DM1

Dyne Therapeutics Reports First Quarter 2024 Financial Results and Recent Business Highlights

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목요일, 5월 2, 2024
Sale, American Academy, Episcopal conference, MDA, Research, Muscular Dystrophy Association, DMD, Conference, Therapy, DYN, DM1, G&A, Pharmaceutical industry

WALTHAM, Mass., May 02, 2024 (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 reported financial results for the first quarter of 2024 and recent business highlights.

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 reported financial results for the first quarter of 2024 and recent business highlights.
  • Research and development (R&D) expenses: R&D expenses were $44.5 million for the quarter ended March 31, 2024, compared to $37.5 million for the quarter ended March 31, 2023.
  • General and administrative (G&A) expenses: G&A expenses were $24.6 million for the quarter ended March 31, 2024, compared to $7.9 million for the quarter ended March 31, 2023.
  • Net loss: Net loss for the quarter ended March 31, 2024 was $65.6 million, or $0.81 per basic and diluted share.

Dyne Therapeutics Announces CEO Transition

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월요일, 3월 25, 2024
Arizona State University, California State University, Cell biology, Research, University, DM1, Dynamos, Biology, MBA, Biogen, Bioverativ, Therapy, DYN, DMD, Management

WALTHAM, Mass., March 25, 2024 (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 John Cox as president, chief executive officer (CEO) and a member of the Board of Directors, effective immediately. He succeeds Joshua Brumm, who has chosen to step down from these roles to pursue a career in healthcare investing. Mr. Brumm will serve as an advisor to Dyne to help ensure a seamless transition.

Key Points: 
  • - John Cox, Industry Leader with Global Experience in Rare Disease Commercialization, Joins Dyne as President and CEO -
    WALTHAM, Mass., March 25, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc .
  • Mr. Brumm will serve as an advisor to Dyne to help ensure a seamless transition.
  • Having worked extensively in rare diseases, I appreciate the meaningful impact that novel therapeutics can have on a patient community,” said Mr. Cox.
  • Most recently, he served as CEO of Repertoire Immune Medicines and its predecessor from 2019 until 2022.

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

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화요일, 3월 19, 2024
Research, Observational study, Cerebellar ataxia, Biotechnology, Gene expression, FXN, Disease, Webcast, Patient, G&A, DM1, Design, IND, Treatment, Clinical trial, Investigational New Drug, Therapy, Completeness, Evaluation, DMPK, FA, HD, Food, FDA, GLP, HTT, Pharmaceutical industry

R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $4.1 million for the quarter ended December 31, 2023, and $21.1 million for the year ended December 31, 2023.
  • Net Loss: Net loss was $11.8 million for the quarter ended December 31, 2023, and $66.9 million for the year ended December 31, 2023.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $281.8 million as of December 31, 2023.

Entrada Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results

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수요일, 3월 13, 2024
Administration, Patient, Regulatory affairs, Research, G&A, EMA, Doctor of Philosophy, DM1, Completeness, Health Canada, Senior, Net operating loss, IND, MHRA, Research and development, Duchenne muscular dystrophy, Clinical trial, Data management, Therapy, Vertex Pharmaceuticals, FDA, DMD, Medication, Pharmaceutical industry

BOSTON, March 13, 2024 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. The Company today reported financial results for the fourth quarter and full year ending December 31, 2023, and highlighted recent business updates.

Key Points: 
  • The Company today reported financial results for the fourth quarter and full year ending December 31, 2023, and highlighted recent business updates.
  • “We continue to make significant progress advancing our growing pipeline of intracellular therapeutics,” said Dipal Doshi, Chief Executive Officer of Entrada Therapeutics.
  • Collaboration Revenue: Collaboration revenue was $41.8 million for the fourth quarter of 2023 and $129.0 million for the full year of 2023.
  • The G&A expenses in the fourth quarter of 2023 were lower than the fourth quarter of 2022 primarily due to a decrease in legal and consulting fees.

