Arrhythmogenic cardiomyopathy

Tenaya Therapeutics Announces Publication of TN-401 Gene Therapy Preclinical Data in Nature Communications Medicine

Retrieved on: 
Monday, March 18, 2024
PKP2, Arrhythmogenic cardiomyopathy, Arrhythmia, Survival, Disease, Risk, Patient, SAN, Hope, Quality of life, Protein, ACM, Clinical trial, Therapy, Heart, Cardiomyopathy, Safety, Vaccine

SOUTH SAN FRANCISCO, Calif., March 18, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced the publication of its preclinical research related to its gene therapy candidate, TN-401, in the current issue of Nature Communications Medicine.

Key Points: 
  • TN-401 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy being developed for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
  • TN-401 is designed to deliver a functional PKP2 gene to heart cells where it works to restore normal protein levels in order to halt or even reverse disease after a single dose.
  • “Following a single infusion of our AAV9-based PKP2 gene therapy in a severe knock-out mouse model of the disease, PKP2 protein levels were restored.
  • Tenaya is currently also conducting the RIDGE™ global non-interventional natural history and serotype study of PKP2-associated ARVC.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, PKD, Personalized medicine, EMA, Research, MAA, Investment, Marketing, Food, Arrhythmogenic cardiomyopathy, Circulation, BLA, PDUFA, Fanconi anemia, Conference, Safety, DCM, European Medicines Agency, Food and Drug Administration, Muscle weakness, Efgartigimod alfa, Pyruvate kinase deficiency, Patient, Disorder, Clinical trial, Medication, ACM, IND, AAV, CGMP, CMC, Priority review, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

AMO Pharma Announces Collaboration with Population Health Research Institute to Advance Proof of Concept Clinical Trial to Assess Efficacy of Tideglusib in Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Thursday, February 15, 2024
Scar, Brock University, Myotonic dystrophy, AMO, Zebrafish, WNT, Hamilton Health Sciences, SCD, Duchenne muscular dystrophy, Tideglusib, Facial muscles, McMaster University, Risk, Health, ICD, Phenotype, DSM-IV codes, Patient, Arrhythmogenic cardiomyopathy, Target, MOA, Hiro, ACM, Research, Pharmaceutical industry

Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.

Key Points: 
  • Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.
  • The TaRGET trial is a critical first step in evaluating this potential."
  • This scarring increases the risk of dangerously fast heart rhythms that can lead to sudden cardiac death (SCD).
  • An implantable cardioverter defibrillator (ICD) is recommended for prevention of SCD for ACM patients considered at high risk for SCD.

AMO Pharma Announces Collaboration with Population Health Research Institute to Advance Proof of Concept Clinical Trial to Assess Efficacy of Tideglusib in Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Thursday, February 15, 2024
Scar, Brock University, Myotonic dystrophy, AMO, Zebrafish, WNT, Hamilton Health Sciences, SCD, Duchenne muscular dystrophy, Tideglusib, Facial muscles, McMaster University, Risk, Health, ICD, Phenotype, DSM-IV codes, Patient, Arrhythmogenic cardiomyopathy, Target, MOA, Hiro, ACM, Research, Pharmaceutical industry

Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.

Key Points: 
  • Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.
  • The TaRGET trial is a critical first step in evaluating this potential."
  • This scarring increases the risk of dangerously fast heart rhythms that can lead to sudden cardiac death (SCD).
  • An implantable cardioverter defibrillator (ICD) is recommended for prevention of SCD for ACM patients considered at high risk for SCD.

Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Retrieved on: 
Monday, December 18, 2023
Food, FDA, PKP2, Therapy, ACM, Orphan drug, Cardiac muscle, Arrhythmogenic cardiomyopathy, Arrhythmia, Biomarker, Patient, Safety, Disorder, Fast Track, Pharmaceutical industry

“Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.

Key Points: 
  • “Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.
  • Preliminary efficacy measures will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden.
  • Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial trial.
  • Fast Track designation allows more frequent FDA interactions to facilitate development and expedite the review process for novel drug candidates that treat serious or life-threatening diseases and address unmet medical needs.

MARKETVUE® REPORT: Arrhythmogenic Cardiomyopathy Gene Therapy Research Gains Momentum

Retrieved on: 
Tuesday, November 7, 2023
Catheter ablation, ICD, PKP2, Penetrance, Physician, Arrhythmogenic cardiomyopathy, REACH, Quality of life, Disease, Risk, Arrhythmia, Subcutaneous Implantable Defibrillator (S-ICD), ACM, Patient, Heart, Mutation, Medical device, Vaccine

NEWTON, Mass., Nov. 7, 2023 /PRNewswire/ -- Arrhythmogenic Cardiomyopathy (ACM) is a rare hereditary cardiac disorder characterized by progressive replacement of myocardial tissue with fibrofatty deposits, leading to life-threatening arrhythmias and cardiac dysfunction.

