Hemolysis

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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星期五, 五月 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.

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

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星期二, 五月 16, 2023
Inhibitor, Neutralizing antibody, Hemolysis, Gene, NOD, MLD, PAH, Serum, SCID, Phenylketonuria, Trabecular meshwork, Gene editing, Gene expression, FIX, Publication, Metachromatic leukodystrophy, Messenger, PKU, Future, Administration, Immunosuppression, Genome, Intravitreal administration, Homologous recombination, Treatment, Vivisection, Immune system, PNH, Cell, Retinal pigment epithelium, NHP, Poster, RPE, Detection, Identification, Virus, ARSA, Society, Vaccine, Pharmaceutical industry, Truffle, Mouse, IND, C5

BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

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

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星期四, 五月 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.

Artificial intelligence identifies anti-aging drug candidates targeting ‘zombie’ cells

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星期四, 五月 4, 2023
Health, Biotechnology, Biomedical engineering, Nobel Prize, Nobel Prize in Chemistry, Institute for Medical Engineering and Science, Human, Broad Institute, McMillan, Nature, Hemolysis, Diabetes, Cardiovascular disease, Ageing, Cancer, Intelligence, Multimedia, Research, Medicine, MIT, Inflammation, Cell, Harvard University, Massachusetts Institute of Technology, Fibrosis, Biology, Machine learning, Apoptosis, Bâtiment des Forces motrices, Drug discovery, Conscience, Mouse, Artificial intelligence, AI, Longevity, Notifiable disease, Princeton University, Synthetic biology, Senolytic, Pharmaceutical industry, Science, List of Nature Research journals, Chemistry

View the full release here: https://www.businesswire.com/news/home/20230504005270/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230504005270/en/
    Senolytics are an emerging class of investigational drug compounds that selectively kill aging-associated senescent cells (left, with red stain) without affecting other cells (right).
  • Using artificial intelligence, researchers from Integrated Biosciences have, for the first time, identified three senolytics with comparable efficacy and superior drug-like properties relative to leading investigational compounds.
  • (Photo: Business Wire)
    “This research result is a significant milestone for both longevity research and the application of artificial intelligence to drug discovery,” said Felix Wong, Ph.D., co-founder of Integrated Biosciences and first author of the publication.
  • Integrated Biosciences was founded in 2022 to overcome these obstacles, target other neglected hallmarks of aging, and advance anti-aging drug development more generally using artificial intelligence, synthetic biology and other next-generation tools.

Omeros Corporation Reports Interim Data with Alternative Pathway Inhibitor OMS906 as Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria

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星期二, 四月 25, 2023
Health, Clinical Trials, General Health, Research, Pharmaceutical, Science, Myelodysplastic syndrome, Omeros, Hemolysis, Paroxysmal nocturnal hemoglobinuria, Half-life, AMD, Hemoglobin, Aplastic anemia, Clinical trial, LDH, Geographic atrophy, Multimedia, Disorder, Corporation, HGB, Iron deficiency, Patient, Physician, Risk, PNH, Hematology, Aristotle University of Thessaloniki, Observation, Kidney disease, Disease, Pharmaceutical industry, Surveying, C3

To date, all patients have received only the lowest subcutaneous dose of OMS906 in this multidose trial.

Key Points: 
  • To date, all patients have received only the lowest subcutaneous dose of OMS906 in this multidose trial.
  • This single-arm, open-label clinical trial is evaluating the effect of once-monthly subcutaneous administration of OMS906 in patients with PNH.
  • OMS906 is Omeros’ lead investigational humanized monoclonal antibody targeting MASP-3, the key and most upstream activator of the alternative pathway of complement.
  • “We are excited by the data resulting from this trial of treatment-naïve patients,” said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer.

Global Vacuum Blood Collection Tube Market Report to 2030: Rising Number of Blood Transfusions Drives Growth - ResearchAndMarkets.com

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星期三, 三月 29, 2023
Medical Devices, Health, Infection, Syringe, Disease, University, Hemolysis, Prevalence, Centrifugation, HIV, Diabetes, Serum, COVID-19, Injury, Pathology, Ageing, Cancer, Hepatitis B, Pathogen, Government, ASCP, Collection, Laboratory, Risk, Incineration, Hypokalemia, Anemia, Blood, Growth, WHO, Leukemia, Glass, Safety, BD, Substance abuse, Vaccine, Medical device, Pharmaceutical industry, Video game

Increasing prevalence of chronic and infectious diseases, a growing number of blood transfusions, coupled with rising hematological disorders are boosting the adoption of blood collection tubes.

Key Points: 
  • Increasing prevalence of chronic and infectious diseases, a growing number of blood transfusions, coupled with rising hematological disorders are boosting the adoption of blood collection tubes.
  • In addition, frequent disease outbreaks in endemic areas are anticipated to increase the demand for blood collection tubes for clinical diagnostics.
  • A blood test is performed for numerous indications, such as infection, anemia, hypokalemia, leukemia, and kidney function.
  • Vacuum tubes are used routinely for the collection of blood specimens in laboratory diagnostics, globally.

