Danon disease

Rocket Pharmaceuticals Announces $26.4 Million Private Placement

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월요일, 8월 30, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Company, Private Securities Litigation Reform Act, Investment, Securities Act of 1933, Genetic disorder, U.S. Securities and Exchange Commission, LVV, AAV, SEC, Research, Bone marrow failure, IMO, RTW, PKD, Osteopetrosis, Marketing, Fanconi anemia, LP, Medication, NASDAQ, Prospectus, Safety, Pyruvate kinase deficiency, Patient, Sale, FA, Lists of diseases, Annual report, COVID-19, Heart failure, Danon disease, Pharmaceutical industry

The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.
  • Rocket expects to use the net proceeds from the private placement to continue to advance and expand its pipeline of product candidates, for research and development expenses and for working capital.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that aim to correct the root cause of complex and rare childhood disorders.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501

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월요일, 8월 16, 2021
Other Health, Research, General Health, Pharmaceutical, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, FDA, Other Science, Health, Tissue, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, Bone marrow failure, Danon disease, Adult, Male, Genetic disorder, FA, Company, Risk, Food, Private Securities Litigation Reform Act, Lists of diseases, Death, COVID-19, Program, LVV, SEC, Pyruvate kinase deficiency, Autophagy, FDA, Safety management system, Gene expression, PKD, Fanconi anemia, Patient, Osteopetrosis, Lifting, Marketing, IMO, Medication, Heart failure, Glycogen, Safety, AAV, Cardiac muscle, Autophagosome, Prevalence, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.

Key Points: 
  • We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
  • We look forward to progressing this critical work on behalf of all Danon patients, said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Rockets Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs at 24th Annual Meeting of the American Society of Gene and Cell Therapy

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목요일, 5월 13, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Branches of biology, Biology, Applied genetics, Genetics, Gene delivery, Parvovirinae, Emerging technologies, Molecular biology, Adeno-associated virus, Danon disease, AAV, Gene therapy

All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.

Key Points: 
  • All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.
  • Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia.
  • Rocket\xe2\x80\x99s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Reports First Quarter 2021 Financial Results and Highlights Recent Progress

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월요일, 5월 10, 2021
Biotechnology, Pharmaceutical, Oncology, General Health, Health, FDA, Genetics, Clinical trials, Danon disease

Shah continued, \xe2\x80\x9cWe are encouraged by the positive low dose RP-A501 Phase 1 trial results in Danon Disease disclosed today.

Key Points: 
  • Shah continued, \xe2\x80\x9cWe are encouraged by the positive low dose RP-A501 Phase 1 trial results in Danon Disease disclosed today.
  • We anticipate a one quarter delay in enrollment to address FDA requests for risk mitigation methods in our protocol.
  • These updated results increase our confidence in the low dose as a potentially viable dose for patients with Danon Disease.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Reports Positive Long-Term Clinical Data from RP-L201 Trial for the Treatment of Leukocyte Adhesion Deficiency-I at the Clinical Immunology Society 2021 Annual Meeting

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수요일, 4월 14, 2021
Health, Genetics, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Rare diseases, Parvovirinae, Branches of biology, Adeno-associated virus, Parvoviruses, Danon disease, AAV, Clinical medicine, Applied sciences, Leukocyte Adhesion Deficiency-I, Rocket Pharmaceuticals, Inc.

LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal.

Key Points: 
  • LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal.
  • In all patients treated, CD18 expression has substantially exceeded the 4-10% threshold associated with survival into adulthood, with consistent peripheral blood vector copy number levels.
  • Rocket\xe2\x80\x99s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Announces Buildout of R&D and Manufacturing Facility to Support Development of Innovative Gene Therapy Pipeline

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월요일, 1월 11, 2021
Health, Stem cells, Genetics, Pharmaceutical, Cardiology, Biotechnology, Branches of biology, Biology, Genetics, Applied genetics, Gene delivery, Parvovirinae, Emerging technologies, Medical genetics, Adeno-associated virus, Danon disease, AAV, Gene therapy

This new 103,720 ft2 facility will support clinical development of Rockets growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercialization.

