Thrombotic microangiopathy

Oxford Nanopore Technologies and SeqOne Partner to Support Interpretation of Nanopore Sequencing in Clinical Use

Retrieved on: 
Montag, März 11, 2024
Health Technology, Technology, Nanotechnology, Pharmaceutical, Oncology, Professional Services, Genetics, Artificial Intelligence, Clinical Trials, Science, Biotechnology, Software, Data Analytics, Health, Research, WGS, Nephrology, APHP, STR, Single-nucleotide polymorphism, Partnership, CFH, Patient, Chelsea Handler, RUO, HPO, Oxford Nanopore Technologies, Thrombotic microangiopathy, Developed country, Nanopore sequencing, Thrombosis, CNV, Pharmaceutical industry

Designed to streamline whole genome variant interpretation at scale, SeqOne DiagAi (RUO) saves time and reduces costs by ranking, shortlisting, and suggesting causative variants with best-in-class accuracy.

Key Points: 
  • Designed to streamline whole genome variant interpretation at scale, SeqOne DiagAi (RUO) saves time and reduces costs by ranking, shortlisting, and suggesting causative variants with best-in-class accuracy.
  • It also enables one-click HPO extraction from clinical notes with the DiagAI Notes2HPO large language model.
  • In the future, the collaboration will deliver other variant interpretation applications addressing the needs of cancer predisposition and somatic analysis.
  • “We are excited to collaborate with SeqOne to provide end-to-end solutions for our customers in rare disease and oncology.

TECHNOCLONE RECEIVES US FDA MARKETING AUTHORIZATION FOR THE FIRST ADAMTS13 ACTIVITY DIAGNOSTIC TEST

Retrieved on: 
Dienstag, März 5, 2024
Doctor of Philosophy, MD, FDA, Food, De novo, ELISA, Thrombotic microangiopathy, TTP, Patient, TMA, CSO, Plasma, Diagnosis, Thrombotic thrombocytopenic purpura, Laboratory, ADAMTS13

WEST CHESTER, Ohio, March 5, 2024 /PRNewswire/ -- Technoclone Herstellung von Diagnostika und Arzneimitteln GmbH, world-leading producer of diagnostic tests, equipment, and research products in the field of blood clotting and its exclusive distributor in the USA, DiaPharma Group, Inc., are pleased to announce that the US Food and Drug Administration (FDA) granted De Novo marketing authorization for Technozym ADAMTS13 Activity ELISA.

Key Points: 
  • WEST CHESTER, Ohio, March 5, 2024 /PRNewswire/ -- Technoclone Herstellung von Diagnostika und Arzneimitteln GmbH, world-leading producer of diagnostic tests, equipment, and research products in the field of blood clotting and its exclusive distributor in the USA, DiaPharma Group, Inc., are pleased to announce that the US Food and Drug Administration (FDA) granted De Novo marketing authorization for Technozym ADAMTS13 Activity ELISA.
  • This is the first ADAMTS13 activity test authorized by the FDA.
  • The Technozym ADAMTS13 Activity assay is an enzyme-linked immunosorbent assay (ELISA) intended for the qualitative determination of ADAMTS13 activity in platelet poor human citrated plasma.
  • "In line with our commitment to provide ADAMTS13 diagnostics globally, we are excited about this milestone, having the first ADAMTS13 activity test granted with marketing authorization by the FDA", says Nikolaus Binder, MD, PhD, CSO of Technoclone.

Takeda’s ADZYNMA (ADAMTS13, recombinant-krhn) Approved by U.S. FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Retrieved on: 
Donnerstag, November 9, 2023
FDA, Health, Genetics, General Health, Pharmaceutical, Biotechnology, Migraine, Safety, Ohio State University, Toll, Food, Incidence, Abdominal pain, Upper respiratory tract infection, Headache, TTP, Disease, Hematology, Mortality, Internal medicine, Thrombotic thrombocytopenic purpura, Patient, Thrombocytopenia, Microangiopathic hemolytic anemia, Diarrhea, Nausea, Thrombotic microangiopathy, Dizziness, Vaccine, Pharmaceutical industry, ADAMTS13, Takeda

Takeda ( TSE:4502/NYSE:TAK ) today announced that the U.S. Food and Drug Administration (FDA) has approved ADZYNMA (ADAMTS13, recombinant-krhn) for the prophylactic and on-demand treatment of adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced that the U.S. Food and Drug Administration (FDA) has approved ADZYNMA (ADAMTS13, recombinant-krhn) for the prophylactic and on-demand treatment of adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).
  • “As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology.
  • “ADZYNMA provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favorable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies.
  • ADZYNMA is a recombinant form of the ADAMTS13 protein.

eGenesis Presents New Data on Long-Term Survival in Preclinical Kidney Xenotransplantation at American Transplant Congress 2023

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Montag, Juni 5, 2023
Xenotransplantation, Thrombotic microangiopathy, Tissue, Survivor, TGS, ATI, DNA, Immunology, Serum, Biotechnology, Gene editing, Survival, AMR, Cell, Antibody, Doctor of Philosophy, TG, Research, DSA, Harvard Medical School, Transplant, Chemistry, Sera, Protein, Public, Animal, MD, Plasma, Risk, Coagulation, Filtration, Hospital, ATC, Hokkaido University, SLA, Health, Safety, Pelvic floor dysfunction, Postdoctoral researcher, TMA, Medical device, Dentistry, Vaccine, Dietary supplement, Primate

CAMBRIDGE, Mass., June 05, 2023 (GLOBE NEWSWIRE) -- eGenesis, a biotechnology company developing human-compatible organs for the treatment of organ failure, will present new data during the American Transplant Congress (ATC) 2023, held June 3 – June 7, 2023 in San Diego, CA.

Key Points: 
  • CAMBRIDGE, Mass., June 05, 2023 (GLOBE NEWSWIRE) -- eGenesis, a biotechnology company developing human-compatible organs for the treatment of organ failure, will present new data during the American Transplant Congress (ATC) 2023, held June 3 – June 7, 2023 in San Diego, CA.
  • “The data we are presenting at ATC 2023 demonstrate eGenesis’ continued leadership in the field of xenotransplantation,” said Mike Curtis, Ph.D., CEO of eGenesis.
  • Oral Presentation: Genetically Modified Porcine Kidneys Expressing Human Transgenes Support >2-year Survival in Porcine Donor to Cynomolgus Macaque Recipient Xenotransplantation.
  • eGenesis scientists genetically engineered porcine donors to remove three known xenoantigens implicated in hyperacute rejection.

Tune in to Bloomberg to Watch Advancements on Saturday, April 15th at 8:00 PM ET

Retrieved on: 
Montag, April 10, 2023
MAHA, Advanced manufacturing, ANR, Deepfake, Thrombotic microangiopathy, Atmosphere, Hearing, Microangiopathic hemolytic anemia, Research, Carbon, Language, Capillary, Patient, Arteriole, Transport, NEO, TMA

JUPITER, Fla., April 10, 2023 /PRNewswire-PRWeb/ -- Advancements with Ted Danson is excited to announce the broadcast of an upcoming episode, scheduled to air on Saturday, April 15th at 8:00 pm ET.

Key Points: 
  • Viewers will learn how these microthrombi cause the widespread and varied end organ injury that can be seen in patients diagnosed with a TMA.
  • In addition, audiences will discover how improvements in real-time application technology are helping real estate buyers, agents, and developers.
  • Finally, the series will uncover how improvements in technology are promoting global unity by enabling shared cultural experiences across language barriers.
  • "We are excited to explore how technology continues to progress across several sectors today," said Sarah McBrayer, Creative Director for DMG Productions and the Advancements series.

Akari Therapeutics Announces Presentation of a Case Study from Phase 3 Part A Clinical Trial of Nomacopan in Pediatric HSCT-TMA as a Late-Breaker at The Transplantation & Cellular Therapy Tandem Meetings

Retrieved on: 
Dienstag, Februar 21, 2023
DSM-IV codes, Royal Manchester Children's Hospital, AML, PRES, CH50, Thrombocytopenia, Hope, HSCT, Viremia, LTB4, Diagnosis, Leukotriene B4, NHS foundation trust, Thrombotic microangiopathy, Maintenance, GVHD, Division, Hematopoietic stem cell transplantation, Patient, Hospital, Clinical trial, Inflammation, Center for International Blood and Marrow Transplant Research, TMA, University, Research, Therapy, Male, Hypertension, TCA, Death, University of Manchester, Thiotepa, End organ damage, Manchester University NHS Foundation Trust, Cytomegalovirus, System, Transplant, Society, Syndrome, Hot game, Rosenthal, CMV, Pharmaceutical industry, Dentistry, Pediatrics

The case study, Clinical Response to Nomacopan in the Pediatric HSCT-TMA Setting, was presented on Thursday February 16, at 5:45 pm ET at the Transplantation & Cellular Therapy Tandem Meetings in Orlando, Florida.

