Pyruvate kinase deficiency

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

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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.

Agios Reports Business Highlights and Third Quarter 2023 Financial Results

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Thursday, November 2, 2023
Anti-transglutaminase antibodies, Research, RISE, PKU, Research and development, IND, AGIO, Rare, Sickle cell disease, MDS, Public expenditure, Enrollment, SG, Pyruvate kinase deficiency, R, Society, Phenylketonuria, ASH, Thalassemia, PK, Security, Abstract, Phenylalanine hydroxylase, TMPRSS6, Agios Pharmaceuticals, PAH, Agios, Patient, Administration, Nursing, Cryptocurrency, Pharmaceutical industry, Medical device, Video game, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023.
  • A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023.
  • Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
  • ET to discuss third quarter 2023 financial results and recent business activities.

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

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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.

Agios Appoints Catherine Owen to Board of Directors

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Thursday, May 25, 2023
University, Pyruvate kinase deficiency, PK, Thalassemia, Gastroenterology, Marketing, Multiple myeloma, Health care, Ustekinumab, Lung cancer, Bristol Myers Squibb, Pharmacy, Royal Pharmaceutical Society of Great Britain, Immunology, Ulcerative colitis, Kidney, AGIO, Dermatology, Patient, Syndrome, Rheumatology, Janssen Pharmaceuticals, Sickle cell disease, Royal Pharmaceutical Society, Bone marrow, RNA interference, Johnson & Johnson, Agios Pharmaceuticals, Rare, Journal of Medical Marketing, Pharmaceutical industry, Alnylam Pharmaceuticals, Infliximab

CAMBRIDGE, Mass., May 25, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that Catherine Owen has been appointed to its board of directors, effective June 13, 2023. Agios also announced that, following a collaborative succession planning process, John Maraganore, Ph.D., who has served as a valuable board member and advisor to the company since 2011, will step down from the board of directors, effective immediately, and that Kaye Foster, who has served as a member of the board of directors since 2014, will assume the role of lead independent director.

Key Points: 
  • CAMBRIDGE, Mass., May 25, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that Catherine Owen has been appointed to its board of directors, effective June 13, 2023.
  • Agios also announced that, following a collaborative succession planning process, John Maraganore, Ph.D., who has served as a valuable board member and advisor to the company since 2011, will step down from the board of directors, effective immediately, and that Kaye Foster, who has served as a member of the board of directors since 2014, will assume the role of lead independent director.
  • “We would also like to express our gratitude to John for nearly a dozen years of dedicated service to our board.
  • “I am excited about the future for Agios, and I look forward to continued progress in the years to come.”

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

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Tuesday, May 23, 2023
Biotechnology, FDA, Pharmaceutical, Health, LV, Doctor of Pharmacy, Drug development, Hemolysis, Gene, Prevalence, Quality of life, PKD, MBA, Disorder, Pyruvate kinase deficiency, Medication, Breakthrough therapy, Patient, Anemia, Cell, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, BLA, Society, Food, Disease, 21st Century Cures Act, Pharmaceutical industry, Medical device

The designation will provide the benefits of added FDA guidance and expedited review through the program’s development.

Key Points: 
  • The designation will provide the benefits of added FDA guidance and expedited review through the program’s development.
  • “Receiving RMAT designation from the FDA for RP-L301 is a major achievement in our pursuit to bring the first, potentially curative gene therapy treatment to patients living with PKD who have high unmet need.
  • The first pediatric patient has shown promising initial results similar to the adults, and enrollment has been completed in the Phase 1 study.
  • In addition to RMAT, RP-L301 has also received Fast Track and Orphan Drug Designation.

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)

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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.

Agios to Present Clinical and Translational Data at European Hematology Association Annual Congress

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Thursday, May 11, 2023
Abstract, RBC, CDL, Pyruvate kinase deficiency, Pharmacy, University, Iron overload, European Hematology Association, Vall d'Hebron University Hospital, Hemolysis, University Medical Center, Effect, Sickle cell disease, Department of Medicine – University of Pamplona, Rated R, Teaching hospital, Harvard Medical School, AGIO, Hematologic disease, Congress, University of Milan, Pyruvate kinase, University Medical Center Utrecht, Translational research, Treatment, University of Verona, CEST, EHA, Utrecht University, Red blood cell, Clinical, Patient, Agios Pharmaceuticals, Rare, Palacký University Olomouc, Myelodysplastic syndrome, Fondazione Child, Dietary supplement, Pharmaceutical industry, Nursing, Doctor of Philosophy, Cancer

CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the company and its collaborators will present a broad range of clinical and translational data at the European Hematology Association (EHA) Hybrid Congress, hosted virtually and in person in Frankfurt, Germany, June 8-11, 2023, and continuing virtually on the Congress platform June 14-15, 2023.

