Factor VIII

2seventy bio Announces Expanded Collaboration Agreement With Novo Nordisk to Continue Development of in vivo Gene Editing Approach

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Thursday, January 6, 2022
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, Haemophilia A, Blood, Organic acid, Vaccine, Megat Burhainuddin, Liver, RNA transfection, Vivo, Gene editing, Translation, Haemophilia, Research, Patient, LNP, DNA, Obesity, Goal, HES, Social media, Compte rendu, Bluebird bio, U.S. Securities and Exchange Commission, Twitter, Security (finance), Disease, Diabetes, TAL, NASDAQ, LinkedIn, Conditional sentence, FDA, Partnership, Genome, RNA, Sale, TIME, Multiple myeloma, Novo Nordisk, YouTube, Technology, Risk, CAR, Novo, Lists of diseases, Factor VIII, Pharmaceutical industry, Video game console, Carla Geneve, Facebook

2seventy bio, Inc. (NASDAQ:TSVT) today announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A.

Key Points: 
  • 2seventy bio, Inc. (NASDAQ:TSVT) today announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A.
  • We are also excited to announce a partnership between 2seventy bio and Genevant Sciences for the use of Genevants lipid nanoparticle (LNP) platform in our collaboration with Novo Nordisk.
  • The collaboration agreement with Novo Nordisk builds upon the original research collaboration signed between bluebird bio and Novo Nordisk in 2019, focused on identifying a development gene therapy candidate for people with hemophilia A.
  • Under the terms of the agreement, Novo Nordisk will obtain the option to exclusively license 2seventy bios in vivo mRNA platform and gene editing technology for use in the treatment of patients with hemophilia A.

Data from two Phase 3 studies demonstrating fitusiran significantly reduced bleeds in people with hemophilia A or B, with or without inhibitors, were featured at ASH’s plenary and late-breaking sessions

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Tuesday, December 14, 2021
Alnylam Pharmaceuticals, Injection, RNA, Pain, BPA, Doctor of Philosophy, Adult, Haemophilia A, Caregiver, Vaccine, Hemostasis, ABR, Thrombosis, Adolescence, Safety, Patient, Disease, Quality of life, Haemophilia, Bleeding, VIII, IX, ESC, Factor VIII, MD, Sanofi, ASH, Sirna, Health, Society, Thrombin, Pharmaceutical industry, Cryptocurrency

The Phase 3 studies compared once monthly fitusiran prophylaxis (80mg) with on-demand use of factor concentrates in the ATLAS-A/B study, and on-demand use of bypassing agents in the ATLAS-INH study.

Key Points: 
  • The Phase 3 studies compared once monthly fitusiran prophylaxis (80mg) with on-demand use of factor concentrates in the ATLAS-A/B study, and on-demand use of bypassing agents in the ATLAS-INH study.
  • There continues to be a significant need for transformative therapies that offer people with hemophilia consistent protection from bleeds while reducing treatment burden.
  • Study participants (n=120) were randomized 2:1 to receive either once-monthly 80mg subcutaneous fitusiran prophylaxis or on-demand factor therapy for bleeding episodes.
  • 65.8% (n=25) participants in the fitusiran prophylaxis arm had zero treated bleeds compared to 5.3% (n=1)) in the BPA on-demand arm.

Interim Data From Phase III HAVEN 6 Study Demonstrate Favorable Safety and Efficacy Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Moderate or Mild Hemophilia A

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Monday, December 13, 2021
Science, Other Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Clinical Trials, Hoffmann-La Roche, Phase, Headache, Poster, Blood, AE, Thrombotic microangiopathy, Protein, 1991 World Sportscar Championship, Ixa, Factor, TMA, AES, Population, Brain, TES, APCC, Woman, Thrombus, Pharmacokinetics, Lung, Head, ABR, TE, RO, Safety, Man, Patient, Leg, Exposition, Haemophilia, Ageing, Arm, VIII, Factor VIII, Bleeding, ASH, HAVEN, Skin, Society, ROG, Factor X, Pharmaceutical industry, Birth control, Genentech, Emicizumab, EU

We remain committed to working together with the hemophilia community as we further explore the efficacy and safety of Hemlibra in broader populations.