Key Points: 
  • This new 103,720 ft2 facility will support clinical development of Rockets growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercialization.
  • This new facility is instrumental in bolstering Rockets gene therapy capabilities to rapidly advance multiple platforms and programs efficiently and effectively.
  • The other half features state-of-the-art R&D labs to support the expanding pipeline and Quality Control (QC) laboratories to support CMC development for process and analytics.
  • Rocket recently reported positive interim Phase 1 results for its first AAV-based gene therapy, RP-A501 for the treatment of Danon Disease.

Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease

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화요일, 12월 8, 2020
Health, Stem cells, Genetics, Pharmaceutical, Cardiology, Biotechnology, Branches of biology, Rare diseases, Health, Danon disease, Clinical medicine, Adeno-associated virus, Creatine kinase, Creatine, Heart, Autophagic Vacuolar Myopathy, Danon Disease, Rocket Pharmaceuticals, Inc.

In addition, RP-A501 demonstrated reduction of myocardial cell disarray and accumulation of autophagic vacuoles, a hallmark of Danon Disease.

Key Points: 
  • In addition, RP-A501 demonstrated reduction of myocardial cell disarray and accumulation of autophagic vacuoles, a hallmark of Danon Disease.
  • Males with Danon Disease typically have elevated BNP, transaminases and creatine kinase as a result of skeletal and heart muscle damage.
  • These early results suggest a path to a potentially transformative option for Danon Disease, and possibly the first viable gene therapy approach for cardiac diseases.
  • 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 First Patient Treated in Higher Dose Cohort in Phase 1 Clinical Trial of RP-A501 for Danon Disease

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수요일, 9월 2, 2020
FDA, Genetics, Clinical trials, Stem cells, Cardiology, Biotechnology, Health, Pharmaceutical, Oncology, Branches of biology, Biology, Applied genetics, Genetics, Danon disease, Emerging technologies, Medical genetics, Adeno-associated virus, A501, Gene therapy, Danon Disease, Rocket Pharmaceuticals, Inc.

The second cohort of the Phase 1 study evaluates RP-A501 at a higher dose level of 1.11014 genome copies/kilogram in male patients 15 years of age and older.

Key Points: 
  • The second cohort of the Phase 1 study evaluates RP-A501 at a higher dose level of 1.11014 genome copies/kilogram in male patients 15 years of age and older.
  • RP-A501 represents a potentially holistic and transformative approach to Danon Disease, a devastating disease often resulting in end-stage heart failure and early mortality.
  • The non-randomized, open-label Phase 1 trial aims to enroll both pediatric and young adult male patients in escalating dose cohorts.
  • 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 FDA Clearance of IND for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis

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월요일, 6월 29, 2020
Health, Stem cells, Genetics, Pharmaceutical, Cardiology, Biotechnology, Branches of biology, Biology, Rare diseases, Genetics, Gene delivery, Applied genetics, Osteopetrosis, Gene therapy, Danon disease, Viral vector, Adeno-associated virus, Malignant infantile osteopetrosis, Infantile Malignant Osteopetrosis, Rocket Pharmaceuticals, Inc.

RP-L401 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure.

Key Points: 
  • RP-L401 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure.
  • This FDA acceptance marks the fifth Rocket-Sponsored IND cleared for our gene therapy platform in less than two years and is an important milestone for Rocket, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket.
  • Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease

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월요일, 6월 8, 2020
Medicine, Rare diseases, Clinical medicine, Health, Inborn errors of carbohydrate metabolism, Lysosomal storage diseases, Glycogen storage disease type II, Hepatology, Alglucosidase alfa, Pompe, Danon disease, Lysosomal storage disease

Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.

Key Points: 
  • Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.
  • The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to avalglucosidase alfa for the treatment of patients with a confirmed diagnosis of Pompe disease.
  • Pompe disease is often classified as late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD).
  • Avalglucosidase alfa is an investigational ERT for Pompe disease designed to improve the delivery of enzyme to the cells in the muscles, most notably into skeletal muscle.