Bone marrow failure

Rocket Pharmaceuticals Announces Participation in 11th Annual SVB Leerink Global Healthcare Conference

Retrieved on: 
Thursday, February 10, 2022
General Health, Health, Genetics, Pharmaceutical, Clinical Trials, NASDAQ, Private Securities Litigation Reform Act, Heart failure, Genetic disorder, LVV, Conference, Danon disease, Lists of diseases, Investor, PKD, Pyruvate kinase deficiency, Marketing, Medication, Patient, Safety, SEC, Lifting, Bone marrow failure, FA, AAV, Fanconi anemia, Risk, COVID-19, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m.
  • The webcast replay will be available on the Rocket website following the conference.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that 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.

Active Biotech enters into global patent license agreement with Oncode Institute for tasquinimod in myelofibrosis

Retrieved on: 
Wednesday, February 9, 2022
NASDAQ, CFO, Multiple myeloma, Trial of the century, Erasmus, Laquinimod, Patent, III, Ruxolitinib, ACTI, Naptumomab estafenatox, Pancreatic serous cystadenoma, Marketing, Lymphatic system, Infection, Incidence, Therapy, Justicia oncodes, MC, Risk, Erythropoietin, Patient, Spleen, Survival, Anemia, Haematopoietic system, Acute leukemia, CEO, CET, Bone marrow failure, Death, Biotechnology, Erasmus MC, Fever, Disease, EU, Partnership, Bone marrow, Fibrosis, JAK, Pharmaceutical industry, MF

Under the terms of the agreement, Oncode Institute, a foundation incorporated under the laws of the Netherlands, acting on behalf of Erasmus Universiteit Medisch Centrum (Erasmus MC), grants a global exclusive license to develop and commercialize tasquinimod worldwide in MF to Active Biotech.

Key Points: 
  • Under the terms of the agreement, Oncode Institute, a foundation incorporated under the laws of the Netherlands, acting on behalf of Erasmus Universiteit Medisch Centrum (Erasmus MC), grants a global exclusive license to develop and commercialize tasquinimod worldwide in MF to Active Biotech.
  • Active Biotech will pay to Oncode Institute, contingent of marketing approval, milestones as well as low single-digit royalties on net sales.
  • The results suggest that tasquinimod can act as a disease modifying agent in MF.
  • Active Biotech and Erasmus MC will initiate a research collaboration related to use of tasquinimod in MF, that includes preclinical as well as a clinical proof of concept study in patients with MF.

Rocket Pharmaceuticals Highlights Anticipated 2022 Milestones Across Lentiviral and AAV Gene Therapy Clinical Programs

Retrieved on: 
Tuesday, January 11, 2022
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, FA, Patient, PKD, Conference, CD18, Genetic disorder, Danon disease, Private Securities Litigation Reform Act, SEC, COVID-19, MBA, Safety, Risk, NASDAQ, LVV, Pyruvate kinase deficiency, Lists of diseases, Medication, Heart failure, Leukocyte adhesion deficiency, Fanconi anemia, Lifting, Marketing, AAV, Pharming, Bone marrow failure, Pharmaceutical industry, EU

The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.

Key Points: 
  • The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.
  • Rockets 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 to Present at the 40th Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Tuesday, January 4, 2022
Health, Genetics, Other Health, Clinical Trials, Research, Science, Safety, Genetic disorder, COVID-19, Investor, AAV, Private Securities Litigation Reform Act, Heart failure, IMO, Lists of diseases, LVV, FA, Medication, Lifting, NASDAQ, Conference, SEC, Danon disease, Fanconi anemia, Pyruvate kinase deficiency, Bone marrow failure, Osteopetrosis, Marketing, Patient, Risk, PKD, Pharmaceutical industry

A live audio webcast of the presentation will be available under Events in the Investors section of the Companys website at https://ir.rocketpharma.com/ .