Key Points: 
  • We remain committed to working together with the hemophilia community as we further explore the efficacy and safety of Hemlibra in broader populations.
  • HAVEN 6 is a Phase III study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild hemophilia A without factor VIII inhibitors.
  • This interim analysis was conducted after 50 participants with moderate hemophilia A completed at least 24 weeks in the study or withdrew.
  • These data further confirm the favorable safety profile of Hemlibra, consistent with results from previous HAVEN and STASEY studies.

DGAP-News: Biotest AG: Biotest adjusts guidance

Retrieved on: 
Wednesday, December 8, 2021
Haemophilia, Plasma, Antibody, EBIT, Factor VIII, VIII, Blood product

Therefore, Biotest will only produce factor VIII in a sales-correlated manner in the future and make depreciations on existing raw material in the amount of approximately 38 - 45million.

Key Points: 
  • Therefore, Biotest will only produce factor VIII in a sales-correlated manner in the future and make depreciations on existing raw material in the amount of approximately 38 - 45million.
  • Accordingly, Biotest is correcting its EBIT guidance for 2021 from -5 to -10 million to approximately -43 to -55 million.
  • Biotest develops and markets immunoglobulins, coagulation factors and albumin based on human blood plasma.
  • The ordinary and preference shares of Biotest AG are listed in the Prime Standard on the German stock exchange.

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia A

Retrieved on: 
Monday, November 29, 2021
Patient, Security (finance), Mucopolysaccharidosis, CEO, Safety, Cell, Protein, Diabetes, Company, Haemophilia, Clinical trial, SEC, U.S. Securities and Exchange Commission, Factor VIII, GLOBE, NASDAQ, Massachusetts Institute of Technology, Conditional sentence, Biotechnology, Massachusetts, FDA, Observation, Flagship Pioneering, Therapy, Fibrosis, Enzyme, Medical device, Pharmaceutical industry

Upon inspection, it was determined that the spheres placed in the patient had fibrosed and that cells within the spheres were no longer viable.

Key Points: 
  • Upon inspection, it was determined that the spheres placed in the patient had fibrosed and that cells within the spheres were no longer viable.
  • While the Company investigates the fibrosed spheres in this patient, all three patients enrolled in the SIG-001 trial will continue to be followed per study protocol.
  • We are gathering information in order to understand these observations, said Rogerio Vivaldi, M.D., President and CEO of Sigilon.
  • Sigilon Therapeutics seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform.

Genentech to Present New Pivotal Data at ASH 2021 From Broad and Comprehensive Portfolio, Challenging Treatment Standards for People With Blood Disorders

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Tuesday, November 23, 2021
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, People, Patient, Birth, Bleeding, Disorder, Muscle, Arthralgia, Deformity, Vitamin, Needle, Haemophilia, APCC, Ixa, Headache, Blood, Pain, Breast milk, Haemophilia A, VIII, Biotechnology, Hematology, Antibody, FDA, Hoffmann-La Roche, Medicine, Factor VIII, Medication, Joint, Tenderness, Syringe, Hospital, Birth control, Pharmaceutical industry, Medical device, Polivy® (polatuzumab vedotin-piiq), Hemlibra® (emicizumab-kxwh), Hemophilia A, Genentech in Hematology, Genentech, POLIVY® (POLATUZUMAB VEDOTIN-PIIQ), HEMLIBRA® (EMICIZUMAB-KXWH), HEMOPHILIA A, GENENTECH IN HEMATOLOGY, GENENTECH

Females who are able to become pregnant should use birth control (contraception) during treatment with Hemlibra.

Key Points: 
  • Females who are able to become pregnant should use birth control (contraception) during treatment with Hemlibra.
  • Patients should tell their healthcare provider about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements.
  • Patients should keep a list of them to show their healthcare provider and pharmacist when they get a new medicine.
  • Today, were investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood.

Plasma Protein Therapeutics Global Market to 2026 - Insights and Forecast with Impact of COVID-19 - ResearchAndMarkets.com

Retrieved on: 
Thursday, November 11, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Factor VIII, Value, Industry, Therapy, PID, Growth, Antibody, Hemostasis, Application, Haemophilia, Awareness, Laboratory, Health policy, ITP, Blood product

The plasma protein therapeutics market is also driven by rising incidences of genetic disorder diseases worldwide.