PepGen Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Corporate Developments

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수요일, 3월 6, 2024
CONNECT, SAD, Research, Dystrophin, Clinical trial, Food, DMD, Common, DM1, Safety, Health care, Patient, MHRA, Disease, Three Months, Male, IND, Enrollment, PGN, U.K, MBNL1, CTA, Medication, Pharmaceutical industry

BOSTON, March 06, 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 for the fourth quarter and full year ended December 31, 2023, and highlighted recent corporate developments.

Key Points: 
  • “With important data readouts expected from our two lead programs, 2024 has the potential to be a transformational year for PepGen.
  • People with myotonic dystrophy type 1 (DM1) currently have no approved treatment options that target the root cause of the disease.
  • Financial Results for the Three Months and Twelve Months ended December 31, 2023
    Cash and cash equivalents were $110.4 million as of December 31, 2023.
  • Net loss was $78.6 million for the year ended December 31, 2023, compared to $69.1 million for the same period in 2022.

Dyne Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

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화요일, 3월 5, 2024
Therapy, Myotonia, Central nervous system, Research, DYN, Dystrophin, Safety, DMPK, DMD, Exercise, Standard of care, Conference, DM1, Eteplirsen, Muscular Dystrophy Association, Fatigue, Myotonic dystrophy, Patient, Clinical trial, PMO, Male, G&A, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., March 05, 2024 (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 reported financial results for the fourth quarter and full year 2023 and recent business highlights.

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 reported financial results for the fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1

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화요일, 2월 20, 2024
Fast Track, FDA, Food, Drug development, Communication, DM1, Life expectancy, Patient, Disease, PGN, Plasma protein binding, Pharmaceutical industry

“Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen.

Key Points: 
  • “Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen.
  • Once a drug candidate receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and regulatory review process.
  • For more information about Fast Track designation, please visit the FDA website ( www.fda.gov ).
  • (ClinicalTrials.gov identifier: NCT06204809)
    The Company previously announced that the FDA granted Orphan Drug Designation to PGN-EDODM1 in September 2023.

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

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금요일, 3월 8, 2024
Pharmaceutical, Genetics, Health, Clinical Trials, Wheelchair, Myotonia, Cataract, DMPK, Wasting, Exercise, Arrowhead, Conference, DM1, RNA, Muscle weakness, Patient, CUG, Primate, MDA, RNA interference, Episcopal conference, Vaccine

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial

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월요일, 3월 4, 2024
Therapy, Division, PD, PRO, Natural history, Doctor of Philosophy, Myotonic dystrophy, MD, Friends, Neurology, Hope, Neuromuscular medicine, Conference, DM1, Facial muscles, Safety, Muscular Dystrophy Association, Biomarker, RNA, Webcast, Patient, Elbow, Family, Disease, QMT, Pediatrics, AOC, Publication, MDA, Volume Eight, Episcopal conference, Partnership, Pharmaceutical industry

SAN DIEGO, March 4, 2024 /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 new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.

Key Points: 
  • These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1.
  • Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.
  • Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.
  • ET to discuss new positive long-term del-desiran (AOC 1001) data from the MARINA-OLE™ trial in people living with DM1.

Avidity Biosciences Honors Rare Disease Day®

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목요일, 2월 29, 2024
Rare disease, Therapy, SAN, Avidity, Caregiver, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Hope, Learning, DM1, RNA, Rare Disease Day, FSHD, Patient, Disease, AOC, Myopathy, Research

"Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.

Key Points: 
  • "Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.
  • "Recently, we had the privilege of hosting individuals affected by rare muscle diseases at Avidity.
  • Rare Disease Day takes place on the last day of February each year with the goal to raise awareness of the impact of rare diseases worldwide.
  • EURORDIS established Rare Disease Day in 2008 and coordinates with more than 70 national alliance patient organizations each year to honor those living with rare diseases as well as their families and caregivers.