PKP2

Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

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星期三, 五月 29, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Patient, Disorder, PKP2, European Medicines Agency, Medication, Disease, Safety, Person, Marketing, Arrhythmogenic cardiomyopathy, Civil service commission, European Commission, EMA, Therapy, Committee, COMP, European, Arrest

PKP2-ACM is a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.

Key Points: 
  • PKP2-ACM is 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.
  • Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the European Union (EU).
  • The study is assessing the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, clinical predictors of life-threatening ventricular arrhythmias, and sudden cardiac death.

Tenaya Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

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星期二, 五月 14, 2024
Patient, Epidemiology, Cell, Survival, University, PLN, Risk, Nature Communications, PKP2, Cardiomyopathy, Research and development, Workforce, Heart failure, Investment, AAV, Clinical trial, Doctor of Philosophy, ARVC, Therapy, Safety, HCM, Chemistry, Biopsy, CMC, HEK, Gene, Enrollment, Cas9, HDAC6, Society, CRISPR, G&A, Administration, Second Half, Antibody, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., May 14, 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 first quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • “The year is off to a strong start for Tenaya,” said Faraz Ali, Chief Executive Officer of Tenaya.
  • Tenaya anticipates sharing initial safety, biopsy and biomarker data from the first cohort of patients in the MyPEAK-1 trial in the second half of 2024.
  • General & Administrative (G&A) Expenses: Year-over year G&A expenses were relatively flat at $8.7 million for the first quarter of 2024 and $8.1 million for the first quarter of 2023.
  • Non-cash stock-based compensation included in G&A expense was $2.2 million for the first quarter of 2024 compared to $1.9 million for the first quarter of 2023.

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

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星期一, 三月 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

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星期一, 三月 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

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星期二, 一月 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

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星期日, 二月 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)

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星期一, 十二月 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

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星期三, 十一月 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

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星期二, 十一月 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

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星期一, 十一月 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.