Key Points: 
  • The case study, Clinical Response to Nomacopan in the Pediatric HSCT-TMA Setting, was presented on Thursday February 16, at 5:45 pm ET at the Transplantation & Cellular Therapy Tandem Meetings in Orlando, Florida.
  • The patient received 7 granulocyte infusions peri-transplant as part of an experimental protocol to augment the graft-versus-leukemia effect.
  • His immediate post-transplant course was complicated by engraftment syndrome, acute gut graft-versus-host disease (GVHD) grade 3 and cytomegalovirus (CMV) viraemia.
  • Nomacopan was stopped for 3 days and restarted after the diagnosis was deemed to be unrelated to nomacopan treatment.

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

Retrieved on: 
Donnerstag, Februar 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.

Anti-Bb Antibody (NM8074) Receives US FDA Clearance to Start Clinical Trial in Naïve aHUS Patients (Rare Disease)

Retrieved on: 
Montag, Januar 30, 2023
Disease, AP, Food, Patient, BB, Efficacy, Acquisition, Thrombotic microangiopathy, Safety, CP, Phase, Complement, Hemolysis, Partnership, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Ahus, Anemia, C3b, Inflammation, Pharmaceutical industry, Eculizumab, Adult

CLEVELAND, Jan. 30, 2023 (GLOBE NEWSWIRE) -- NovelMed announced today that the U.S. Food and Drug Administration (FDA) cleared the Company’s investigational drug NM8074 to initiate an efficacy trial in patients with aHUS in coming months. NovelMed is advancing its anti-Bb antibody (NM8074) to treat patients with evidence of treatment-related resistance or complement-mediated relapses in aHUS, a rare disease with unmet need. NovelMed is a clinical-stage biopharma company focused on the development of humanized monoclonal antibody treatments for several complement-mediated disorders.

Key Points: 
  • NovelMed is advancing its anti-Bb antibody (NM8074) to treat patients with evidence of treatment-related resistance or complement-mediated relapses in aHUS, a rare disease with unmet need.
  • NovelMed is a clinical-stage biopharma company focused on the development of humanized monoclonal antibody treatments for several complement-mediated disorders.
  • NM8074 has recently completed a Phase I trial in healthy volunteers with no safety concerns.
  • NM8074 blocks the AP-mediated formation of C3b, an important molecule responsible for incurable anemia seen in both Soliris and Ultomiris treated patients.

Akari Therapeutics to Present at Biotech Showcase™ 2023

Retrieved on: 
Mittwoch, Januar 4, 2023
Akari, Biotechnology, Therapy, Inflammation, LTB4, Thrombotic microangiopathy, Autoimmunity, Leukotriene B4, C5, Seasonal adjustment, Geographic atrophy, Pharmaceutical industry

NEW YORK and LONDON, Jan. 04, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced that Akari President and CEO Rachelle Jacques will present a company overview at Biotech Showcase 2023 on Monday, January 9, 2023 at 9 am ET.

Key Points: 
  • NEW YORK and LONDON, Jan. 04, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced that Akari President and CEO Rachelle Jacques will present a company overview at Biotech Showcase 2023 on Monday, January 9, 2023 at 9 am ET.
  • The presentation will include progress highlights from Akari’s two priority pipeline programs: a Phase 3 clinical trial of nomacopan, a novel bispecific inhibitor of complement C5 and leukotriene B4 (LTB4), for the treatment of severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy, and a pre-clinical program investigating long-acting PAS-nomacopan as a potential treatment for geographic atrophy.

Interim Data From Phase III Study Presented at ASH 2022 Show Hemlibra (emicizumab-kxwh) Achieved Meaningful Bleed Control in Infants From Birth

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Sonntag, Dezember 11, 2022
Research, Parenting, Baby, Maternity, Biotechnology, General Health, Pharmaceutical, Consumer, Health, Science, Other Science, Intracranial hemorrhage, Phase, Arm, Leg, Lung, Ixa, Factor, Female, Thrombotic microangiopathy, Haemophilia, Inherited disorders of trafficking, Life, Protein, Skin, APCC, Society, TMA, ABR, Patient, Blood, RO, HAVEN, Parent, Bleeding, AE, Woman, World government, Quality of life, ROG, Caregiver, Clinical trial, VIII, Safety, Brain, Hoffmann-La Roche, Pharmacokinetics, World Federation of Hemophilia, Trauma, AES, ASH, Factor X, Risk, Birth control, Pharmaceutical industry, Emicizumab, Genentech

The new data were presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition taking place in New Orleans from December 10-13, 2022.

Key Points: 
  • The new data were presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition taking place in New Orleans from December 10-13, 2022.
  • Hemlibra provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies.
  • HAVEN 7 is a Phase III, multi-center, open-label study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of Hemlibra in infants with severe hemophilia A without factor VIII inhibitors.
  • Genentech also presented data from the European Haemophilia Safety Surveillance (EUHASS) database and the prospective observational ATHN 7 study at ASH 2022.