Key Points: 
  • A live audio webcast of the presentation will be available under Events in the Investors section of the Companys website at https://ir.rocketpharma.com/ .
  • The webcast replay will be available on the Rocket website following the conference.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that 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 Presents Positive Clinical Data from Company’s Lentiviral Gene Therapies for Treatment of Fanconi Anemia, LAD-I and PKD at the 63rd American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Monday, December 13, 2021
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, CIEMAT, Lists of diseases, HSCT, NASDAQ, Survival, Pyruvate kinase deficiency, Anemia, Risk, MMC, California Institute for Regenerative Medicine, Squamous cell carcinoma, Patient, CIRM, Adult, Blood, FDA, Pneumonia, DNA repair, Diagnosis, DEB, PKD, Splenectomy, FANCA, AAV, Head, Mutation, Regenerative medicine, Pyruvate kinase, Fanconi anemia, Safety, Quality of life, Grant, Birth, ASH, Union, ITGB2, Osteopetrosis, Heart failure, Investor, COVID-19, IMO, SEC, Research, Marketing, DNA, Prevalence, Sepsis, Centro, Danon disease, FA, Șaeș, Death, Life, Private Securities Litigation Reform Act, Genetic disorder, CD18, Mosaic (genetics), Hematopoietic stem cell transplantation, Bone marrow failure, Mortality, LVV, PKLR, Infection, Pharmaceutical industry, Vaccine, Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, Rocket Pharmaceuticals, Inc., FANCONI ANEMIA, LEUKOCYTE ADHESION DEFICIENCY-I, PYRUVATE KINASE DEFICIENCY, ROCKET PHARMACEUTICALS, INC.

RP-L301 continues to be well tolerated, with no drug product related SAEs or infusion-related complications observed through 12-months post-treatment.

Key Points: 
  • RP-L301 continues to be well tolerated, with no drug product related SAEs or infusion-related complications observed through 12-months post-treatment.
  • At approximately 12-months post-treatment the patient had normalized hemoglobin levels of 14.8 g/dL, compared to a pre-treatment baseline of ~7.0 g/dL.
  • Rocket will host an in-person Investor and Analyst Event that will simultaneously be webcast tomorrow, Dec. 14, at 7:30 a.m.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.

Journal Nature Medicine Features Co-development of "Game-changing" Drug by Versiti Chief Scientific Officer Dr. Michael Deininger

Retrieved on: 
Tuesday, December 14, 2021
B cell, Food, Allergy, Lymphatic system, Research, Cancer, Weight loss, Nature Medicine, ASM, HCI, Growth, Medical college, Allergen, Professor, Mast cell, Flushing, Survival, Anemia, MCW, Transplant, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Patient, Bone marrow failure, Quality of life, Fracture, Immune system, Transfusion medicine, Medical College of Wisconsin, Multimedia, University, Bleeding, Pep Deininger, Jon M. Huntsman Center, Division, Hematology, Health, Diarrhea, Pharmaceutical industry, Vaccine, Medicine

Dr. Michael Deininger, Versiti's chief scientific officer and director of the Versiti Blood Research Institute, is a co-senior author of a paper published Dec. 6 in the journal Nature Medicine detailing the safety and efficacy of avapritinib, a drug approved June of 2021 by the U.S. Food and Drug Administration for use against ASM.

Key Points: 
  • Dr. Michael Deininger, Versiti's chief scientific officer and director of the Versiti Blood Research Institute, is a co-senior author of a paper published Dec. 6 in the journal Nature Medicine detailing the safety and efficacy of avapritinib, a drug approved June of 2021 by the U.S. Food and Drug Administration for use against ASM.
  • It occurs most commonly in older adults but is not associated with any racial or geographic groups.
  • When mast cells accumulateand become unstable, they often release substances that can trigger severe allergic reactions, flushing,bone painand diarrhea.
  • Deininger describes the drug as "undoubtedly a game-changer" for its effect on ASM's symptoms and patients' survival prospects.

Rocket Pharmaceuticals Appoints Martin L. Wilson as General Counsel and Chief Compliance Officer

Retrieved on: 
Wednesday, December 8, 2021
Science, Research, Pharmaceutical, Health, FDA, Infectious Diseases, Genetics, Clinical Trials, Bone marrow failure, Endo, Lists of diseases, General counsel, Fanconi anemia, Business development, Heart failure, Endo International, SEC, Compliance, Biotechnology, Par, Human resources, Risk, Villanova University, Leadership, Genetic disorder, FA, Safety, Drug development, IMO, Private Securities Litigation Reform Act, PKD, Food, Patient, Danon disease, Corporation, LVV, Human, Pyruvate kinase deficiency, Lifting, Senior, COVID-19, Osteopetrosis, Medication, FDA, Marketing, NASDAQ, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Law, AAV, Juris Doctor, License, Acquisition, Schering-Plough, Assistant, Management, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President.
  • Prior to joining Rocket, Mr. Wilson was General Counsel and Chief Corporate Officer at Ichnos Sciences, where he oversaw Legal, Compliance, Business Development, Human Resources and IT.
  • Before Ichnos, Mr. Wilson served as General Counsel, Chief Compliance Officer, Corporate Secretary and Head of Human Resources at Teligent, Inc.
  • Prior to Teligent, he was Vice President and Assistant General Counsel at Endo Pharmaceuticals following the acquisition of Par Pharmaceuticals.