Babson Diagnostics and ClosedLoop Team Selected as 1st Place Winner of ‘Help with Hemolysis’ Data Analytics Challenge

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星期二, 三月 28, 2023
Other Health, Retail, Other Science, Artificial Intelligence, Medical Devices, Professional Services, Science, Technology, Clinical Trials, Managed Care, Medical Supplies, Other Communications, Data Analytics, Public Relations, Investor Relations, Marketing, Other Retail, Health, Communications, Hemolysis, AACC, Diagnosis, Intelligence, Washington University in St. Louis, Collection, Pathology, Machine learning, Informatics, Medical device, Babson

Babson Diagnostics, a science-first healthcare technology company, and ClosedLoop, the leading healthcare data science platform, today announced their team was selected as the first-place winner of the ‘ Help with Hemolysis ’ Data Analytics Challenge.

Key Points: 
  • Babson Diagnostics, a science-first healthcare technology company, and ClosedLoop, the leading healthcare data science platform, today announced their team was selected as the first-place winner of the ‘ Help with Hemolysis ’ Data Analytics Challenge.
  • Babson Diagnostics and ClosedLoop have worked together closely since 2021, when the two companies collaborated to develop artificial intelligence solutions, which help detect and prevent sample quality errors that can occur when working with capillary blood samples.
  • So it was a natural fit for the companies to team up to tackle the ‘Help with Hemolysis’ challenge.
  • “We felt uniquely positioned to solve this challenge with our partners at Babson Diagnostics.

Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for the Fourth Quarter and Full Year 2022

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星期四, 三月 9, 2023
Hemolysis, University of Cape Town, PRC2, Research, FDA, G&A, Telephone, University, FSHD, Fetal hemoglobin, Biomarker, Al-Abid, IND, Letter, Conference, Interim, REACH, Bone marrow, Cohort, Therapy, SCD, GSK plc, Risk, CMO, Communication, Pathology, Bachelor, Experimental pathology, Harvard University, Sickle cell disease, Conference call, Webcast, Collaboration, Merck & Co., Patient, Lundbeck, Surgery, Life insurance, Pharmaceutical industry, Cryptocurrency, Dietary supplement, Merck, MD, Bachelor of Medicine, Bachelor of Surgery, HBF, Agency, Medicine, Doctor of Philosophy, Fulcrum, Biochemistry, Science

The Agency requested that Fulcrum further define the population where the potential benefit of continued treatment with FTX-6058 outweighs potential risk.

Key Points: 
  • The Agency requested that Fulcrum further define the population where the potential benefit of continued treatment with FTX-6058 outweighs potential risk.
  • Collaboration Revenue: Collaboration revenue was $0.7 million for the fourth quarter of 2022 as compared to $5.1 million for the fourth quarter of 2021.
  • Net Loss: Net loss was $26.1 million for the fourth quarter of 2022, as compared to a net loss of $23.5 million for the fourth quarter of 2021.
  • ET to review the fourth quarter and full year 2022 recent business highlights and financial results.

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

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星期一, 二月 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.

SeqOne Genomics and the French Thrombotic MicroAngiopathies National Reference Center (CNR-MAT) Pioneer the Use of Oxford Nanopore Sequencing Technology to Improve Patient Outcomes in Kidney Disease While Reducing Turnaround Times

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星期四, 二月 9, 2023
Research, Nursing, Infectious Diseases, Genetics, Biotechnology, Health, Pharmaceutical, Science, Oncology, DNA, Nephrology, Thrombocytopenia, Diagnosis, Patient, DSM-IV codes, Microangiopathy, Oxford Nanopore Technologies, Thrombotic microangiopathy, Vitamin B12, Thrombosis, TMA, Nanopore sequencing, Chelsea Handler, Hypertension, Gene, Disease, Kidney, Prognosis, Multimedia, Oxford, Hemolysis, Genome, Comment (computer programming), Dietary supplement, Medical imaging, Eculizumab

Laurent Mesnard, Co-Director of the French National center for Thrombotic MicroAngiopathies (CNR-MAT), aimed at improving the diagnosis and management of patients suspected of suffering from thrombotic microangiopathies and atypical Hemolytic syndrome (aHUS).

Key Points: 
  • Laurent Mesnard, Co-Director of the French National center for Thrombotic MicroAngiopathies (CNR-MAT), aimed at improving the diagnosis and management of patients suspected of suffering from thrombotic microangiopathies and atypical Hemolytic syndrome (aHUS).
  • The program involved using Oxford Nanopore’s revolutionary technology to sequence patients at the bedside, dramatically reducing turnaround times to obtaining actionable insights.
  • This Oxford Nanopore-based analysis was compared with traditional short read sequencing approaches in order to establish a benchmark against current standards of care and hence quantify benefits of nanopore sequencing.
  • In order to support the program, SeqOne implemented an Oxford Nanopore compatible long-read support capable of managing adaptive sampling data.