Fanconi anemia

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Pharmaceutical, Oncology, General Health, Health, Genetics, Clinical Trials, Other Health, Transplant, DNA repair, Medication, Biologics license application, MAA, Risk, FANCA, Patient, Fanconi anemia, Pharming, DNA, BMF, HSCT, EMA, Bone marrow failure, Death, Birth, Life, MBA, European Medicines Agency, Marketing, FA, Disorder, FDA, BLA, Genetic disorder, Food

“The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.

Key Points: 
  • “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.
  • Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
  • The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
  • The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, February 26, 2024
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, PKD, Personalized medicine, EMA, Research, MAA, Investment, Marketing, Food, Arrhythmogenic cardiomyopathy, Circulation, BLA, PDUFA, Fanconi anemia, Conference, Safety, DCM, European Medicines Agency, Food and Drug Administration, Muscle weakness, Efgartigimod alfa, Pyruvate kinase deficiency, Patient, Disorder, Clinical trial, Medication, ACM, IND, AAV, CGMP, CMC, Priority review, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.
  • “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
  • Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
  • As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million.

Jasper Therapeutics Highlights Recent Accomplishments and Key Upcoming Milestones

Retrieved on: 
Friday, January 5, 2024
Omalizumab, Subcutaneous, CD117, Risk, IND, EMA, FA, Chairperson, Cold urticaria, Therapy, MDS, Chronic, Fanconi anemia, ASH, Annual general meeting, European Medicines Agency, Syndrome, CSU, SVP, Patient, Acute myeloid leukemia, Safety, AML, CTA, Pharmaceutical industry

REDWOOD CITY, Calif., Jan. 05, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) in mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), as well as lower to intermediate risk myelodysplastic syndromes (LR-MDS) and novel stem cell transplant conditioning regimens, today announced its recent accomplishments, including the authorization by the European Medicines Agency (EMA) of the Company’s Clinical Trial Applications (CTA) for its Phase 1b/2a trials of briquilimab in CIndU and CSU, and outlined its corporate priorities and anticipated milestones for 2024. Called SPOTLIGHT – “Study (Phase 1b/2a) Of subcuTaneous brIquilimab in patients diaGnosed with cHronic inducible urTicaria” – the CIndU study will evaluate single doses of subcutaneous briquilimab in adult patients with cold urticaria or symptomatic dermographism.

Key Points: 
  • “2023 was a strategically important year for Jasper,” said Ronald Martell, President and Chief Executive Officer of Jasper.
  • “We secured IND clearance and CTA authorization for the Phase 1b/2a BEACON study of briquilimab in CSU and successfully dosed the first patient.
  • Our achievements in 2023 set the stage for a transformational year ahead with multiple key clinical milestones on the horizon across multiple indications.
  • Strengthened the organization with key leadership appointments including Thomas Wiggans as Chairperson of the Board of Directors, Scott Brun, M.D.

Jasper Therapeutics Presents Positive Final Results from Phase 1 Study of Briquilimab in Patients with AML or MDS Undergoing Hematopoietic Cell Transplant in Oral Presentation at ASH 2023

Retrieved on: 
Sunday, December 10, 2023
TBI, Exposition, Risk, CD117, Division, MRD, Chimera, Haematopoietic system, FA, Pediatrics, Therapy, MDS, Fanconi anemia, Transplant, ASH, RFS, HCT, NRM, Syndrome, CSU, Assistant, GVHD, Acute myeloid leukemia, Patient, Safety, AML, Pharmaceutical industry

The study enrolled 32 adult patients (62 to 79 years) with AML in CR (n=13), MDS (n=16), or AML not in CR (n=3).

Key Points: 
  • The study enrolled 32 adult patients (62 to 79 years) with AML in CR (n=13), MDS (n=16), or AML not in CR (n=3).
  • Peripheral blood grafts were infused on TD0 (10-14 days after briquilimab).
  • Further, briquilimab dosing resulted in predictable pharmacokinetics and allowed donor cell infusion 9-14 days after administration, and all patients who engrafted achieved neutrophil recovery before TD +26.
  • The 2a portion of the study is ongoing and continues to enroll patients.

Rocket Pharmaceuticals Appoints R. Keith Woods to Board of Directors

Retrieved on: 
Tuesday, December 12, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Efgartigimod alfa, Senior, Disorder, Florida State University, Hematology, Patient, Eisai, Alexion Pharmaceuticals, Marketing, FA, Muscle weakness, Roche, R, Fanconi anemia, Medication, Amgen, Pharmaceutical industry, Management, Eculizumab

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 the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.

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 the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.
  • Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain.
  • “I’m thrilled to welcome Keith to our Board of Directors, who brings deep industry expertise and invaluable experience to the Rocket Board as we build upon our commercial and operational infrastructure to support our anticipated first product launches for severe Leukocyte Adhesion Deficiency (LAD-I) and Fanconi Anemia (FA),” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Earlier in his career, Mr. Woods held commercial and sales positions of increasing responsibility at Roche, Amgen and Eisai.

