Myotonic Dystrophy Foundation

Juvena Therapeutics Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1

Retrieved on: 
Tuesday, January 23, 2024
Biotechnology, FDA, Pharmaceutical, Health, Entrepreneurship, Obesity, DSM-IV codes, Erythropoietin, Therapy, Facial muscles, Muscular dystrophy, Walking, Orphan drug, Homeostasis, Growth hormone, Drug discovery, Insulin, Disorder, Food, Human, DM1, Protein, Myotonic Dystrophy Foundation, Myotonic dystrophy, Metabolism, Pharmaceutical industry

Juvena Therapeutics , a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161.

Key Points: 
  • Juvena Therapeutics , a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161.
  • JUV-161 is an investigational therapeutic for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare, multi-systemic, autosomal dominant inherited disease and the most common form of adult muscular dystrophy.
  • “People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair and restore tissue health to improve muscle function and metabolism,” said Dr. Hanadie Yousef, Co-Founder and CEO of Juvena Therapeutics.
  • Additionally, Juvena has identified multiple secreted protein hits that induce disease-modifying effects across several metabolic organs and therapeutic areas.

PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1

Retrieved on: 
Wednesday, September 6, 2023
Orphan Drug Act of 1983, Neuromuscular disease, PGN, Myotonic Dystrophy Foundation, NOL, NMD, DM1, Health Canada, CTA, SAD, Patient, FDA, Conference, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MDF, IND, Food, Safety, D.C, Disease, Pharmaceutical industry, Biology

In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.

Key Points: 
  • In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.
  • The FREEDOM-DM1 clinical trial is a randomized, placebo-controlled, single ascending dose (SAD) study, intended to enroll approximately 24 adult patients with DM1 to evaluate the safety and tolerability of PGN-EDODM1.
  • We expect to report initial results from this study in 2024,” said James McArthur, Ph.D., President and CEO of PepGen.
  • PepGen continues to work closely with the FDA to resolve the clinical hold in the United States as quickly as feasible.

PepGen Inc. Presents PGN-EDODM1 Preclinical Data Supporting the Company’s Enhanced Delivery Oligonucleotide Platform and PGN-EDODM1 Program at Two Medical Conferences

Retrieved on: 
Wednesday, September 6, 2023
Neuromuscular disease, PGN, Heart, Human, Myotonic Dystrophy Foundation, Duchenne muscular dystrophy, Therapy, NMD, DM1, Safety, Peptide, Cell, RNA, Duchenne, Myotonic dystrophy, Myotonia, EDO, DMD, Electrophysiology, NHP, Conference, MDF, Oligonucleotide, Potential, Disease, Pharmaceutical industry, Fine chemical, Tolerability, Pharmacokinetics, Biology

BOSTON, Sept. 06, 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 key highlights from the upcoming presentation of preclinical non-human primate (NHP) data supporting PepGen’s proprietary Enhanced Delivery Oligonucleotide (EDO) platform at the 6th Ottawa International Conference on Neuromuscular Disease and Biology (NMD) being held on September 7-9, 2023 in Ottawa, ON, Canada, and at the 2023 Myotonic Dystrophy Foundation (MDF) Annual Conference being held on September 7-9, 2023 in Washington, D.C.

Key Points: 
  • Additionally, the presentation reported that EDO technology was also shown to improve in vivo nuclear delivery, with 72% of skeletal muscle nuclei in NHPs positive for oligonucleotide following two doses at 30mg/kg.
  • PepGen previously demonstrated that a single dose of 30 mg/kg of PGN-EDODM1 corrected myotonia in the DM1 HSALR mouse model as measured by electrophysiology.
  • Treatment emergent adverse events in this study at these dose levels were transient, mild (grade 1), and reversible and did not require intervention.
  • “We have previously demonstrated the ability of the EDO technology to deliver up to 50 nM of oligonucleotide to biceps in humans following a single dose.

Rgenta Therapeutics Announces Formation of Repeat Expansion Diseases Focused Scientific Advisory Board

Retrieved on: 
Wednesday, June 21, 2023
UK Academy for Information Systems, SAB, Huntington's disease, New England, DNA, Myotonic Dystrophy Foundation, Muscular dystrophy, Disease, Pathology, Research, Brain, Department of Neurology, Xuanwu hospital, RNA, Genome, Boston University, Science, Human genetics, Gene silencing, Genetics, Facioscapulohumeral muscular dystrophy, Virginia Commonwealth University, Neurology, Knowledge, Ghana Infectious Disease Centre, Reds, Therapy, Gene, NIH, Myotonic dystrophy, ALS, Fragile X syndrome, DNA repair, Pharmaceutical industry, Chobanian, Medicine

CAMBRIDGE, Mass., June 21, 2023 /PRNewswire/ -- Rgenta Therapeutics today announced the formation of a Repeat Expansion Diseases (REDs) Focused Scientific Advisory Board (REDs SAB).

