Arrhythmogenic cardiomyopathy

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.

Rocket Pharmaceuticals Receives FDA Fast Track and Orphan Drug Designations for RP-A601 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Retrieved on: 
Thursday, June 8, 2023
Biotechnology, FDA, Pharmaceutical, Health, Communication, PKP2, Arrest, Survival, Arrhythmia, Disorder, Medication, Investigational New Drug, Arrhythmogenic cardiomyopathy, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Safety, Disease, Pharmaceutical industry

RP-A601 is Rocket’s adeno-associated virus (AAV.rh74)-based gene therapy for PKP2-ACM, a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.

Key Points: 
  • RP-A601 is Rocket’s adeno-associated virus (AAV.rh74)-based gene therapy for PKP2-ACM, a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
  • The current standard of care consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablation procedures, none of which are curative.
  • Orphan Drug designation is granted to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the U.S.
  • RP-A601 is the first gene therapy in development for PKP2-ACM to receive IND clearance and the second of three programs in the Company’s AAV-based cardiovascular gene therapy franchise.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy Programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

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Friday, May 19, 2023
Health, Pharmaceutical, Biologics license application, PVC, LV, Hemolysis, Gene, ACM, Arrest, Survival, Bone marrow, Monogenic, Quality of life, PKD, Liver injury, Research, Arrhythmia, AAV, Fanconi anemia, Disorder, Pyruvate kinase deficiency, Natriuresis, Medication, First, MMC, HIV disease progression rates, Patient, Arrhythmogenic cardiomyopathy, Recombinant, Busulfan, Hematology, Disease, Cell, Kaplan–Meier estimator, Toxicology, Danon disease, Pivotal, Mortality, FA, FDA, BLA, IND, Society, Safety, Pharmaceutical industry, Medical device, Nursing, Birth control, Vaccine, PKP2

“I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.

Key Points: 
  • “I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.
  • Both adult patients reported improved quality of life with documented improvements via formal quality of life assessments.
  • The safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.
  • Rocket is initiating Phase 1 study start-up activities and rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic.

Rocket Pharmaceuticals Announces FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Retrieved on: 
Tuesday, May 9, 2023
Health, FDA, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, GMP, Heart, Arrhythmia, Partnership, Doctor of Pharmacy, University, Gene, Arrest, Medulla oblongata, Survival, NYU, FACC, New York University Grossman School of Medicine, Philadelphia, MBA, Child, Disorder, UC San Diego Health, Medication, Arrhythmogenic cardiomyopathy, Patient, Division, Cell, Hospital, Recurrence, Regulation of tobacco by the U.S. Food and Drug Administration, IND, News, Society, Food, Safety, Disease, Pharmaceutical industry, Vaccine, PKP2, FAAP

The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative.

Key Points: 
  • The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative.
  • The study will assess the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death.
  • These studies showed that RP-A601 decreased arrhythmias, improved right ventricular morphology and function, and increased survival in PKP2 knockout mice.
  • Rocket is initiating Phase 1 study start-up activities and rapidly working towards first patient treatment.

BioAro Unveils Its Targeted Cardiac Panel for Patients Facing the Risk of Heart Failure During the Peak of Heart Month

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Monday, February 20, 2023
Death, Cardiology, Facial muscles, Dilated cardiomyopathy, Medicine, Cancer, Diagnosis, Mortality, Athlete, Physician, Genetic counseling, Gene, Risk, Cardiovascular disease, Research, Whole genome sequencing, Patient, Senior counsel, Genetics, Heart failure, History, Environment, Cardiomyopathy, Splenic infarction, Genetic, Health, Aneurysm, Sport, Arrhythmogenic cardiomyopathy, Genetic testing, Hypertrophic cardiomyopathy, Cardiac arrest, Person, Genome, Medical device, Pharmaceutical industry, Dietary supplement

Calgary, Alberta--(Newsfile Corp. - February 20, 2023) - Canada-based biotechnology company BioAro announces the unveiling of its new targeted cardiac panel for patients who face the potential risks of heart failure.

Key Points: 
  • Calgary, Alberta--(Newsfile Corp. - February 20, 2023) - Canada-based biotechnology company BioAro announces the unveiling of its new targeted cardiac panel for patients who face the potential risks of heart failure.
  • Almost 1 in 3 Canadians are touched by heart failure, and over 100,000 Canadians get diagnosed with a new diagnosis of heart failure every year.
  • The launch also seeks to raise awareness of various heart diseases, as part of international heart month in February 2023.
  • BioAro promotes this month to take extra care of heart health and help reduce the mortality rate due to heart diseases.

BioAro is the first Canadian company to launch comprehensive genetic testing panels for various heart diseases

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Thursday, February 9, 2023
Medicine, Splenic infarction, Athlete, Heart, Dilated cardiomyopathy, Genetic testing, Multimedia, Physician, Facial muscles, Heart failure, Risk, AB, Genetics, Blood pressure, GMP, Hypercholesterolemia, Kidney, Blood, Artery, Diagnosis, Cardiac arrest, Patient, Cardiology, IHD, Skin, Sport, Arrhythmogenic cardiomyopathy, Gene, Environment, Ageing, Woman, Arrest, Cardiovascular disease, Parent, Hypertension, Stroke, Dietary supplement, Pharmaceutical industry, Medical device, Vaccine

Almost 1 in 3 Canadians are touched by heart failure, and over 100,000 new Canadians are diagnosed with heart failure every year.

