Hemophilia B Mouse - Molecular Innovations, Inc.
Molecular Innovations would like to introduce our hemophilia B mouse model. This model was created by targeted disruption of Factor IX using programed nuclease. Mutations are confirmed by PCR genotyping, Sanger sequencing, and RT-PCR. Male hemizygotes display severe hemophilia B phenotype with prolonged blood clotting time and exhibit death after tail clipping without cauterizing. This hemophilia B mouse model will be useful for studying gene therapy methods.. Our hemophilia B mice are from an ongoing colony made in-house at our state of the art facility in the U.S.A and available immediately. No waiting for cryo recovery or costly breeding.. DETAILS. Strain background: C57BL/ ...http://mol-innov.com/products/hemophilia-b-mouse/
Hemophilia B Pipeline Review H2 2016: Stage of Development and Molecule Type Analysis | Business
Before It's News). Latest Hemophilia B Pipeline Review, H2 2016, provides in depth analysis on Cellular Tumor Antigen P53 (Tumor Suppressor P53 or Antigen NY-CO-13) targeted pipeline therapeutics.. Hemophilia B therapeutics industry report provides comprehensive information on the therapeutics under development for Hemophilia B, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Hemophilia B and features dormant and discontinued projects.. Browse more detail information about Hemophilia B market report at: ...http://beforeitsnews.com/business/2016/11/hemophilia-b-pipeline-review-h2-2016-stage-of-development-and-molecule-type-analysis-3032835.html
Haemophilia B - Wikipedia
Haemophilia B (or hemophilia B) is a blood clotting disorder caused by a mutation of the factor IX gene, leading to a deficiency of factor IX. It is the second-most common form of haemophilia, rarer than haemophilia A. Haemophilia B was first recognized as a different kind of haemophilia in 1952. It is sometimes called Christmas disease, named after Stephen Christmas, the first patient described with this disease. In addition, the first report of its identification was published in the Christmas edition of the British Medical Journal. The presentation of hemophilia B is consistent with easy bruising, urinary tract bleed and nosebleeds. Additionally, the affected individual may experience bleeding into their joints. The factor IX gene is located on the X chromosome (Xq27.1-q27.2). It is an X-linked recessive trait, which explains why, as in haemophilia A, usually only males are affected. In 1990, George Brownlee and Merlin Crossley showed that two sets of genetic mutations ...https://en.wikipedia.org/wiki/Haemophilia_B
... is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding or oozing following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor trauma or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms may not become apparent until abnormal bleeding occurs following surgery or a serious injury. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood but have few bleeding problems after puberty. Hemophilia B is inherited in an X-linked recessive pattern and is caused by mutations in the F9 gene ...http://diseaseinfosearch.org/Hemophilia+B/3315
... is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding or oozing following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor trauma or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms may not become apparent until abnormal bleeding occurs following surgery or a serious injury. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood but have few bleeding problems after puberty. Hemophilia B is inherited in an X-linked recessive pattern and is caused by mutations in the F9 gene ...http://diseaseinfosearch.org/Hereditary+factor+IX+deficiency+disease/3315
CHAPTER 123 HEMOPHILIA A AND HEMOPHILIA B | Free Medical Textbook
CHAPTER 123 HEMOPHILIA A AND HEMOPHILIA B Williams Hematology CHAPTER 123 HEMOPHILIA A AND HEMOPHILIA B HAROLD R. ROBERTS MAUREANE HOFFMAN Hemophilia A (Classic Hemophilia, Factor Viii Deficiency) Definition and History Etiology and Pathogenesis Genetics Prenatal Diagnosis and Carrier Detection Clinical Features Laboratory Features Differential Diagnosis Therapy Course and Prognosis Hemophilia B (Factor IX Deficiency,…https://medtextfree.wordpress.com/2012/02/09/chapter-123-hemophilia-a-and-hemophilia-b/
Early safety results promising for Phase I/II trial of gene therapy treatment of hemophilia B - Healthcanal.com : Healthcanal...
Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.. This trial was designed primarily as a safety test, with low and intermediate doses of the vector expected to produce little detectable Factor IX. The Factor IX protein helps the blood form clots. Individual with hemophilia B lack adequate levels of this clotting factor. The first participant in the open-label Phase I/II trial had an unexpectedly high level of Factor IX expression after receiving the lowest dose of the vector being tested in this study. Levels of the Factor IX protein rose from less than 1 percent to 2 percent of normal after the experimental vector was infused into the patient.. The patient's Factor IX production remains elevated more than nine months later. Since the infusion the ...https://www.healthcanal.com/blood-heart-circulation/12949-early-safety-results-promising-for-phase-iii-trial-of-gene-therapy-treatment-of-hemophilia-b.html
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B - Full...
Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes. This clinical program will test a gene transfer approach involving the use of a gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable plasma FIX activity between 10% and 40% of normal activity ...https://clinicaltrials.gov/ct2/show/NCT01687608
Gene therapy found effective in hemophilia B » Haemophilia Foundation of New Zealand Inc.
In 10 patients with severe haemophilia B, the infusion of a single dose of gene therapy with an AAV8 vector resulted in long-term therapeutic factor IX expression associated with clinical improvement. With a follow-up period of up to 3 years, no late toxic effects from the therapy were reported.. A recently published study updates an earlier one from 2011, in which six volunteers were successfully treated with various doses of the treatment that uses a virus to insert genetic material into the liver. The four additional patients received the highest dose. Dr. Andrew Davidoff of St. Jude Children's Research Hospital in Memphis, Tennessee and his colleagues found that the higher the dose of the engineered virus, the more factor IX the body produced. The researchers used an adeno-associated virus or AAV, which infects the liver without causing apparent illness.. The therapy seemed to have no serious side effects and is designed to eliminate the need to give patients injections of factor IX. The ...http://www.haemophilia.org.nz/community/news/gene-therapy-found-effective-in-hemophilia-b/
Biogen Idec seeks FDA approval for a new therapy that treats hemophilia B - The Boston Globe
Weston-based Biogen Idec Inc. has applied to the Food and Drug Administration for approval to market a new therapy to treat hemophilia B, the biotechnology company said Friday. Hemophilia B is a rare inherited disease that impairs patients' blood from clotting and can cause bleeding, joint damage and life-threatening hemorrhages. Biogen Idec said it submitted its biologics license application to the FDA late in the fourth quarter and expects a decision some time in 2013http://www.bostonglobe.com/business/2013/01/04/biogen-idec-seeks-fda-approval-for-new-therapy-that-treats-hemophilia/RrBu2KO3ewkhQEECw4KiQI/story.html
IDELVION for Hemophilia B Receives U.S. FDA Approval
IDELVION, the first & only hemophilia B therapy with up to 14-day dosing intervals, receives U.S. FDA approval. Learn about IDELVION for hemophilia B here.http://www.cslbehring.com/s1/cs/enco/1255923338532/news/1252902316508/prdetail.htm?tabSelections=1255923338644¤tPage=1
Paper: Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study
Introduction Despite the advent of extended half-life factor IX (FIX) products with the potential for 14 days or longer prophylactic dosing in patients with severe hemophilia B, long-term data on the use of ≥14-day dosing are lacking for prolonged periods of observation. A post hoc analysis of the B-LONG Phase 3 trial showed that ~50% of subjects in the individualized interval prophylaxis treatment arm (Arm 2, n=29) used extended dosing intervals (≥14 days) and had low annualized bleed rates (ABRs) that were similar to ABRs in subjects on other dosing intervals in Arm 2.1. The purpose of this analysis was to evaluate whether sustained ≥14-day dosing with recombinant FIX Fc fusion protein (rFIXFc) can provide safe and effective protection from bleeding in selected patients with severe hemophilia B over time in a near real-world setting. Methods B-LONG (NCT01027364) enrolled 123 adults and adolescents ≥12 years of age into 1 of 4 treatment arms: weekly prophylaxis (50 IU/kg ...https://ash.confex.com/ash/2017/webprogram/Paper102683.html
Groundbreaking Gene Therapy Drastically Reduces Bleeding Episodes In Hemophilia B Patients - Home Healthcare Austin
Home Health Care, which includes physical, occupational and speech therapies, as well as nursing, encompasses a broad spectrum of health and social services for recovering, disabled or chronically ill persons... ...http://homehealthcareaustin.com/groundbreaking-gene-therapy-drastically-reduces-bleeding-episodes-in-hemophilia-b-patients/
Ipsen's Partner Announces Hold Of Phase III Trials Of IB1001 For Hemophilia B
Ipsen's (IPSEF.PK) partner, Inspiration Biopharmaceuticals Inc., was notified by the Food and Drug Administration or FDA that both studies evaluating the safety and efficacy of IB1001, an investigational intravenous recombinant factor IX (rFIX) therapy for the treatment and prevention of bleeding episodes in people with hemophilia B, were placed on clinical hold.http://www.rttnews.com/1919496/ipsen-s-partner-announces-hold-of-phase-iii-trials-of-ib1001-for-hemophilia-b.aspx?type=qf
