DM1

PepGen Reports Fourth Quarter and Full Year 2022 Financial Results and Recent Corporate Developments

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목요일, 3월 23, 2023
PGN, Heart, GLP, Myogenesis, Duchenne muscular dystrophy, Therapy, DM1, Muscular dystrophy, RCT, Clinical trial, SAD, American Academy, Research, Cell, CUG, Research and development, Development, CTG, DMD, Patient, EDO, Physician, Episcopal conference, Administration, DMPK, MAD, NHP, HV, American Academy of Neurology, IND, Conference, Caregiver, Disease, Pharmaceutical industry, Cryptocurrency, Neurology

In December 2022, PepGen announced IND-enabling preclinical data supporting the progression of PGN-EDODM1 for the treatment of DM1 into clinical studies.

Key Points: 
  • In December 2022, PepGen announced IND-enabling preclinical data supporting the progression of PGN-EDODM1 for the treatment of DM1 into clinical studies.
  • Net loss was $14.9 million for the three months ended December 31, 2022, compared to $7.1 million for the same period in 2021.
  • Net loss was $69.1 million for the year ended December 31, 2022, compared to $27.3 million for the same period in 2021.
  • PepGen had approximately 23.7 million shares outstanding on December 31, 2022

PepGen Presents Clinical and Nonclinical Data at the 2023 Annual Muscular Dystrophy Association Clinical and Scientific Conference

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수요일, 3월 22, 2023
PGN, Muscular Dystrophy Association, Dystrophin, Human, Myogenesis, MBNL1, Duchenne muscular dystrophy, DM1, Clinical trial, Pathology, SAD, Doctor of Philosophy, CTG, DMD, Patient, EDO, Episcopal conference, MAD, DdPCR, NHP, Research, MDA, HV, Conference, Pharmacology, Safety, Pharmaceutical industry, Vaccine

Even more encouraging was the increase in exon 51 skipping as measured by ddPCR in non-human primates (NHPs) dosed with PGN-EDO51 at 20 mg/kg.

Key Points: 
  • Even more encouraging was the increase in exon 51 skipping as measured by ddPCR in non-human primates (NHPs) dosed with PGN-EDO51 at 20 mg/kg.
  • PepGen is also presenting preclinical in vitro and in vivo pharmacology data from its DM1 program, PGN-EDODM1, in an oral presentation at MDA.
  • “Our PGN-EDODM1 preclinical data showed the potential of an investigational therapy designed to liberate MBNL1,” commented James McArthur, President and Chief Executive Officer of PepGen.
  • PepGen plans to report safety, correction of transcript mis-splicing and clinical data from the FREEDOM-DM1 trial in 2024.

Dyne Therapeutics Announces Presentations on its DM1 and DMD Programs at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference

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월요일, 3월 20, 2023
Myopathy, DYN, Muscular Dystrophy Association, Asian literature, Duchenne muscular dystrophy, Therapy, DM1, Sarcolemma, Poster, Episcopal conference, Dystrophin, MDA, Phenotype, MPH, Conference, Disease, Television, Pharmaceutical industry, Duchenne, DMD, Fibrosis, Dyne

WALTHAM, Mass., March 20, 2023 (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 three poster presentations at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 19-22, 2023, in Dallas, TX, and virtually.

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 announced three poster presentations at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 19-22, 2023, in Dallas, TX, and virtually.
  • Dyne anticipates reporting initial data from these clinical trials in the second half of 2023.
  • These data build upon Dyne’s comprehensive preclinical data and further support the company’s ongoing development in DMD.
  • The posters will also be available in the Scientific Publications & Presentations section of Dyne’s website .

Design Therapeutics Highlights Pipeline Progress and Reports Fourth Quarter and Full Year 2022 Financial Results

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화요일, 3월 14, 2023
Mutation, Therapy, FA, D, DM1, R, Design, Friedreich's ataxia, MAD, Muscle weakness, FXN, Second Half, Patient, GAA, DMPK, Research, Gene expression, Disease, G&A, SAD, DSM-IV codes, Cerebellar ataxia, Safety, Clinical trial, Investigational New Drug, Lead, IND, CTG, Pharmaceutical industry, Vaccine, Cryptocurrency

CARLSBAD, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today highlighted recent progress and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported fourth quarter and full year 2022 financial results.