Key Points: 
  • CAMBRIDGE, Mass., June 21, 2023 /PRNewswire/ -- Rgenta Therapeutics today announced the formation of a Repeat Expansion Diseases (REDs) Focused Scientific Advisory Board (REDs SAB).
  • REDs SAB is an esteemed group of preclinical and clinical experts who contribute to basic and translational research on the mechanisms and treatments for repeat expansion diseases.
  • "We are proud and honored to introduce our REDs focused SAB," said Dr Zhiping Weng, Rgenta SAB chair.
  • "Their knowledge and insights to the repeat expansion diseases will help advance the preclinical and clinical development of our small molecule programs."

PepGen Welcomes Esteemed Neurologist Dr. John Day to Scientific Advisory Board

Retrieved on: 
Wednesday, October 13, 2021
Degenerative disease, Myotonic dystrophy, Division, National Advisory Committee on Immunization, Organization, San Francisco, Professor, Duchenne muscular dystrophy, LinkedIn, Lists of diseases, DMD, Pathology, Coronavirus Scientific Advisory Board (Turkey), DM1, Neuromuscular medicine, Society, University of California, San Francisco, Albert Einstein College of Medicine, American Academy, GLOBE, Myotonic Dystrophy Foundation, University, Muscle, American Academy of Neurology, Twitter, Education, Edos, Pharmaceutical industry

BOSTON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- PepGen, Inc. , a company advancing next-generation oligonucleotide therapies for neuromuscular and neurologic diseases,today announcedthe appointment of neuromuscular neurologist John W. Day, M.D., Ph.D., to its Scientific Advisory Board.

Key Points: 
  • BOSTON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- PepGen, Inc. , a company advancing next-generation oligonucleotide therapies for neuromuscular and neurologic diseases,today announcedthe appointment of neuromuscular neurologist John W. Day, M.D., Ph.D., to its Scientific Advisory Board.
  • Dr. Day is a Professor of Neurology, Pediatrics (Genetics) and Pathology and Director of the Division of Neuromuscular Medicine at Stanford University.
  • Its my pleasure to welcome Dr. John Day, who brings decades of experience working and teaching in the neuromuscular disease space, to PepGens Scientific Advisory Board, said James McArthur, Ph.D., President and Chief Executive Officer of PepGen.
  • John is a renowned expert in neuromuscular diseases and particularly myotonic dystrophy, a severe, progressive disorder that currently has no cure.

NeuBase Therapeutics to Present at the Myotonic Dystrophy Foundation 2020 Virtual Conference

Retrieved on: 
Thursday, September 17, 2020
Neurological disorders, Organ systems, Musculoskeletal disorders, Myotonic Dystrophy Foundation, RTT, Myotonic dystrophy, DM1, Dystrophy, Myotonia

PITTSBURGH, Sept. 17, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (NeuBase or the Company), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today announced that Dietrich A. Stephan, Ph.D., chief executive officer, is scheduled to present at the Myotonic Dystrophy Foundation 2020 Virtual Conference on September 25 between 4:00 p.m. and 4:45 p.m.

Key Points: 
  • PITTSBURGH, Sept. 17, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (NeuBase or the Company), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today announced that Dietrich A. Stephan, Ph.D., chief executive officer, is scheduled to present at the Myotonic Dystrophy Foundation 2020 Virtual Conference on September 25 between 4:00 p.m. and 4:45 p.m.
  • The conference will be held virtually on September 25-26, 2020 and is designed as an educational event for individuals and families living with myotonic dystrophy.
  • The presentation will provide an overview of NeuBases PATrOL platform and the potential of its therapeutic approach for myotonic dystrophy type 1 (DM1).
  • "We are honored to be participating in this years Myotonic Dystrophy Foundation conference and are looking forward to engaging with the community, reaffirming NeuBases commitment to finding a new treatment option and introducing patients and families to the transformational work being done at NeuBase, said Dr. Stephan.

Triplet Therapeutics Announces Participation in Natural History Study of Myotonic Dystrophy Type 1

Retrieved on: 
Tuesday, August 18, 2020
Research, Other Health, General Health, Pharmaceutical, Other Education, University, Genetics, Education, Clinical trials, Science, Biotechnology, Health, Other Science, Organ systems, Neurological disorders, RTT, Musculoskeletal disorders, Myotonic dystrophy, DM1, Myotonic Dystrophy Foundation, Dystrophy, Muscular dystrophy, Myotonia, Trinucleotide repeat disorder, Myotonic Dystrophy Type 1, Triplet Therapeutics, VCU and VCU Health

Triplet Therapeutics, Inc., a biotechnology company leveraging insights from the human genetics of repeat expansion disorders (REDs), today announced its participation in END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1), a large international natural history study designed to advance understanding of myotonic dystrophy type 1 (DM1) and ultimately support the development of new clinical approaches to address this unmet medical need.

