DUX4

Solid Biosciences Announces Licensing Agreement with Armatus Bio for the Use of AAV-SLB101, a Proprietary, Muscle-Targeted Capsid, in the Development of an RNAi Therapy to treat FSHD

Retrieved on: 
Thursday, March 7, 2024
Liver, Duchenne, Toxicity, Inflammation, DMD, Facioscapulohumeral muscular dystrophy, DUX4, Solid, FSHD, Capsid, MicroRNA, Oxidative stress, B cell, IND, RNA interference, AAV, Atrophy, Vaccine

The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.

Key Points: 
  • The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.
  • Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of FSHD and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development.
  • Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101.
  • With this license in place, we are eager to advance our optimized ARM-201 construct as the lead candidate toward clinical evaluation.”

Satellos Announces Promising Preliminary Data in Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Tuesday, February 13, 2024
Health, Neurology, Other Health, General Health, Pharmaceutical, Biotechnology, MSCL, Research, Myotonic dystrophy, Dystrophin, Facioscapulohumeral muscular dystrophy, Duchenne muscular dystrophy, AAK1, Entrepreneurship, Conference, Muscular dystrophy, DUX4, Science, Phenotype, Patient, Mouse

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD).
  • We look forward to presenting these data at the MDA Clinical and Scientific Conference 2024 and advancing SAT-3247 into clinical trials for Duchenne muscular dystrophy mid-year.”
    Neil Camarta, Co-Founder and CEO of the FSHD Canada Foundation, said “I’m very pleased.
  • I’m looking forward to working with Satellos to accelerate the development of this promising drug candidate, because time is muscle!”
    FSHD is the third most common muscular dystrophy behind Duchenne (& Beckers) and myotonic dystrophy.
  • FSHD is an adult onset muscular dystrophy that results in the progressive destruction of muscle tissue, owed to the erroneous expression of a gene product called DUX4.

Orphan designation: humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker treatment of facioscapulohumeral muscular dystrophy, 15/02/2023 Positive

Retrieved on: 
Saturday, January 20, 2024
COMP, Immunoglobulin G, DUX4, European Commission, Small RNA, Transferrin, EMA, Facioscapulohumeral muscular dystrophy, IRIS, Committee, Pharmaceutical industry

EU/3/23/2756 - orphan designation for treatment of facioscapulohumeral muscular dystrophy

Key Points: 
  • EU/3/23/2756 - orphan designation for treatment of facioscapulohumeral muscular dystrophy
    humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker
    OrphanHuman
    MWB Consulting
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

FDA Grants Orphan Drug Designation to EPI-321, Epic Bio's Novel Genetic Medicine Candidate for Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Retrieved on: 
Thursday, November 16, 2023
Safety, SAN, Food, Disease, Biotechnology, FSHD, Patient, FDA, Gene expression, DCAS, Trial of the century, Muscular dystrophy, Facioscapulohumeral muscular dystrophy, DUX4, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., Nov. 16, 2023 (GLOBE NEWSWIRE) -- Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults. EPI-321 is an investigational therapy being developed as a potential single-dose treatment to suppress abnormal expression of the DUX4 gene.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 16, 2023 (GLOBE NEWSWIRE) -- Epic Bio , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults.
  • EPI-321 is an investigational therapy being developed as a potential single-dose treatment to suppress abnormal expression of the DUX4 gene.
  • Epic plans to initiate a first-in-human, Phase 1/2 clinical study of EPI-321 in the first half of 2024.
  • The multi-center study will be designed to assess the safety, activity, and preliminary efficacy of EPI-321 in individuals with FSHD.

Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate a Phase 1/2 Study of ARO-DUX4 for Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Monday, July 17, 2023
Infectious Diseases, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, General Health, Oncology, Molecule, Clinical trial, Committee, Pharmacokinetics, Medicine, RNA, FSHD, Facioscapulohumeral muscular dystrophy, Patient, RNA interference, DUX4, Gene, Safety, Disease, Pharmaceutical industry, Arrowhead

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD).
  • ARO-DUX4 is the first clinical candidate utilizing Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform to target disease associated genes in skeletal muscle.
  • We believe ARO-DUX4 has the potential to be an impactful new medicine for patients,” said Chris Anzalone, Ph.D., president and CEO of Arrowhead.
  • Pending clearance, Arrowhead intends to proceed with ARODUX4-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DUX4 in adult patients with FSHD type 1.

Epic Bio Presents Preclinical Data on EPI-321 for Facioscapulohumeral Muscular Dystrophy at ASGCT 26th Annual Meeting

Retrieved on: 
Friday, May 19, 2023
GEMS, Cell death, Therapy, Gene, Biotechnology, Survival, DCAS, Clinical trial, Gene expression, FSHD, Facioscapulohumeral muscular dystrophy, SAN, Cell, DUX4, IND, Society, Disease, Vaccine, Pharmaceutical industry, Science

SOUTH SAN FRANCISCO, Calif., May 19, 2023 (GLOBE NEWSWIRE) -- Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today presented promising preclinical data supporting development of EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The data were shared in an oral presentation at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 19, 2023 (GLOBE NEWSWIRE) -- Epic Bio , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today presented promising preclinical data supporting development of EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
  • The data were shared in an oral presentation at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.
    “We’re proud to share the preclinical data from our lead program, EPI-321, in an oral presentation at the distinguished ASGCT meeting,” said Alexandra Collin de l’Hortet, Ph.D., head of therapeutics at Epic Bio.
  • “This validation of our GEMS platform’s ability to produce potentially transformative therapies is very exciting,” said Amber Salzman, Ph.D., chief executive officer of Epic Bio.
  • The presentation will be made available under the publications section of the Science page on the Epic Bio website.