Key Points: 
  • Design is advancing a Phase 1 MAD trial, with initial results expected in mid-2023, and final data expected by year-end.
  • R&D Expenses: Research and development (R&D) expenses were $14.3 million for the quarter ended December 31, 2022, and $48.6 million for the year ended December 31, 2022.
  • G&A Expenses: General and administrative (G&A) expenses were $5.1 million for the quarter ended December 31, 2022, and $19.0 million for the year ended December 31, 2022.
  • Net Loss: Net loss was $17.3 million for the quarter ended December 31, 2022, and $63.3 million for the year ended December 31, 2022.

Entrada Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results

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월요일, 3월 6, 2023
Science, Research and development, Vertex, Entrada, G&A, Therapy, Administration, Research, General Administration of Sport of China, MDA, Investigational New Drug, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, Duchenne muscular dystrophy, Food, EEV, Muscular Dystrophy Association, Conference, FDA, EST, R, Neuromuscular junction, DM1, Pharmaceutical industry, Cryptocurrency, IND, Duchenne

BOSTON, March 06, 2023 (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 reported financial results for the fourth quarter and full year ending December 31, 2022 and highlighted recent business updates.

Key Points: 
  • BOSTON, March 06, 2023 (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 reported financial results for the fourth quarter and full year ending December 31, 2022 and highlighted recent business updates.
  • Research & Development (R&D) Expenses: R&D expenses were $15.7 million for the fourth quarter of 2022 and $66.6 million for the full year of 2022, compared to $12.4 million and $35.9 million for the same periods in 2021, respectively.
  • General & Administrative (G&A) Expenses: G&A expenses were $9.9 million for the fourth quarter of 2022 and $30.6 million for the full year of 2022, compared to $6.1 million and $15.2 million for the same periods in 2021, respectively.
  • Net Loss: Net loss was $24.6 million for the fourth quarter of 2022 and $94.6 million for the full year of 2022, compared to net loss of $18.4 million and $51.2 million for the same periods of 2021, respectively.

Dyne Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Business Highlights

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목요일, 3월 2, 2023
Myopathy, Safety, Rare disease, Episcopal conference, Facial muscles, Duchenne muscular dystrophy, Conference, Patient, DYN, MAD, Research, Therapy, Pharmacokinetics, DMD, G&A, Muscular Dystrophy Association, Dystrophin, DM1, MDA, R, Biomarker, Enrollment, Pharmaceutical industry, Medical imaging, Cryptocurrency, Dyne

WALTHAM, Mass., March 02, 2023 (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 2022 and business highlights.

Key Points: 
  • WALTHAM, Mass., March 02, 2023 (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 2022 and business highlights.
  • R&D expenses were $142.8 million and $121.3 million for the years ended December 31, 2022 and 2021, respectively.
  • Net loss: Net loss for the quarter ended December 31, 2022 was $38.8 million, or $0.74 per basic and diluted share.
  • Net loss for the year ended December 31, 2022 was $168.1 million, or $3.23 per basic and diluted share.

Avidity Advances RNA Programs and Expands into New Therapeutic Areas Utilizing its AOC™ Platform

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월요일, 2월 27, 2023
Cardiology, Antibody-oligonucleotide conjugate, Eli Lilly and Company, Muscle atrophy, Glycogen storage disease type II, Duchenne muscular dystrophy, DMD, NYSE, Disease, Research, LLY, DM1, Facioscapulohumeral muscular dystrophy, Therapy, SAN, AOC, AOCS, Avidity, Lilly, FSHD, Bristol Myers Squibb, RNA, Vaccine, Pharmaceutical industry

SAN DIEGO, Feb. 27, 2023 /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 the advancement and expansion of its internal discovery pipeline with the addition of new research and development candidates to treat conditions in skeletal muscle and cardiology. The preclinical programs have been engineered using Avidity's proprietary AOC platform technology, which is designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics.

Key Points: 
  • "With the revolutionary advancement of demonstrating the first-ever successful targeted delivery of RNA into muscle, we are keen to broaden the utility of the AOC platform.
  • Avidity has decided not to pursue research and development programs in muscle atrophy and Pompe disease at this time.
  • In addition to its own internal research programs, Avidity continues to explore the full potential of its AOC platform through collaborations and partnerships.
  • Avidity also has a research collaboration with MyoKardia, a wholly-owned subsidiary of Bristol Myers Squibb, to demonstrate the potential utility of AOCs in cardiac tissue.

