

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목요일, 9월 1, 2022
AOCS, RNA, SAN, Antibody-oligonucleotide conjugate, AOC, LinkedIn, Science, DMD, Conference, DM1, Nasdaq, Therapy, Twitter, FSHD, Patient, Vaccine, Avidity

Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs).

Key Points: 
  • Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs).
  • Avidity is on track to have three programs in clinical development by the end of 2022.
  • Avidity anticipates both programs will enter the clinic by the end of 2022.
  • Avidity is headquartered in SanDiego,CA.For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter .

Entrada Therapeutics to Present at H.C. Wainwright 24th Annual Global Investment Conference

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화요일, 8월 30, 2022
Central nervous system, EEV, Therapy, Therapeutic index, Tissue, Entrada, LinkedIn, Investor relations, Duchenne muscular dystrophy, DMD, Degenerative disease, GLOBE, Conference, DM1, Twitter, Patient, Fine chemical

BOSTON, Aug. 30, 2022 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV) therapeutics as a new class of medicines, today announced that Nathan Dowden, Chief Operating Officer, will present virtually at the H.C. Wainwright 24th Annual Global Investment Conference taking place September 12 14, 2022.

Key Points: 
  • BOSTON, Aug. 30, 2022 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV) therapeutics as a new class of medicines, today announced that Nathan Dowden, Chief Operating Officer, will present virtually at the H.C. Wainwright 24th Annual Global Investment Conference taking place September 12 14, 2022.
  • A replay will be available on the Entrada website for 90 days following the event.
  • The Companys EEV therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues with an improved therapeutic index.
  • For more information about Entrada, please visit our website, www.entradatx.com , and follow us on Twitter and LinkedIn .

Dyne Therapeutics Appoints Francesco Bibbiani, M.D., as Senior Vice President, Head of Development

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월요일, 8월 29, 2022
Myopathy, Patient, COVID-19, MPH, University, DYN, Security (finance), Degenerative disease, Therapy, Experiment, FORCE, Form 10-Q, Duchenne, Facioscapulohumeral muscular dystrophy, Private Securities Litigation Reform Act, LinkedIn, SEC, Duchenne muscular dystrophy, Neurology, National, U.S. Securities and Exchange Commission, DMD, FSHD, GLOBE, PTC Therapeutics, Nasdaq, DM1, Eisai, Twitter, Terminology, Industry, Drug development, New Drug Application, Pharmaceutical industry, Dyne, Facebook, Ultragenyx

WALTHAM, Mass., Aug. 29, 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 the appointment of Francesco Bibbiani, M.D., as senior vice president, head of development. Dr. Bibbiani brings more than two decades of experience across development, including a focus on rare neuromuscular diseases as well as Duchenne muscular dystrophy (DMD).

Key Points: 
  • WALTHAM, Mass., Aug. 29, 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 the appointment of Francesco Bibbiani, M.D., as senior vice president, head of development.
  • Previously, Dr. Bibbiani was the vice president of clinical development for PTC Therapeutics, Inc., overseeing multiple clinical trials in DMD and lifecycle management for all products in its neuromuscular portfolio.
  • Prior to PTC Therapeutics he held several roles at Eisai, Inc. as a project and clinical lead, overseeing global clinical development plans and leading multiple New Drug Applications.
  • Having spent many years deeply involved in rare muscle disease drug development, I know first hand the urgent need for new therapeutics.

PepGen Reports Second Quarter 2022 Financial Results and Recent Corporate Developments

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금요일, 8월 12, 2022
FDA, Neuromuscular Disorders, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, CEO, Annual conferences, GLOBE, Duchenne muscular dystrophy, Form, NHP, Private Securities Litigation Reform Act, SEC, Clinical trial, IND, Investigational New Drug, TREAT-NMD, EDO, Caregiver, PGN, Research, Achievement, Home After Three Months Away, Marketing, Patient, Male, Research and development, Program, Therapy, Safety, Form 10-Q, Myotonic dystrophy, DM1, HNV, DMD, Conference, Development, IPO, Administration, Food, Peptide, Adult, Disease, Pharmaceutical industry, Online gambling

BOSTON, Aug. 12, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (PepGen), 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 for the second quarter ended June 30, 2022.

Key Points: 
  • BOSTON, Aug. 12, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (PepGen), 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 for the second quarter ended June 30, 2022.
  • In May, PepGen completed an initial public offering, raising $122.9 million in gross proceeds before the deduction of underwriting and offering expenses.
  • PGN-EDO53: PepGen anticipates reporting NHP exon skipping data in the second half of 2022 for the Companys second DMD program for the treatment of DMD patients who are amenable to an exon 53 skipping approach.
  • Financial Results for the Three Months Ended June 30, 2022
    Cash and cash equivalents were $218.8 million as of June 30, 2022, which includes the proceeds from the Companys IPO in May 2022.