Rocket Pharmaceuticals to Host In-Person Investor and Analyst Event and Webcast on Tuesday, Dec. 14 at 7:30 a.m. ET to Discuss Updated Clinical Data Presented at the 63rd American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Tuesday, December 7, 2021
Health, Genetics, Other Health, General Health, Pharmaceutical, Cardiology, Bone marrow failure, Lists of diseases, Investor, Fanconi anemia, Heart failure, SEC, Genetic disorder, FA, Safety, IMO, Private Securities Litigation Reform Act, PKD, Patient, Danon disease, LVV, ASH, Pyruvate kinase deficiency, Lifting, COVID-19, Osteopetrosis, Medication, Marketing, NASDAQ, AAV, Society, Risk, Pharmaceutical industry

Clinical data for all three trials will be presented at the 63rd American Society of Hematology (ASH) Annual Meeting being held Dec. 11-14, 2021.

Key Points: 
  • Clinical data for all three trials will be presented at the 63rd American Society of Hematology (ASH) Annual Meeting being held Dec. 11-14, 2021.
  • A simultaneous webcast of the event and the presentation will be available under Events in the Investors section of the Companys website at: https://ir.rocketpharma.com/ .
  • The webcast replay will be available on the Rocket website upon completion of the event.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.

Kronos Bio Announces First Patient Dosed in AGILITY Phase 3 Clinical Trial of Entospletinib in Patients With Newly Diagnosed NPM1-mutated Acute Myeloid Leukemia

Retrieved on: 
Monday, December 6, 2021
MLL, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Cell, Haematopoietic system, Disease, Lymphatic system, Gilead Sciences, SYK, Hematology, NPM1, DNA, Mutation, Risk, GLOBE, Infection, CDK9, Bone marrow, LinkedIn, Department, Food, Private Securities Litigation Reform Act, Security (finance), Cytarabine, Cancer, Acute myeloid leukemia, U.S. Securities and Exchange Commission, Research, Polymerase chain reaction, Bleeding, CR, MFC, Allele, Bone marrow failure, Protein, EFS, Forward-looking statement, Therapy, MRD, Survival, Adult, FDA, Minimal residual disease, Patient, Heart failure, Safety, PCR, SEC, SAN, City, COVID-19, KRON, Nasdaq, MEIS1, Antibody, HOXA9, Entospletinib, Factor X, Pharmaceutical industry

SAN MATEO, Calif. and CAMBRIDGE, Mass., Dec. 06, 2021 (GLOBE NEWSWIRE) -- Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, today announced that the first patient has been dosed in the registrational Phase 3 AGILITY clinical trial of entospletinib, a selective inhibitor targeting spleen tyrosine kinase (SYK), in combination with standard of care anthracycline and cytarabine (7+3) chemotherapy. This trial is the first in acute myeloid leukemia (AML) to use measurable residual disease (MRD) as the primary endpoint and has the potential to support accelerated approval of entospletinib by the U.S. Food and Drug Administration (FDA) as a treatment for patients newly diagnosed with NPM1-mutated AML who are fit for intensive induction.

Key Points: 
  • Entospletinib is Kronos Bios lead product candidate, and the company expects to share data from the trial in the second half of 2023.
  • This trial will test the hypothesis, based on robust preclinical and Phase 2 clinical data, that NPM1 mutation leads to dependency on SYK signaling.
  • In the trial, patients will be randomized 1:1 to receive either entospletinib or placebo in combination with standard induction and consolidation chemotherapy.
  • Kronos Bio is developing entospletinib for the frontline treatment of NPM1-mutated acute myeloid leukemia (AML).

Rocket Pharmaceuticals Announces Participation in Evercore ISI 4th Annual HEALTHCONx Virtual Conference

Retrieved on: 
Monday, November 22, 2021
General Health, Other Health, Health, Genetics, Cardiology, Patient, FA, Private Securities Litigation Reform Act, Osteopetrosis, Heart failure, IMO, Safety, Risk, Conference, Lists of diseases, Evercore, PKD, SEC, Investor, COVID-19, LVV, Event-driven architecture, Marketing, Pyruvate kinase deficiency, AAV, Medication, Fanconi anemia, Danon disease, Genetic disorder, Lifting, NASDAQ, Bone marrow failure, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Evercore ISI 4th Annual HEALTHCONx Virtual Conference on Wednesday, Dec. 1, 2021 at 10:55 a.m.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Evercore ISI 4th Annual HEALTHCONx Virtual Conference on Wednesday, Dec. 1, 2021 at 10:55 a.m.
  • The webcast replay will be available on the Rocket website following the conference.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that 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.