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

Retrieved on: 
Thursday, November 9, 2023
CSU, FDA, Research, Trial of the century, IND, Anemia, CD117, Syndrome, Fanconi anemia, Risk, Chairperson, Hives, Stanford University, Omalizumab, Therapy, Patient, Pharmaceutical industry, Cryptocurrency, Jasper, EU

We also continued to strengthen our board of directors and senior leadership team with multiple key additions during the period.

Key Points: 
  • We also continued to strengthen our board of directors and senior leadership team with multiple key additions during the period.
  • Jasper expects to enroll the first patient by the end of 2023 and to report interim data on multiple cohorts by mid-2024.
  • Jasper continued to strengthen the organization with the appointment of Thomas Wiggans as Chairperson of the Board of Directors and Herb Cross as Chief Financial Officer.
  • Jasper reported a net loss of $17.5 million, or basic and diluted net loss per share attributable to common stockholders of $0.16, for the three months ended September 30, 2023.

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.

Jasper Therapeutics to Present New Positive Data on Briquilimab Conditioning in Patients with Fanconi Anemia at the 2023 Fanconi Anemia Research Fund Scientific Symposium

Retrieved on: 
Thursday, September 21, 2023
Pediatrics, Definitive, Clinical trial, Stanford University, Fanconi anemia, Haematopoietic system, MDS, Bone marrow failure, Patient, Syndrome, Risk, Stanford University School of Medicine, Toxic epidermal necrolysis, FA, Therapy, CD117, Pharmaceutical industry, Jasper

The data will be featured in a presentation at the 2023 Fanconi Anemia Research Fund (FARF) Scientific Symposium, taking place on September 28 – October 1, 2023, in Vancouver, Canada.

Key Points: 
  • The data will be featured in a presentation at the 2023 Fanconi Anemia Research Fund (FARF) Scientific Symposium, taking place on September 28 – October 1, 2023, in Vancouver, Canada.
  • The ongoing investigator initiated Phase 1/2 clinical trial is evaluating a conditioning regimen that includes briquilimab as a potential treatment for FA patients in bone marrow failure.
  • “All three FA patients who underwent conditioning with briquilimab achieved full blood count recovery.
  • These outcomes underscore the potential for briquilimab conditioning regimen to redefine the landscape of FA therapy.

Celebrating Art for a Cause: St. Jude Children’s Research Hospital Patient Artwork Featured During 2023 FedEx St. Jude Championship, Available in Fan Shop and Charity Auction

Retrieved on: 
Monday, August 7, 2023
Managed Care, Sports, Family, Philanthropy, Oncology, Consumer, Golf, Hospitals, Foundation, Children, Other Philanthropy, Health, Fanconi anemia, Survival rate, Travel, Housing, Food, Life, FedEx, Parent, NCI-designated Cancer Center, ALSAC, Bone marrow, Osteosarcoma, Cancer, Hope, National Cancer Institute (Egypt), Multimedia, TPC Southwind, Creativity, National Cancer Institute, Patient, Acushnet Company, Child, Love, Blood, St. Jude Championship, PGA, Hospital, Mental health, Birdy, 2021 WGC-FedEx St. Jude Invitational, Nursing, Jude the Apostle

A philanthropic tradition continues as art by St. Jude Children’s Research Hospital® patients will be featured throughout the 2023 FedEx St. Jude Championship , Aug. 10-13 at TPC Southwind.

Key Points: 
  • A philanthropic tradition continues as art by St. Jude Children’s Research Hospital® patients will be featured throughout the 2023 FedEx St. Jude Championship , Aug. 10-13 at TPC Southwind.
  • View the full release here: https://www.businesswire.com/news/home/20230807454741/en/
    A philanthropic tradition continues as art by St. Jude Children’s Research Hospital® patients will be featured throughout the 2023 FedEx St. Jude Championship, Aug. 10-13 at TPC Southwind.
  • Saving children.®
    In addition, select items featuring St. Jude patient art are now up for bid through Aug. 13 on the Birdies for St. Jude online auction.
  • “St.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy Programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Friday, May 19, 2023
Health, Pharmaceutical, Biologics license application, PVC, LV, Hemolysis, Gene, ACM, Arrest, Survival, Bone marrow, Monogenic, Quality of life, PKD, Liver injury, Research, Arrhythmia, AAV, Fanconi anemia, Disorder, Pyruvate kinase deficiency, Natriuresis, Medication, First, MMC, HIV disease progression rates, Patient, Arrhythmogenic cardiomyopathy, Recombinant, Busulfan, Hematology, Disease, Cell, Kaplan–Meier estimator, Toxicology, Danon disease, Pivotal, Mortality, FA, FDA, BLA, IND, Society, Safety, Pharmaceutical industry, Medical device, Nursing, Birth control, Vaccine, PKP2

“I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.

Key Points: 
  • “I am pleased that we continue to deliver outstanding clinical results that highlight the momentum across both our LV hematology and AAV cardiovascular platforms,” said Jonathan Schwartz, M.D., Chief Gene Therapy Officer, Rocket Pharma.
  • Both adult patients reported improved quality of life with documented improvements via formal quality of life assessments.
  • The safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.
  • Rocket is initiating Phase 1 study start-up activities and rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic.