DM1

PepGen Inc. Provides Update on Planned Initiation of Phase 1 Study of PGN-EDODM1 in Myotonic Dystrophy Type 1

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화요일, 6월 13, 2023
PGN, Duchenne muscular dystrophy, DM1, Patient, FDA, Letter, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Disease, Geography, Pharmaceutical industry

“After careful consideration, we decided to pause additional regulatory filings for clearance to initiate a Phase 1 study of PGN-EDODM1 in order to address the official hold letter once received from the FDA,” said James McArthur, Ph.D., President and CEO of PepGen.

Key Points: 
  • “After careful consideration, we decided to pause additional regulatory filings for clearance to initiate a Phase 1 study of PGN-EDODM1 in order to address the official hold letter once received from the FDA,” said James McArthur, Ph.D., President and CEO of PepGen.
  • “People living with DM1 are eager for innovative, potential new treatment options for this serious disease, and transparency with that community and PepGen’s shareholders is always a top priority for our team.
  • We will continue to work closely with the FDA to lift the clinical hold whilst we remain fully committed to initiating a Phase 1 study of PGN-EDODM1 as quickly as feasible.
  • We also remain very focused on advancing our Phase 2 CONNECT1-EDO51 study in our lead program in Duchenne muscular dystrophy, which was cleared to proceed last month by Health Canada.”

Kate Therapeutics Debuts With $51 Million Series A to Develop Next-Generation Genetic Medicines to Treat Muscle and Heart Diseases

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목요일, 6월 8, 2023
Tissue, Heart, Versant, Capsid, Broad Institute, University, DM1, Pardis Sabeti, Cardiac muscle, Research, MIT, FSHD, RNA, Harvard University, AAV, Facioscapulohumeral muscular dystrophy, Patient, Machine learning, SAN, Cellular Dynamics International, Therapy, X-linked myotubular myopathy, Safety, Pharmaceutical industry, Vaccine, Cell

SAN DIEGO, June 8, 2023 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, today emerged from stealth mode with a $51 million Series A financing co-led by founding investor Westlake Village BioPartners and Versant Ventures, with participation from Osage University Partners and UF Innovate | Ventures. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM), the details of which are described in a separate press release issued today.

Key Points: 
  • "We are excited to announce KateTx's launch and what this means for patients suffering from muscle and heart diseases," said Kevin Forrest, Ph.D., president, CEO and a director of KateTx.
  • "KateTx is applying novel capsid and cargo technology platforms to enable skeletal and cardiac muscle targeting and liver de-targeting.
  • KateTx's current focus is identifying and advancing clinical candidates for DM1 and FSHD, as well as for other genetic muscle and heart diseases.
  • "With this Series A financing and licensing agreement, KateTx will be able to progress its deep pipeline of internal programs."

PepGen Inc. Announces Clinical Hold in the U.S. on IND Application to Initiate a Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)

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화요일, 5월 30, 2023
PGN, Duchenne muscular dystrophy, NOL, DM1, New Drug Application, Clinical trial, CTA, BioCompute Object, DMD, Patient, MAD, FDA, Health Canada, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Pharmaceutical industry

BOSTON, May 30, 2023 (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 announced that the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding their Investigational New Drug Application (IND) to initiate a Phase 1 study of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1 (DM1).

Key Points: 
  • BOSTON, May 30, 2023 (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 announced that the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding their Investigational New Drug Application (IND) to initiate a Phase 1 study of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1 (DM1).
  • The FDA indicated its intention to provide an official clinical hold letter to PepGen stating the reasons for the clinical hold within 30 days.
  • “We are disappointed to receive a clinical hold notice on our planned PGN-EDODM1 study in the U.S., and we will work closely with the FDA to lift the hold as quickly as possible,” said James McArthur, Ph.D., President and CEO of PepGen.
  • The clinical hold in the U.S. placed on PGN-EDODM1 does not impact the CONNECT1-EDO51 study which has been cleared to proceed in Canada.

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-101

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목요일, 5월 25, 2023
Myopathy, DYN, Therapy, DM1, European Medicines Agency, Research, EMA, Clinical trial, European, Diagnosis, MPH, Second Half, Pharmaceutical industry, Dyne

WALTHAM, Mass., May 25, 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 that the European Medicines Agency (EMA) has granted orphan drug designation for DYNE-101. DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1 (DM1).

Key Points: 
  • DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1 (DM1).
  • “We are pleased to receive orphan drug designation from the EMA for DYNE-101, further supporting our efforts to develop a potentially transformative therapy for DM1,” said Wildon Farwell, M.D., MPH, chief medical officer of Dyne.
  • “The DM1 community has waited far too long for a therapy that addresses the underlying cause of this devastating rare muscle disease.
  • Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of market exclusivity in the European Union if approved.

PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy

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목요일, 5월 18, 2023
Dystrophin, Duchenne muscular dystrophy, DSMB, NOL, DM1, Clinical trial, Drug, Pharmacokinetics, Hope, CTA, Trial of the century, DMD, EDO, Patient, MAD, HV, SVP, Health Canada, Safety, Medical imaging, Pharmaceutical industry

BOSTON, May 18, 2023 (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 announced that the company has received a No Objection Letter (NOL) for its Clinical Trial Application (CTA) from Health Canada for its Phase 2 CONNECT1-EDO51 study to initiate an open label, multiple ascending dose (MAD) clinical trial of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) amenable to an exon 51 skipping approach. The Company expects to begin dosing patients with PGN-EDO51 in 2H 2023. 

Key Points: 
  • The Company expects to begin dosing patients with PGN-EDO51 in 2H 2023.
  • “Today is the next step in our development of PGN-EDO51, a potentially transformative treatment candidate for people living with DMD.
  • “At these dose levels, the majority of treatment emergent adverse events (TEAE) were assessed as mild and resolved without any intervention.
  • The Phase 2 study will evaluate safety, tolerability, dystrophin levels, pharmacokinetics and clinical assessments.

Dyne Therapeutics’ Preclinical Data Demonstrating FORCE™ Platform Delivery to CNS Featured in Oral Presentation at ASGCT Annual Meeting

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수요일, 5월 17, 2023
DYN, Central nervous system, Asian literature, Therapy, Gene, IV, DM1, Brain, Cardiac muscle, RNA, CNS, CTG, Patient, FORCE, Deficit spending, Cell, DMPK, ASO, Phenotype, Oligonucleotide, Society, Transferrin, Disease, Vaccine, Medical device, Dyne

WALTHAM, Mass., May 17, 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, is delivering an oral presentation today at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles highlighting new preclinical data demonstrating the FORCE™ platform achieved delivery to the central nervous system (CNS) in non-human primates and robust pharmacological effects in the brain in a model of myotonic dystrophy type 1 (DM1).

Key Points: 
  • The presentation will be available in the Scientific Publications & Presentations section of Dyne’s website following the session.
  • The FORCE platform was designed to overcome the limitations of delivering oligonucleotide therapeutics to muscle tissue by leveraging transferrin receptor 1 (TfR1).
  • TfR1-mediated delivery also has been shown by the field to facilitate uptake of therapeutics by the CNS.
  • Highlights from the ASGCT data include:
    In NHPs, FORCE conjugate achieved superior delivery compared to unconjugated ASO when both were administered via IV.

Dyne Therapeutics Reports First Quarter 2023 Financial Results and Business Highlights

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목요일, 5월 11, 2023
DYN, Muscular Dystrophy Association, Dystrophin, Duchenne muscular dystrophy, Therapy, Gene, DM1, Sarcolemma, Pharmacokinetics, American Academy, G&A, Oligonucleotide, Facial muscles, Biomarker, CNS, Sale, DMD, Patient, Enrollment, Episcopal conference, FORCE, Annual general meeting, Cell, MAD, R, Research, FDA, MDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Society, Conference, Food, Transferrin, Pharmaceutical industry, Medical imaging, Cryptocurrency, Brain, Fibrosis, Dyne

WALTHAM, Mass., May 11, 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 first quarter of 2023 and 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 2023 and business highlights.
  • Research and development (R&D) expenses: R&D expenses were $37.5 million for the quarter ended March 31, 2023, compared to $28.2 million for the quarter ended March 31, 2022.
  • General and administrative (G&A) expenses: G&A expenses were $7.9 million for the quarter ended March 31, 2023, compared to $7.5 million for the quarter ended March 31, 2022.
  • Net loss: Net loss for the quarter ended March 31, 2023 was $44.2 million, or $0.78 per basic and diluted share.