Key Points: 
  • ~70% of physicians surveyed by REACH Market Research report there is an extremely high unmet medical need for novel treatments for ACM.
  • NEWTON, Mass., Nov. 7, 2023 /PRNewswire/ -- Arrhythmogenic Cardiomyopathy (ACM) is a rare hereditary cardiac disorder characterized by progressive replacement of myocardial tissue with fibrofatty deposits, leading to life-threatening arrhythmias and cardiac dysfunction.
  • ACM patients typically receive treatment with beta blockers and anti-arrhythmic drugs to manage symptoms and arrhythmias.
  • To access REACH's MarketVue® Report on ACM, visit https://reachmr.com or contact us at [email protected] .

Tenaya Therapeutics Announces FDA Clearance to Begin Clinical Testing of TN-401 Gene Therapy for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy

Retrieved on: 
Thursday, October 26, 2023
PVC, Medicine, Survival, FDA, CGMP, Lightheadedness, Plasma, VT, IND, Food, ICD, Heart, Biomarker, PKP2, Palpitations, ARVC, Premature ventricular contraction, Beckman Center for Molecular and Genetic Medicine, Fear, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, HIV disease progression rates, Protein, NSVT, Arrhythmogenic cardiomyopathy, Disease, Risk, Cardiomyopathy, Arrhythmia, 52 Weeks Make A Year, Safety, ACM, Therapy, Patient, Tachycardia, DSM-IV codes, SAN, Investigational New Drug, Pharmaceutical industry, Vaccine, Medical device

SOUTH SAN FRANCISCO, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, announced today that the U.S. Food and Drug Administration (FDA) has provided clearance of the company’s Investigational New Drug (IND) application to initiate clinical testing of TN-401.

Key Points: 
  • TN-401 is Tenaya’s adeno-associated virus serotype 9 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) gene.
  • Tenaya is currently conducting the RIDGE™ global non-interventional natural history and serotype study of PKP2-associated ARVC.
  • ARVC, also known as arrhythmogenic cardiomyopathy (ACM), is a chronic, progressive, familial disease that typically presents before age 40.
  • People with ARVC experience symptoms related to ventricular arrhythmias, including palpitations, lightheadedness and fainting, and are at increased risk of sudden cardiac death.

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

Retrieved on: 
Thursday, October 19, 2023
Research, Genetics, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Patient, PKP2, Medication, Danon disease, MBA, Arrhythmogenic cardiomyopathy, Pharming, PKD, Cell, Fanconi anemia, Disorder, Gene, ESGCT, European Society of Gene and Cell Therapy, Pyruvate kinase deficiency, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27.
  • Updated data will be presented from the Phase 1 clinical trial of RP-L301 for Pyruvate Kinase Deficiency (PKD).
  • Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L102 for Fanconi Anemia, Phase 1 trial of RP-A501 for Danon Disease and preclinical studies supporting the Phase 1 trial of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).
  • Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma, will also give an Invited Talk about the importance of collaboration with and commitment to patients in gene therapy development.

Rejuvenate Bio Announces Preclinical Data for Gene Therapy Candidate RJB-0402 for the Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Monday, October 9, 2023
Genetics, Clinical Trials, Cardiology, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Disease, Cardiac cycle, HIV disease progression rates, Arrhythmia, Rejuvenation, Research, Trial of the century, B cell, DSM-IV codes, MD, Liver, ACM, IND, Heart, Sudden death, Mutation, FDA, Heart failure, AAV, Gene, ARVC, Genetic disorder, PVC, DSP, SADS, Arrhythmogenic cardiomyopathy, Cardiac muscle, FGF21, Patient, Quality of life, Vaccine, Medical device, Pharmaceutical industry

Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.

Key Points: 
  • Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.
  • RJB-0402 demonstrated efficacy in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
  • “What makes RJB-0402 especially promising is its superior performance in the ACM nonclinical model, outperforming gene replacement therapies in reducing PVCs.
  • The initial clinical study for RJB-0402 will focus on patients with Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death.

LEXEO Therapeutics Announces FDA Clearance of IND for LX2020, an AAV-based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy

Retrieved on: 
Tuesday, August 1, 2023
PKP2, University, ACM, Survival, Cardiac muscle, Biomarker, Arrhythmia, AAV, Arrhythmogenic cardiomyopathy, Patient, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Phenotype, Food, Cell adhesion, Safety, Disease, Vaccine, Pharmaceutical industry

NEW YORK, Aug. 01, 2023 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LX2020. LX2020 is an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of Arrhythmogenic Cardiomyopathy (ACM) caused by variants in the PKP2 gene (PKP2-ACM).

Key Points: 
  • LX2020 is an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of Arrhythmogenic Cardiomyopathy (ACM) caused by variants in the PKP2 gene (PKP2-ACM).
  • “This IND clearance marks an important step in advancing a potential one-time treatment for individuals with PKP2-ACM, who are in need of more effective options for this devastating disease.
  • Current clinical management strategies are only marginally effective and primarily focus on symptom management,” said Eric Adler, M.D., Chief Scientific Officer of LEXEO.
  • “LX2020 seeks to address the underlying cause of this disease by delivering a functional PKP2 gene to halt progression and reverse the disease phenotype.