DM1

Dyne Therapeutics Presents New Preclinical Data for its Facioscapulohumeral Muscular Dystrophy Program During the FSHD Society International Research Congress

Retrieved on: 
Donnerstag, Juni 13, 2024
DYN, Oligonucleotide, Wasting, Transferrin, DM1, DMD, Therapy, Data, Congress, Transcriptome, FSHD, DUX4, DSM-IV codes, Facioscapulohumeral muscular dystrophy, Vaccine

WALTHAM, Mass., June 13, 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 highlighted new preclinical data for DYNE-302, its product candidate for facioscapulohumeral muscular dystrophy (FSHD), that demonstrated robust and durable DUX4 suppression and functional benefit. The data were presented during the 31st Annual FSHD Society International Research Congress, being held June 13-14, 2024, in Denver, Colorado.

Key Points: 
  • - Leveraging the FORCE™ Platform, DYNE-302 Achieved Robust and Durable DUX4 Suppression and Functional Benefit in FSHD Preclinical Models -
    WALTHAM, Mass., June 13, 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 highlighted new preclinical data for DYNE-302, its product candidate for facioscapulohumeral muscular dystrophy (FSHD), that demonstrated robust and durable DUX4 suppression and functional benefit.
  • The data were presented during the 31 st Annual FSHD Society International Research Congress , being held June 13-14, 2024, in Denver, Colorado.
  • DYNE-302 also demonstrated high in vitro potency in FSHD patient-derived myotubes.

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

Retrieved on: 
Montag, Mai 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

Retrieved on: 
Sonntag, Mai 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

Retrieved on: 
Dienstag, Mai 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.

Design Therapeutics Announces First Quarter 2024 Financial Results and Highlights Upcoming Program Milestones

Retrieved on: 
Mittwoch, Mai 8, 2024
Patient, Design, Research, DMPK, IND, Observational study, HD, GLP, FA, DM1, HTT, Therapy, Biotechnology, G&A, Pharmaceutical industry

CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.

Key Points: 
  • Starting Phase 1 Development for Fuchs Endothelial Corneal Dystrophy (FECD) in 2024; Observational Study Currently Enrolling Patients
    CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.
  • Design is on track to complete GLP studies for DT-216P2 by year-end 2024 to start patient trials in 2025.
  • R&D Expenses: Research and development (R&D) expenses were $9.8 million for the quarter ended March 31, 2024.
  • G&A Expenses: General and administrative (G&A) expenses were $4.6 million for the quarter ended March 31, 2024.

Entrada Therapeutics Reports First Quarter 2024 Financial Results

Retrieved on: 
Dienstag, Mai 7, 2024
Neuromuscular disease, CTN, DMD, Research, Administration, IND, Research and development, Patient, EEV, Duchenne muscular dystrophy, DM1, Clinical trial, CTAS, Therapy, G&A, Pharmaceutical industry

BOSTON, May 07, 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 first quarter ended March 31, 2024, and highlighted recent business updates.

Key Points: 
  • The Company today reported financial results for the first quarter ended March 31, 2024, and highlighted recent business updates.
  • “The first quarter of 2024 was a period of significant clinical progress for Entrada.
  • Entrada expects to report data from the clinical trial in October of 2024.
  • The increase was primarily a result of the clinical advancement milestone achieved in the first quarter of 2024.

Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1

Retrieved on: 
Mittwoch, Mai 8, 2024
FDA, Food, RNA, QMT, Initiation, Facial muscles, Natural history, AOC, Therapy, Myopathy, European Medicines Agency, EMA, DM1, Avidity, Breakthrough therapy, SAN, Pharmaceutical industry

SAN DIEGO, May 8, 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 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1). Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

Key Points: 
  • Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
  • "We are pleased that the FDA has granted Breakthrough Therapy designation to del-desiran for myotonic dystrophy type 1, underscoring the potential of del-desiran to be an effective treatment and the urgency of bringing this treatment to people living with DM1," said Sarah Boyce, president and chief executive officer at Avidity.
  • Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter.
  • In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1.

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

Retrieved on: 
Donnerstag, Mai 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

Retrieved on: 
Montag, März 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

Retrieved on: 
Dienstag, März 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.