PepGen Reports First Quarter 2023 Financial Results and Recent Corporate Developments

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목요일, 5월 11, 2023
PGN, Therapy, DM1, Muscular dystrophy, RCT, Clinical trial, American Academy, SAD, Trial of the century, Research and development, Development, DMD, EDO, Investment, Patient, Episcopal conference, Administration, MAD, Research, American Academy of Neurology, Three Months, Conference, Safety, Pharmaceutical industry, Cryptocurrency

BOSTON, May 11, 2023 (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 first quarter ended March 31, 2023.

Key Points: 
  • “PepGen has had an impressive start to 2023 and anticipates a meaningful year ahead.
  • PGN-EDODM1: PepGen anticipates initiating, FREEDOM-DM1, a randomized, double-blind, placebo-controlled, single ascending dose (SAD) Phase 1 Study in people living with DM1 in the first half of 2023.
  • Financial Results for the Three Months Ended March 31, 2023
    Cash and cash equivalents were $165.4 million as of March 31, 2023, which is anticipated to fund currently planned operations into early 2025.
  • PepGen had approximately 23.8 million shares outstanding on March 31, 2023.

Entrada Therapeutics Reports First Quarter 2023 Financial Results

Retrieved on: 
수요일, 5월 10, 2023
Muscular Dystrophy Association, Research and development, EEV, DM1, Vertex, Clinical trial, G&A, Research, Duchenne, Entrada, Patient, Episcopal conference, Administration, Astellas Pharma, R, FDA, MDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, General Administration of Sport of China, Therapy, Muscular dystrophy, Conference, Food, Cryptocurrency, Pharmaceutical industry, IND

BOSTON, May 10, 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 first quarter ending March 31, 2023, and highlighted recent business updates.

Key Points: 
  • - $412 million in cash, cash equivalents and marketable securities as of March 31, 2023 -
    BOSTON, May 10, 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 first quarter ending March 31, 2023, and highlighted recent business updates.
  • Under the terms of the agreement, Entrada received an upfront payment of $224 million, as well as an equity investment of $26 million in February 2023.
  • Entrada selected ENTR-601-45 as the second clinical candidate within its Duchenne franchise in January 2023 and plans to submit an IND application in the fourth quarter of 2024.
  • Collaboration Revenue: Collaboration revenue was $25.3 million for the first quarter of 2023 following the closing of the Vertex Agreement in February 2023.

Design Therapeutics Provides Pipeline Updates and Reports First Quarter 2023 Financial Results

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화요일, 5월 9, 2023
Molecule, Fuchs, Cornea, ARVO, Friedreich's ataxia, DM1, Clinical trial, Lead, Mutation, G&A, Gene expression, Cell, IC50, Muscle weakness, Ophthalmology, Ongo, Investigational New Drug, GAA, CTG, Patient, FXN, Blood, Design, DMPK, MAD, R, FA, Research, Therapy, Second Half, IND, Gene, DSM-IV codes, Safety, Disease, D, Pharmaceutical industry, Vaccine

CARLSBAD, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today provided updates and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported first quarter 2023 financial results.

Key Points: 
  • “2023 is poised to be an important year for Design as we work to bring our novel GeneTAC™ small molecules closer to patients suffering from devastating genetic diseases,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics.
  • Design now anticipates presenting initial results from the MAD trial in the third quarter of 2023.
  • R&D Expenses: Research and development (R&D) expenses were $15.7 million for the quarter ended March 31, 2023.
  • Net Loss: Net loss was $19.3 million for the quarter ended March 31, 2023.