Key Points: 
  • The plasma protein therapeutics market is also driven by rising incidences of genetic disorder diseases worldwide.
  • These factors are additionally fuelling the growth of the overall plasma protein market globally.
  • The report presents the analysis of plasma protein market for the historical period 2016-2020 and forecast period of 2021-2026.
  • Global Plasma Protein Market Segmentation - By Product, By Application

GeneVentiv Receives Orphan Drug Designation (ODD) for GENV-HEM for the Treatment of Hemophilia A or B with or without Inhibitors

Retrieved on: 
Tuesday, November 2, 2021
Orphan Drug Act of 1983, Haemophilia, Disease, Prescription Drug User Fee Act, Patient, FDA, Food, Hemostasis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FIX, Dawn of X, Haemophilia A, VIII, Marketing, CEO, FVA, Coronavirus Scientific Advisory Board (Turkey), Therapy, SAB, IX, ODD, AAV, Factor VIII, Pharmaceutical industry

With no approved gene therapies for hemophilia patients with inhibitors, we are dedicated to driving GENV-HEM forward as the first gene therapy for both hemophilia A or B with or without inhibitors," said Damon Race, CEO of GeneVentiv.

Key Points: 
  • With no approved gene therapies for hemophilia patients with inhibitors, we are dedicated to driving GENV-HEM forward as the first gene therapy for both hemophilia A or B with or without inhibitors," said Damon Race, CEO of GeneVentiv.
  • Hemophilia is an inherited bleeding disorder caused by a deficiency of the functional clotting factor (F) VIII (hemophilia A) or FIX (hemophilia B).
  • Approximately 2030%of patients with severe hemophilia A and up to 10% of patients with severe hemophilia B develop inhibitors after protein replacement therapy.
  • GENV-HEM is the first gene therapy treatment in development to treat both hemophilia A or B with or without inhibitors.

Acquired Hemophilia Treatment Market 2021 to 2029: Increasing Prevalence of AH among New Born Demands Effective Treatment - ResearchAndMarkets.com

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Friday, October 1, 2021
Health, Pharmaceutical, Adoption, Rheumatoid arthritis, Mortality, Ferring Pharmaceuticals, FastTrack, Muscle, Neoplasm, Research, Genentech, CAGR, Biogen, Cancer, Patient, GlaxoSmithKline, Pregnancy, AH, Development, Novo Nordisk, Bristol Myers Squibb, CSL Behring, VIII, Antibody, Psoriasis, Growth, Prevalence, Awareness, Key, Pfizer, Research and development, Factor VIII, Ulcerative colitis, Bleeding, Haemophilia B, Haemophilia, Drug discovery, Haemophilia A, EMA, Skin, Bayer, Disorder, Marketing, Birth control, Pharmaceutical industry

The "Acquired Hemophilia Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2021 to 2029" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Acquired Hemophilia Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2021 to 2029" report has been added to ResearchAndMarkets.com's offering.
  • The Acquired Hemophilia Treatment Market is anticipated to grow at a compound annual growth rate of 3.3% for the forecast period of 2021 to 2029.
  • The body produces inhibitors (antibodies) that target clotting factors, most commonly factor VIII, in Acquired Hemophilia.
  • On the basis of regional segmentation, Acquired Hemophilia Treatment Market includes North America, Latin America, Europe, Asia Pacific, Middle East and Africa, and Rest of the World.

Centessa Pharmaceuticals Announces Positive Topline Data from Proof-of-Concept Study of SerpinPC in Severe Hemophilia A and B Patients Not on Prophylaxis

Retrieved on: 
Thursday, September 9, 2021

CAMBRIDGE, Mass. and LONDON, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (“Company”) (Nasdaq: CNTA), together with subsidiary ApcinteX Limited (“ApcinteX”), today announced positive topline results from the Phase 2a part of AP-0101, the six-month repeat dose portion of its ongoing first-in-human proof-of-concept study evaluating SerpinPC in severe hemophilia A and B patients.

Key Points: 
  • All subjects had target joints at the start of the study and 15 subjects had zero target joints at the end of the study.
  • Centessa expects to report results from the OLE portion of this study in the second half of 2022.
  • We see broad utility of SerpinPC across the hemophilia landscape and will seek the most rapid path to bring this potential subcutaneous therapy to hemophilia patients, said Antoine Yver, M.D., M.Sc., Chief Medical Officer of Centessa Pharmaceuticals.
  • Centessa Pharmaceuticals will host a webcast and conference call today, September 9, 2021, at 8:30 a.m. EDT to discuss topline data from the proof-of-concept trial.