Key Points: 
  • – Agios to Present New Analyses from ACTIVATE, ACTIVATE-T and Long-Term Extension Studies in Adults with PK Deficiency Reinforcing Clinical Benefit of PYRUKYND® (mitapivat) on Hemoglobin, Hemolysis, Iron Overload and Patient-Reported Outcomes –
    CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the company and its collaborators will present a broad range of clinical and translational data at the European Hematology Association (EHA) Hybrid Congress, hosted virtually and in person in Frankfurt, Germany, June 8-11, 2023, and continuing virtually on the Congress platform June 14-15, 2023.
  • The accepted abstracts are listed below and are available online on the EHA meeting library website.
  • All posters will be presented during the poster session on Friday, June 9 at 16:30-17:45 CEST.
  • All presentations can be accessed on demand by registered meeting attendees on the EHA Virtual Congress platform until Aug. 15, 2023.

Agios Launches “Red Cell Revolution” in Recognition of Rare Disease Day

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Tuesday, February 28, 2023
Human, Sickle cell disease, Anemia, University, Rare, Hemolytic anemia, AGIO, Disease, Patient, Agios Pharmaceuticals, Hope, University of Connecticut Health Center, Thalassemia, Pyruvate kinase deficiency, Head, Caregiver, Just, PK, Rare disease, Physician, Pharmaceutical industry, Nursing

CAMBRIDGE, Mass., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced the launch of a multi-stakeholder advocacy advisory council for hemolytic anemias including PK deficiency, thalassemia and sickle cell disease.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced the launch of a multi-stakeholder advocacy advisory council for hemolytic anemias including PK deficiency, thalassemia and sickle cell disease.
  • “The Red Cell Revolution advisory council is designed to build connections among PK deficiency, thalassemia and sickle cell disease advocates and thought leaders, and to generate insights to raise disease awareness, inform Agios’ mission and contribute to shaping the broader hemolytic anemia field through evidence-based patient advocacy,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios.
  • I’m grateful to Agios for spearheading this initiative and facilitating these connections which can fuel positive changes for people with sickle cell disease, thalassemia and PK deficiency.”
    To learn more about the Red Cell Revolution, watch this video or listen to this podcast episode , which debuted yesterday across three Agios-sponsored podcasts, including Just Listen: Voices of PK Deficiency , Thal Pals: The Alpha Beta Revolution and Cheat Codes: A Sickle Cell Podcast .
  • The Agios team is also supporting rare disease communities throughout the month of February as a sponsor of the following initiatives:

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Retrieved on: 
Monday, February 27, 2023
Research, Other Health, General Health, Pharmaceutical, Oncology, Biometrics, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, Health, Other Science, CGMP, Fanconi anemia, Bone marrow, Cell, Acquisition, Lighting, Empire, Hemolysis, CD18, Regenerative Medicine Advanced Therapy, AAV, PKD, Novartis, BM, Danon disease, Senior, Biologics license application, Chief of Naval Staff (Pakistan), Patient, LV, Heart Failure Society of America, Rare disease, Pyruvate kinase deficiency, Transplant, Kaplan–Meier estimator, Schering-Plough, Center for International Blood and Marrow Transplant Research, Eli Lilly, Investment, Heart failure, Congress, Gene, Disease, MMC, Quality of life, Survival, LAMP2, PKP2, Disorder, DCM, BLA, FDA, ASH, Conference, FA, Medication, Society, Pharmaceutical industry, Medical device, Cryptocurrency, EU, G&A

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 reported financial and operational results for the fourth quarter and year ended December 31, 2022.

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 reported financial and operational results for the fourth quarter and year ended December 31, 2022.
  • The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022.
  • Based on the data from the Phase 2 pivotal Fanconi Anemia trial, Rocket anticipates submitting a BLA in the fourth quarter of 2023.
  • As of December 31, 2022, Rocket had cash, cash equivalents and investments of $399.7 million.

Remix Therapeutics Appoints Christopher Bowden, M.D., as Chief Medical Officer

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Thursday, January 5, 2023
Cancer, IDH, Hoffmann-La Roche, Providence, Pharmacia, CMO, Biotechnology, Therapy, Genentech, Lymphatic system, Pyruvate kinase, Janssen Pharmaceuticals, Disease, Roger Williams Medical Center, Patient, Providence VA Medical Center, Entrepreneurship, Internal medicine, Bristol Myers Squibb, University, AML, Large-cell lung carcinoma, Pyruvate kinase deficiency, BRAF, No. 90 (Signals) Group RAF, MYB, Oncology, RNA, EGFR, Pharmaceutical industry, Cholangiocarcinoma

CAMBRIDGE, Mass., Jan. 5, 2023 /PRNewswire/ -- Remix Therapeutics (Remix), a biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced the appointment of Christopher Bowden, M.D., as Chief Medical Officer.

Key Points: 
  • CAMBRIDGE, Mass., Jan. 5, 2023 /PRNewswire/ -- Remix Therapeutics (Remix), a biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced the appointment of Christopher Bowden, M.D., as Chief Medical Officer.
  • "We are especially pleased to welcome Chris to the Remix team as his wealth of strategic medical and corporate experience are ideally suited for this role.
  • We look forward to leveraging his extensive clinical expertise as we advance programs from our REMaster platform toward the clinic," said Peter Smith, Ph.D., Co-Founder and Chief Executive Officer of Remix Therapeutics.
  • Dr. Bowden joins Remix from Agios, where he served as a Strategic Advisor and Chief Medical Officer (CMO) from 2014-2022.