Key Points: 
  • Triplet Therapeutics, Inc., a biotechnology company leveraging insights from the human genetics of repeat expansion disorders (REDs), today announced its participation in END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1), a large international natural history study designed to advance understanding of myotonic dystrophy type 1 (DM1) and ultimately support the development of new clinical approaches to address this unmet medical need.
  • DM1, along with Huntingtons disease (HD), is a near-term focus of Triplets therapeutic efforts, and the first of several REDs affecting muscle that Triplet is pursuing.
  • Other supporters include the U.S. Food and Drug Administration, the Myotonic Dystrophy Foundation, the Wyck Foundation, the Muscular Dystrophy Association, and Dyne Therapeutics.
  • Triplet also recently launched SHIELD HD ( ClinicalTrials.gov identifier NCT04406636 ), an international natural history study of HD.

Dyne Therapeutics Announces Appointment of Molly White as Vice President, Medical Communications and Advocacy

Retrieved on: 
Monday, January 13, 2020
Biotechnology, Pharmaceutical, Health, Neurological disorders, Organ systems, Myotonic Dystrophy Foundation, Medicine, RTT, Myotonic dystrophy, Myotonia, Dystrophy

Dyne Therapeutics , a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced the appointment of Molly White as Vice President, Medical Communications and Advocacy.

Key Points: 
  • Dyne Therapeutics , a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced the appointment of Molly White as Vice President, Medical Communications and Advocacy.
  • Ms. White previously served as chief executive officer of Myotonic (formerly the Myotonic Dystrophy Foundation), the world's largest patient organization focused solely on myotonic dystrophy.
  • Were thrilled to begin the year by adding Molly to our rapidly growing team at Dyne, said Joshua Brumm, president and chief executive officer of Dyne.
  • With her experience leading Myotonic, Molly has demonstrated outstanding leadership and passionate advocacy in seeking to change the lives of those affected by myotonic dystrophy.

Muscular Dystrophy Association Names Sharon Hesterlee, Ph.D., Executive Vice President, Chief Research Officer

Retrieved on: 
Monday, December 9, 2019
RTT, Neurological disorders, Organ systems, Health, Amyotrophic lateral sclerosis, Dystrophy, Medical terminology, Muscular Dystrophy Association, Muscular dystrophy, Myotonic dystrophy, MDA, Myotonic Dystrophy Foundation

NEW YORK, Dec. 9, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the appointment of Sharon Hesterlee, Ph.D., as executive vice president, chief research officer.

Key Points: 
  • NEW YORK, Dec. 9, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the appointment of Sharon Hesterlee, Ph.D., as executive vice president, chief research officer.
  • In her new role, she will spearhead innovations in research and science by delivering strategic and operational leadership of MDA's discovery, translational and clinical research portfolio.
  • In the 15 years before transitioning to industry-focused roles, Dr. Hesterlee led research and strategy for several leading nonprofit organizations, having served as Chief Science Officer for the Myotonic Dystrophy Foundation, Vice President of Research for Parent Project Muscular Dystrophy and Scientific Director of the Association for Frontotemporal Degeneration.
  • During her initial work with MDA, she served as Senior Vice President and Executive Director of MDA Venture Philanthropy and Vice President for Translational Research, among other positions.

Locana appoints Kathie Bishop, Ph.D., as Chief Scientific Officer

Retrieved on: 
Wednesday, September 4, 2019
Neurological disorders, Organ systems, RTT, Spinal muscular atrophy, Myotonic Dystrophy Foundation, Dystrophy, Myotonic dystrophy, Locana, Myotonia

SAN DIEGO, Sept. 4, 2019 /PRNewswire/ --Locana, a leading RNA-targeting gene therapy company, today announced the appointment of Kathie M. Bishop, Ph.D. as chief scientific officer.

Key Points: 
  • SAN DIEGO, Sept. 4, 2019 /PRNewswire/ --Locana, a leading RNA-targeting gene therapy company, today announced the appointment of Kathie M. Bishop, Ph.D. as chief scientific officer.
  • "We are delighted that Kathie has joined the Locana team," said Jeffrey M. Ostrove, Ph.D., chief executive officer of Locana.
  • Prior to Otonomy, she served as chief scientific officer of Tioga Pharmaceuticals and was responsible for all scientific and drug development activities of a novel small molecule for the treatment of chronicpruritus.
  • Dr. Bishop currently serves on the scientific advisory board of the Myotonic Dystrophy Foundation and is chief clinical officer of the Spinal Muscular Atrophy Foundation.