Key Points: 
  • Almost 1 in 3 Canadians are touched by heart failure, and over 100,000 new Canadians are diagnosed with heart failure every year.
  • February is known as International Heart month in order to raise awareness about the various heart conditions and create ways to help in reducing the mortality rate caused due to heart diseases.
  • BioAro is taking an initiative to launch the genetic testing of targeted panels for heart disease conditions using high-throughput next generation sequencing technique.
  • Heart Failure: Heart failure is a chronic, progressive condition in which your heart muscle can't pump enough blood to meet your body's needs all the time.

Rocket Pharmaceuticals Expands Cardiac Gene Therapy Portfolio with Addition of RP-A601 for PKP2-ACM and Announces Positive Updated Phase 1 Data for RP-A501 in Danon Disease

Retrieved on: 
Monday, January 9, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, IND, Disease, Cardiomyopathy, New York University Grossman School of Medicine, Danon disease, Toxicology, ARVC, Pharmacology, AAV, Natural history, ACM, FA, Arrhythmia, Degenerative disease, FDA, NYHA, Biomarker, Fanconi anemia, Medication, Genetics, New York Heart Association Functional Classification, Conference, Autosome, Adolescence, BLA, Survival, PVC, Patient, Arrhythmogenic cardiomyopathy, Heart failure, HIV disease progression rates, Mortality, CGMP, NYU, GMP, BNP, Pharmaceutical industry, Vaccine, PKP2, EU

We have shared these updates with the FDA as part of our pivotal Phase 2 study design discussions.

Key Points: 
  • We have shared these updates with the FDA as part of our pivotal Phase 2 study design discussions.
  • “Lastly, I am pleased to note an extension of our cash runway, which we expect to fund operations through 2024.
  • Available treatments fail to address the underlying genetics and disease biology and do not alter disease progression.
  • A live audio webcast of the presentation is available under “Events” in the Investors section of the Company’s website at https://ir.rocketpharma.com .

Renovacor Reports Second Quarter 2022 Financial Results and Provides Corporate Update

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Monday, August 8, 2022
Biotechnology, Health, Pharmaceutical, Clinical Trials, Cardiology, Secondary research, Precision medicine, NYSE, IND, ACM, Degenerative disease, American College, Patient, University, RCSI, Biology, Â, Second Half, Heart, Arrhythmogenic cardiomyopathy, RCOR, AAV, Pharmaceutical industry

Renovacor, Inc. (NYSE: RCOR), a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today reported financial results for the second quarter of 2022 and provided a corporate update.

Key Points: 
  • Renovacor, Inc. (NYSE: RCOR), a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today reported financial results for the second quarter of 2022 and provided a corporate update.
  • The progress achieved at Renovacor this quarter enabled us to advance and expand our pipeline of precision AAV gene therapies that target the underlying drivers of devastating cardiovascular diseases, said Magdalene Cook, M.D., Chief Executive Officer of Renovacor.
  • To accelerate this program, Renovacor entered into a research collaboration with the University of Utahs Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI).
  • The terms of the research agreement grant Renovacor an option for an exclusive license to inventions generated from research conducted under the collaboration.

Renovacor Announces Pipeline Expansion with New Research Program for Multiple Genetic Segments of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Tuesday, July 12, 2022
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To accelerate this new program, Renovacor has entered into a research collaboration with the University of Utahs Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI).

Key Points: 
  • To accelerate this new program, Renovacor has entered into a research collaboration with the University of Utahs Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI).
  • The terms of the research agreement grant Renovacor an option for an exclusive license to inventions generated from the collaboration.
  • The research collaboration will focus on a protein discovered by University of Utah scientists that has the potential to address multiple genetic segments of ACM.
  • The program will be developed for the three largest genetic segments of ACM: plakophilin-2 (PKP2), desmoglein-2 (DSG2), and desmoplakin (DSP) associated ACM.

Tenaya Therapeutics to Present Preclinical Data on Gene Therapy Programs and Platform Capabilities at the American Society of Gene and Cell Therapy 25th Annual Meeting

Retrieved on: 
Tuesday, May 3, 2022
General Health, Other Health, Health, Clinical Trials, Cardiology, Gene, COVID-19, U.S. Securities and Exchange Commission, Cardiomyopathy, Arrhythmogenic cardiomyopathy, University, IND, NASDAQ, DCM, University of Texas Southwestern Medical Center, Degenerative disease, Heart failure, Cell, PKP2, Arrhythmia, AAV, Food, Safety, Securities Act of 1933, Patient, Security (finance), Survival, Clinical trial, Drug Quality and Security Act, Therapy, Industry, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Marketing, Society, Disease, Investigational New Drug, Lists of diseases, Securities Exchange Act of 1934, ARVC, American Society of Gene and Cell Therapy, SERCA, HIV disease progression rates, Precision medicine, Annual general meeting, Model, Gladstone Institutes, Pharmaceutical industry, Vaccine, Tenaya

TN-401 is Tenayas gene therapy candidate being developed for the potential treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations.

Key Points: 
  • TN-401 is Tenayas gene therapy candidate being developed for the potential treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations.
  • These mutations are estimated to affect more than 70,000 patients in the U.S. alone.
  • Data being presented at ASGCT will detail the impact of a single dose of PKP2 gene therapy on arrhythmias, disease progression and survival in a Pkp2-deficient mouse model of ARVC.
  • The companys DWORF gene therapy is being developed for the potential treatment of genetic dilated cardiomyopathy (DCM).