Avidity Biosciences Receives FDA Orphan Drug Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Tuesday, February 14, 2023
Fatigue, Disability, Therapy, Antibody-oligonucleotide conjugate, Pain, Disorder, AOC, Patient, Small RNA, Clinical trial, RNA, Food, FSHD, DUX4, FDA, Avidity, SAN, Orphan drug, Facioscapulohumeral muscular dystrophy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

SAN DIEGO, Feb. 14, 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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • SAN DIEGO, Feb. 14, 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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
  • "We are pleased that the FDA has granted Orphan Drug designation to AOC 1020, reinforcing the importance of finding an effective treatment option for people living with FSHD.
  • There are currently no treatment options to address this devastating and debilitating muscular dystrophy disorder," said Steve Hughes, M.D., chief medical officer at Avidity.
  • "AOC 1020 is designed to directly target the disease-causing gene, DUX4, with the goal of treating the underlying biological cause of FSHD.

Epic Bio Presents Proprietary Gene Expression Modulation System (GEMS) Demonstrating Precise Engineering of DUX4 Gene in Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Thursday, October 13, 2022
Health, Genetics, Health Technology, Clinical Trials, Pharmaceutical, Biotechnology, Gene expression, LinkedIn, Bio, FSHD, Movement, A1AD, IND, Solebury Township, Bucks County, Pennsylvania, DUX4, Biotechnology, Patient, Depression, RNA, Twitter, Tissue, Research, GEMS, DNA-binding protein, AAV, DCAS, Cas9, Face, CRISPR, Horizons Ventures, Scapula, Anxiety, DNA, Chronic pain, Elective surgery, Facioscapulohumeral muscular dystrophy, FDA, Engineering, Muscular dystrophy, Vaccine

I am excited by the data we will present today on Facioscapulohumeral Muscular Dystrophy (FSHD), the second most prevalent form of muscular dystrophy.

Key Points: 
  • I am excited by the data we will present today on Facioscapulohumeral Muscular Dystrophy (FSHD), the second most prevalent form of muscular dystrophy.
  • Notably, our in vitro results demonstrate the utility of our dCas to suppress DUX4 expression by up to 95%.
  • Facioscapulohumeral Muscular Dystrophy is estimated to be the second most common muscular dystrophy, affecting 1 in 8,000 patients1,2.
  • The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies.

Fulcrum Therapeutics to Present New Data from the Open Label Extension of Phase 2 ReDUX4 Study at the World Muscle Society (WMS) Congress in Halifax, Canada

Retrieved on: 
Wednesday, October 12, 2022
Gene expression, LinkedIn, U.S. Securities and Exchange Commission, GSK, Orphan, Fulcrum, Therapy, Ascending limb of loop of Henle, Sickle cell disease, Fetal hemoglobin, Review, Quality, Open-label trial, Thalassemia, WMS, Neuro, Muscle weakness, Facial expression, Patient, Facioscapulohumeral muscular dystrophy, Halifax Convention Centre, DUX4, OLE, Clinical trial, MFI, Twitter, HIV disease progression rates, Security (finance), Collection, Nasdaq, Private Securities Litigation Reform Act, Hemoglobinopathy, Șaeș, PGIC, Quality of life, Fast track (FDA), Muscular dystrophy, REACH, Safety, EDT, GLOBE, RWS, MAPK, FDA, COVID-19, Pharmaceutical industry, FSHD, Industry, Losmapimod

CAMBRIDGE, Mass., Oct. 12, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced today that new data from the open label extension (OLE) portion of the Phase 2 ReDUX4 study of losmapimod for the treatment of FSHD will be featured in a poster presentation at the World Muscle Society (WMS) Hybrid Congress taking place October 11-15 in Halifax, Canada. Fulcrum will also host a symposium entitled “Measuring Progression in FSHD: Implications for Clinical Trials,” featuring an overview of the OLE data on October 12, 2022, at the Halifax Convention Center.

Key Points: 
  • ReDUX4 was a 48-week, placebo-controlled study that enrolled 80 participants between the ages of 18-65 with genetically confirmed FSHD1 (Ricci score 2-4).
  • Of the 77 participants who completed the initial 48-week study, 99% (n=76) enrolled in the OLE.
  • Data from the OLE support findings that losmapimod modifies FSHD disease progression and preserves or improves muscle function.
  • Following the completion of the trial, 99% of eligible participants elected to continue in the Open Label Extension trial.

miRecule Enters into Strategic Collaboration with Sanofi to Accelerate Discovery and Development of a Best-in-Class Antibody-RNA Conjugate to Treat Facioscapulohumeral Muscular Dystrophy (FSHD)

Retrieved on: 
Tuesday, October 4, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Cancer, Facioscapulohumeral muscular dystrophy, ARC, Drug development, Muscular dystrophy, NINDS, Disease, IND, National Institute, National Institute of Neurological Disorders and Stroke, CEO, Head, Family, Antibody, Patient, Philosophy, Ageing, Partnership, RNA, Program, FSHD, Friends, Lists of diseases, Organization, Research, DUX4, Therapy, Pharmacology, Pharmaceutical industry, Stroke, Sanofi

FSHD is the second most common type of muscular dystrophy affecting more than one million individuals worldwide with no approved treatments.

Key Points: 
  • FSHD is the second most common type of muscular dystrophy affecting more than one million individuals worldwide with no approved treatments.
  • Under the terms of the collaboration agreement, miRecule will grant Sanofi an exclusive worldwide license to intellectual property rights to the FSHD therapy.
  • Following candidate selection, Sanofi will assume sole responsibility for IND enabling studies and subsequent development and commercialization activities globally.
  • Sanofi and miRecule look forward to continuing to work with these organizations to help bring forward a groundbreaking treatment for FSHD.