PKP2

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.

Tenaya Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, March 18, 2024
PKP2, Biopsy, ARV, Administration, Fast track (FDA), Gene editing, Patient, SAN, CGMP, BlackRock, HDAC6, Column, G&A, IND, Research and development, Clinical trial, HCM, Investment, Therapy, Antibody, MYBPC3, Safety, FDA, ARVC, Heart failure, LP, Pharmaceutical industry

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 reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update.

Key Points: 
  • “Tenaya had a successful year of sustained execution in 2023 that meaningfully advanced our portfolio of genetic medicines for heart disease.
  • Research & Development (R&D) Expenses: R&D expenses were $22.9 million for the fourth quarter and $98.0 million for the full year ended December 31, 2023.
  • General & Administrative (G&A) Expenses: G&A expenses were $8.6 million for the fourth quarter and $33.2 million for the full year ended December 31, 2023.
  • Net Loss: Net loss was $29.9 million, or $0.40 per share for the fourth quarter ended December 31, 2023.

Gene-Based Therapy May Slow Development of Life-Threatening Heart Condition

Retrieved on: 
Tuesday, January 30, 2024
DSM-IV codes, NYU Langone Health, Survival, Circulation, Cell, PKP2, Research, Arrhythmia, Human, Heart, Cell biology, Ischemia, Patient, Mouse, Safety, Personalized medicine, NYU, MBA, Blood, Chantal, Disease, Risk, ARVC, Doctor of Philosophy, Cardiomyopathy, Cerrone

The heart can also beat irregularly without any warning and sometimes stops working.

Key Points: 
  • The heart can also beat irregularly without any warning and sometimes stops working.
  • While current therapies can help restore the heart's normal rhythm and control symptoms, they fail to provide a cure.
  • "Our findings offer experimental evidence that gene therapy targeting plakophilin-2 can interrupt the progression of a deadly heart condition," says study co-lead author Chantal van Opbergen, PhD, a postdoctoral research fellow at NYU Langone Health.
  • According to the study authors, the most advanced stages of ARVC are marked by irreversible heart damage, sometimes requiring a heart transplant.

Orphan designation: Adeno-associated virus vector serotype 9 containing the PKP2 gene Treatment of arrhythmogenic right ventricular cardiomyopathy due to plakophilin-2 gene mutations, 16/08/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Committee, PKP2, Cardiomyopathy, EMA, Pharmaceutical industry

EU/3/23/2824 - orphan designation for treatment of arrhythmogenic right ventricular cardiomyopathy due to plakophilin-2 gene mutations

Key Points: 
  • EU/3/23/2824 - orphan designation for treatment of arrhythmogenic right ventricular cardiomyopathy due to plakophilin-2 gene mutations
    Adeno-associated virus vector serotype 9 containing the PKP2 gene
    OrphanHuman
    Qdossier B.V.
    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:

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.

Tenaya Therapeutics Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, November 8, 2023
FDA, CGMP, MYBPC3, Heart failure, IND, ARV, AAV, Food, PD, Heart, Fast track (FDA), PKP2, ARVC, G&A, Ischemia, European Medicines Agency, Research and development, SGLT2, HCMS, MAD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, SAD, R, Orphan, Hypertrophic cardiomyopathy, American Heart, Plasma, Clinical trial, HDAC6, EMA, Cardiomyopathy, Gene, General Administration of Sport of China, Heart failure with preserved ejection fraction, Safety, HCM, Therapy, Patient, Investment, AHA, Administration, SAN, Investigational New Drug, Pharmaceutical industry, Vaccine, Medical device, Rare-earth element, Tenaya

SOUTH SAN FRANCISCO, Calif., Nov. 08, 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, today reported financial results and provided a corporate update for the third quarter ended September 30, 2023.

Key Points: 
  • In October 2023, Tenaya shared positive Phase 1 data for TN-301 at the 2023 Heart Failure Society of America (HFSA) Annual Scientific Meeting.
  • General & Administrative (G&A) Expenses: G&A expenses for the quarter ended September 30, 2023, were $7.8 million.
  • Non-cash stock-based compensation included in G&A expense was $2.2 million for the quarter ended September 30, 2023.
  • Net Loss: Net loss for the quarter ended September 30, 2023, was $29.1 million, or $0.39 per share.

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] .

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, November 6, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Consensus, Patient, Research, Busulfan, American College, CGMP, Genetic testing, Clinical trial, AAV, PDUFA, Incidence, IMPD, LV, Danon disease, Malignancy, Medication, Mortality, CMC, Conference, CTA, Acquisition, PKP2, Disease, JACC, Journal, Consensus decision-making, Priority review, Male, PKD, Natural history, MHRA, Disorder, EMA, Arrhythmia, Journal of the American College of Cardiology, LAMP2, Safety, Investment, Diagnosis, BLA, Pediatric Blood & Cancer, Pharmaceutical industry, Medical device, Security (finance), Fine chemical, G&A

Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.

Key Points: 
  • Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.
  • Highlights of the publication include a review of diagnosing Danon Disease emphasizing the importance of genetic testing upon clinical suspicion, natural history, management recommendations and recent advances in potential gene therapy treatment.
  • Rocket is working towards initiation of Phase 2 pivotal trial activities in Europe and the UK.
  • As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million.

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.