Dystrophin

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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星期一, 五月 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.

Dyne Therapeutics to Host Virtual Investor Event to Review New Clinical Data from the ACHIEVE and DELIVER Trials Tomorrow, May 20 at 8:00 a.m. ET

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星期日, 五月 19, 2024
Patient, PMO, Facial muscles, DYN, DM1, OLE, Therapy, Safety, DMD, Dyne, Event, Duchenne muscular dystrophy, LTE, Webcast, Dystrophin, Vaccine

The company intends to issue a press release prior to the start of the event.

Key Points: 
  • The company intends to issue a press release prior to the start of the event.
  • ACHIEVE is a Phase 1/2 global clinical trial evaluating DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1) who are 18 to 49 years of age.
  • In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts.
  • In the upcoming clinical update, Dyne plans to report safety and tolerability data from all enrolled cohorts.

PepGen Reports First Quarter 2024 Financial Results and Recent Corporate Highlights

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星期二, 五月 14, 2024
CTA, Patient, Health care, Research and development, SAD, PGN, Food, MBNL1, Treatment, Clinical trial, Episcopal conference, News, DM1, Medication, IND, Safety, Male, Dystrophin, Conference, DMD, Duchenne, Efficacy, Event, MHRA, RNA, Development, Duchenne muscular dystrophy, Research, Administration, MDA, Potential, Three Months, Pharmaceutical industry

BOSTON, May 14, 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 and recent corporate highlights for the quarter ended March 31, 2024.

Key Points: 
  • BOSTON, May 14, 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 and recent corporate highlights for the quarter ended March 31, 2024.
  • Phase 2 CONNECT1-EDO51 Clinical Trial of PGN-EDO51: In March 2024, PepGen announced that the 5 mg/kg PGN-EDO51 dose cohort was fully enrolled.
  • In March 2024, PepGen presented two posters on the PGN-EDO51 program at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
  • In April 2024, PepGen presented a poster on the PGN-EDODM1 program at The 14th International Myotonic Dystrophy Consortium (2024 IDMC-14) Meeting.

Revolutionary Scientists Honored for Advancements in Gene Therapy for Neuromuscular Diseases and RNA Discoveries: King Faisal Prize Laureates in Medicine, Professor Jerry Mendell, and in Science, Professor Howard Chang, Awarded

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星期一, 四月 22, 2024
Science (journal), Mortality, FDA, Pediatric Research, Protein, Chromatin, Cell, Limb–girdle muscular dystrophy, Research, American Philosophical Society, National Cancer Institute, Molecular biology, Life, Gene, Quran, Food, LGMD, SMA, RNA, DMD, Spinal muscular atrophy, Infant, Dystrophin, Nationwide, Duchenne muscular dystrophy, Ageing, National academy, Hospital, Cancer research, Literature, DNA, Therapy, Gene expression, Muscular dystrophy, Chromosome, USD, Stanford University, Cancer, Islamic studies, Muscle weakness, Disease, Vilcek Foundation, Patient, Assay, Society, Listeria monocytogenes non-coding RNA, Pharmaceutical industry

Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.

Key Points: 
  • Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.
  • Gene therapy offers a solution by addressing this genetic anomaly, allowing the body to produce dystrophin and halt muscle degeneration.
  • Ludwig Professor of Cancer Research at Stanford University, has been awarded King Faisal Prize for Science in Biology.
  • Since 1979, King Faisal Prize in its 5 different categories has awarded 295 laureates who have made distinguished contributions to different sciences and causes.

Duchenne UK and Parent Project Muscular Dystrophy Award $500,000 to Evaluate Safety and Tolerability of Muscle Progenitor Cells in Phase 1 Trial

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星期二, 四月 23, 2024
University, Cell, Hope, Safety, DMD, Research, Dystrophin, Entrepreneurship, Patient, PPMD, Translational medicine, Duchenne muscular dystrophy, Clinical trial, Therapy, Organization, Injection, CGMP, Pharmaceutical industry

The grant, totaling $500,000, will support Dr. Kang and his co-investigators in evaluating the safety and tolerability of muscle progenitor cells for Duchenne muscular dystrophy.

Key Points: 
  • The grant, totaling $500,000, will support Dr. Kang and his co-investigators in evaluating the safety and tolerability of muscle progenitor cells for Duchenne muscular dystrophy.
  • Dr. Kang's project builds on previous research, utilizing muscle progenitor cells, known as MyoPAXon, developed by Dr. Rita Perlingeiro and her team at the University of Minnesota.
  • This Phase 1 trial led by Dr. Kang's team will provide valuable insights into the safety and feasibility of transplanting MyoPAXon cells.
  • Emily Reuben, Co-Founder and CEO of Duchenne UK, explains:
    "Duchenne UK has supported Dr. Perlingeiro's work on developing muscle progenitor cells for a number of years.

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

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星期二, 四月 9, 2024
Eurostat, Civil service commission, Wasting, Diagnosis, Atrophy, Mutation, B cell, Weakness, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Death, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, Safety, European Commission, Muscle weakness, Marketing, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Key Points: 


Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

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星期二, 四月 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

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星期三, 三月 6, 2024
Therapy, Facial muscles, Pharmacology, Central nervous system, Research, GSK, MD, Liver disease, Dystrophin, Obesity, Cerebrospinal fluid, N-Acetylgalactosamine, HD, Weight loss, DMD, Duchenne muscular dystrophy, Lof, Exercise, Lipolysis, Exon, MBA, Conference, Wave, Activin and inhibin, AATD, Health, Safety, Biomarker, RNA, Greenshoe, Genetics, Pharmacokinetics, Webcast, Patient, Clinical trial, HTT, Q&A, Small RNA, Serum, Episcopal conference, Human, Coronary artery disease, Pharmaceutical industry

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

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

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星期三, 三月 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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星期二, 三月 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.