PKD

Rocket Pharmaceuticals Announces Updated Positive Preliminary Clinical Data from Phase 1 Trial of RP-L301 for the Treatment of Pyruvate Kinase Deficiency

Retrieved on: 
Monday, March 22, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Branches of biology, Medical specialties, Hemoglobins, Tetrapyrroles, Pharmaceutical industry, Biology, Equilibrium chemistry, Respiratory physiology, Bilirubin, Validation, PKD, Pyruvate Kinase Deficiency, Rocket Pharmaceuticals, Inc.

The positive updates on our PKD trial represent continued validation of RP-L301s robust potential benefit in addressing the challenges of PKD.

Key Points: 
  • The positive updates on our PKD trial represent continued validation of RP-L301s robust potential benefit in addressing the challenges of PKD.
  • A similar trend in hemoglobin and bilirubin normalization were observed at three months post-treatment in patient 2.
  • We are excited to share this data as results show the potential of RP-L301 to address the extensive burden PKD places on patients and their families.
  • We look forward to presenting more comprehensive and updated data at a scientific venue this year.

Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency

Retrieved on: 
Monday, July 13, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Branches of biology, Biology, Applied genetics, Gene delivery, Inborn errors of carbohydrate metabolism, Pyruvate kinase, Emerging technologies, Medical genetics, PKD, PKLR, Adeno-associated virus, Glycolysis

We believe gene therapy treatment with RP-L301 has the potential to be a safe and transformative approach to improve long-term patient outcomes.

Key Points: 
  • We believe gene therapy treatment with RP-L301 has the potential to be a safe and transformative approach to improve long-term patient outcomes.
  • The global Phase 1 open-label, single-arm, clinical trial is expected to enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe.
  • Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Announces Clearance of IND for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency

Retrieved on: 
Tuesday, October 22, 2019
Research, FDA, Genetics, Clinical trials, Cardiology, Stem cells, Biotechnology, Pharmaceutical, Health, Science, Branches of biology, Biology, Life sciences, Applied genetics, Biotechnology, Pyruvate kinase, Glycolysis, Gene therapy, PKD, Food and Drug Administration

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces the clearance of the Companys Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L301.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces the clearance of the Companys Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L301.
  • The RP-L301 IND marks an important milestone as it is the first global Phase 1 study for Rocket in the U.S. and EU.
  • Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases.

Global Polycystic Kidney Disease (PKD) Market Report 2019: Insights, Epidemiology & Forecast 2017-2028 - ResearchAndMarkets.com

Retrieved on: 
Thursday, October 17, 2019
Health, Infectious diseases, Kidney diseases, Ciliopathy, Polycystic kidney disease, PKD, Polycystic disease

The Polycystic Kidney Disease (PKD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 10 emerging countries.

Key Points: 
  • The Polycystic Kidney Disease (PKD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 10 emerging countries.
  • According to this report, the total number of prevalent cases of Polycystic Kidney Disease (PKD) in 10 EM was found to be 1,912,820 in the year 2017.
  • This segment of the Polycystic Kidney Disease report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs.
  • The market forecast focuses on the market revenue generated by the pharmacological treatment which are primarily symptomatic or palliative in nature.

Global Polycystic Kidney Disease Drugs Market 2019-2023 | 6% CAGR Projection over the Next Five Years | Technavio

Retrieved on: 
Wednesday, June 19, 2019
Health, Biotechnology, Genetics, Pharmaceutical, FDA, Kidney diseases, Medical specialties, Medicine, Autosomal dominant polycystic kidney disease, Polycystic kidney disease, Polycystic disease, Kidney disease, Autosomal recessive polycystic kidney disease, PKD

View the full release here: https://www.businesswire.com/news/home/20190619005315/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20190619005315/en/
    Technavio has published a new market research report on the global polycystic kidney disease drugs market from 2019-2023.
  • (Graphic: Business Wire)
    A key factor driving the growth of the global polycystic kidney disease drugs market size is the special regulatory designations.
  • This global polycystic kidney disease drugs market 2019-2023 research report also analyzes other important trends and market drivers that will affect market growth over 2019-2023.
  • Global polycystic kidney disease drugs market: Segmentation analysis
    This market research report segments the global polycystic kidney disease drugs market by type (ADPKD, and ARPKD), and geographic regions (North America, Europe, Asia, and ROW).

Rocket Pharmaceuticals Sponsors 3rd Annual PKD Patients’ Forum

Retrieved on: 
Wednesday, May 29, 2019
Stem cells, Health, Biotechnology, Clinical trials, Genetics, Pharmaceutical, Branches of biology, Biology, Life sciences, PKD, Pyruvate kinase deficiency, Pyruvate kinase, Gene therapy

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today sponsors the 3rd Annual Pyruvate Kinase Deficiency (PKD) Day in Madrid, Spain, a day devoted to patients, families, clinicians and global scientific experts.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today sponsors the 3rd Annual Pyruvate Kinase Deficiency (PKD) Day in Madrid, Spain, a day devoted to patients, families, clinicians and global scientific experts.
  • At Rocket, we are making significant progress towards developing transformative gene therapies for patients living with devastating, rare diseases, including PKD, said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Executive Vice President of Development of Rocket.
  • We are pleased to sponsor the 3rd Annual PKD Patients Forum to discuss PKD diagnosis and present and future treatments, including the forthcoming gene therapy clinical trial for PKD, said Dr. Segovia.
  • Internationally recognized PKD experts, as well as clinicians in close contact with the patients are attending todays Forum.

JYNARQUE™ Approved as First Treatment for Polycystic Kidney Disease

Retrieved on: 
Tuesday, April 24, 2018
Kidney diseases, Polycystic kidney disease, Medicine, PKD, Anatomy, Polycystic disease, Kidney, Clinical medicine, Critical Path Institute, Autosomal dominant polycystic kidney disease

The PKD Foundation not only supported early studies that lead to the development of JYNARQUE as a treatment, but also helped guide PKD patients to the JYNARQUE clinical trials.

Key Points: 
  • The PKD Foundation not only supported early studies that lead to the development of JYNARQUE as a treatment, but also helped guide PKD patients to the JYNARQUE clinical trials.
  • JYNARQUE slows the growth of cysts and the progression of the disease and has been shown to preserve kidney function.
  • The PKD Foundation has been dedicated since its founding in 1982 to supporting and improving the lives of patients affected by polycystic kidney disease.
  • We fund research, advocate for patients and build a community for all affected by polycystic kidney disease (PKD).