AMO Pharma Announces MHRA Confirmation of CDM1-RS as Approvable Primary Outcome Measure in Myotonic Dystrophy

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월요일, 2월 27, 2023
Medicine, Innovation, Therapy, University, Choking, Wales, Myotonic dystrophy, National Institute for Health and Care Excellence, Phenotype, DM, SMC, FDA, NHS, Regulation of tobacco by the U.S. Food and Drug Administration, AMO, MHRA, DM1, National Health Service, Birth defect, Muscular dystrophy, Healthcare Improvement Scotland, Professor, Caregiver, Tideglusib, Food, TDP, Health care, NICE, Pharmaceutical industry

LONDON, Feb. 27, 2023 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare childhood-onset neurogenetic disorders with limited or no treatment options, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has confirmed that the Congenital DM1 Rating Scale (CDM1-RS) can be considered an approvable primary outcome measure in clinical trials to develop a treatment for congenital myotonic dystrophy type 1 (CDM1). 

Key Points: 
  • Following advice from the U.S. Food and Drug Administration (FDA), the scale was incorporated by AMO Pharma into the REACH-CDM pivotal study in CDM1.
  • The MHRA reviewed data from AMO Pharma on how the scale was developed using information from natural history studies, consultation with therapeutic area experts and feedback from patient advocates.
  • The MHRA concluded the measure could be considered an approvable primary outcome measure in the event of a positive result in the REACH-CDM study of AMO-02 in CDM1.
  • In December 2022 AMO Pharma announced completion of patient enrollment in the company's REACH-CDM study of the investigational therapy AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy.

AMO Pharma Announces MHRA Confirmation of CDM1-RS as Approvable Primary Outcome Measure in Myotonic Dystrophy

Retrieved on: 
월요일, 2월 27, 2023
Medicine, Innovation, Therapy, University, Choking, Wales, Myotonic dystrophy, National Institute for Health and Care Excellence, Phenotype, DM, SMC, FDA, NHS, Regulation of tobacco by the U.S. Food and Drug Administration, AMO, MHRA, DM1, National Health Service, Birth defect, Muscular dystrophy, Healthcare Improvement Scotland, Professor, Caregiver, Tideglusib, Food, TDP, Health care, NICE, Pharmaceutical industry

LONDON, Feb. 27, 2023 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare childhood-onset neurogenetic disorders with limited or no treatment options, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has confirmed that the Congenital DM1 Rating Scale (CDM1-RS) can be considered an approvable primary outcome measure in clinical trials to develop a treatment for congenital myotonic dystrophy type 1 (CDM1). 

Key Points: 
  • Following advice from the U.S. Food and Drug Administration (FDA), the scale was incorporated by AMO Pharma into the REACH-CDM pivotal study in CDM1.
  • The MHRA reviewed data from AMO Pharma on how the scale was developed using information from natural history studies, consultation with therapeutic area experts and feedback from patient advocates.
  • The MHRA concluded the measure could be considered an approvable primary outcome measure in the event of a positive result in the REACH-CDM study of AMO-02 in CDM1.
  • In December 2022 AMO Pharma announced completion of patient enrollment in the company's REACH-CDM study of the investigational therapy AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy.

NeuBase Therapeutics Reports Business Update and Financial Results for the First Quarter of Fiscal Year 2023

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화요일, 2월 14, 2023
Public expenditure, Biotechnology, Gene editing, Cancer, CRISPR, Research, DM1, Therapy, KRAS, Disease, Calendar, Dietrich Stephan, Fiscal year, HD, Vaccine, Pharmaceutical industry, Cryptocurrency

“We plan on leveraging our PATrOL™ platform to perform ‘nuclease free’ in vivo gene editing to restore healthy gene function.

Key Points: 
  • “We plan on leveraging our PATrOL™ platform to perform ‘nuclease free’ in vivo gene editing to restore healthy gene function.
  • This technology complements the field of CRISPR/Cas editors, base editors, and prime editors, with the potential to address the majority of disease-causing mutations.
  • We believe the high fidelity and lack of immunogenicity of our editing approach offer the possibility to address tissue turnover by redosing.
  • Throughout calendar year 2023, we anticipate sharing data on ex vivo and in vivo editing results against high-value genetic mutations, together with associated performance metrics, such as fidelity and efficiency.