Repositorio da Producao Cientifica e Intelectual da Unicamp: RNA-based gene therapy for haemophilia B
Haemophilia B, a deficiency in clotting factor IX (FIX), occurs in about I in 25.000 males. Of these patients, approximately 40% are characterized as having 'severe' haemophilia (FIX below 1% of normal). Although the use of plasma-derived or rFIX has extended the lifespan of these patients, they remain afflicted by a variety of sequelae of the disease. Interestingly, a relatively small increase in the levels of FIX results in a dramatic increase in the quality of life, requiring rFIX substitution only prior to dental and surgical procedures. This latter trait of the disease suggests that gene therapy only needs to achieve modest expression and makes this disease a prime candidate for proof-of-concept of gene therapy. Current protocols for gene therapy entail the risk of malignant transformation. In addition the viral vectors may cause substantial pathology. Hence, the field requires the development of inherently safe gene therapy. Recently, developed protocols have been developed to transduce ...http://repositorio.unicamp.br/jspui/handle/REPOSIP/71153
Hemophilia B (Factor IX Deficiency) - Congenital
Neurology news, research and treatment studies for epilepsy, neurodegenerative disorders, patients with MS and other brain and central nervous system disorders and diseases.http://www.neurologyadvisor.com/labmed/hemophilia-b-factor-ix-deficiency-congenital/article/612701/
Paper: Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal...
Background: Data from earlier hemophilia B (HB) AAV-mediated liver gene transfer trials demonstrated a dose-dependent, capsid-specific immune response that may result in clearance of transduced hepatocytes and loss of transgene expression (Manno et al. 2006, Mingozzi et al. 2007). This has not posed major safety concerns, but may limit efficacy. Prior work incorporated the use of steroids to abort this immune response and maintain factor IX (FIX) expression (Nathwani et al 2014). The percent of transgene expression lost increased with number of elapsed days from transaminase elevation to steroid initiation. Once vector responsiveness to steroids is established, the need for steroids is not a limitation in and of itself but highlights the requirement to reliably and expeditiously recognize an immune response and initiate steroids. Here we present our immunomonitoring data following infusion of SPK-9001, an AAV vector designed to achieve therapeutic FIX:C at a low vector dose to minimize ...https://ash.confex.com/ash/2017/webprogram/Paper106110.html
UC San Diego Health Joins National Clinical Trial on Hemophilia B Gene Therapy
The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B.https://health.ucsd.edu/news/releases/pages/2016-03-22-clinical-trial-for-hemophilia-b-gene-therapy.aspx
New Method To Detect Antibodies Linked To Inhibitor Development - CIBD | Center For Inherited Blood Disorders
In an article release by the CDC (Center for Disease Control and Prevention), research reports key findings related to the formation of inhibitors (antibodies) in patients who have hemophilia. The study shows that hemophilia A and hemophilia B patients who develop inhibitors are likely to have a specific type of antibody called immunoglobulin G subclass 4, or IgG4. In the event that traditional inhibitor tests do not provide clear results, testing for these antibodies instead may help clarify whether an inhibitor is present.. Inhibitors are antibodies (protein) that prevents or stops infused factor from working. For those with bleeding disorders like hemophilia A and hemophilia B, inhibitors often develop because the body's immune system identifies the clotting factor as foreign. A blood test called Nijmegen-Bethesda Assay (NBA) is used to diagnose inhibitors.. For some cases, NBA can produce an unclear result as to whether ...http://cibd-ca.org/new-method-to-detect-antibodies-linked-to-inhibitor-development/
Lirias: Efficacy and safety of lentiviral versus adeno-associated viral vectors based on serotype 8 and 9 for hemophilia B gene...
Efficacy and safety of lentiviral versus adeno-associated viral vectors based on serotype 8 and 9 for hemophilia B gene therapy (Invited speaker ...https://lirias.kuleuven.be/handle/123456789/216789
Aptevo Therapeutics Presents New Clinical Data Evaluating the Safety and Efficacy of IXINITY in Children With Hemophilia B
SEATTLE, April 10, 2017 (GLOBE NEWSWIRE) -- Aptevo Therapeutics Inc. (Nasdaq:APVO), a biotechnology company focused on developing novel oncology and hematology therapeutics, today announced the presentation of new clinical data evaluating the safety and efficacy of IXINITY� [Coagulation Factor IX (Recombinant)] in previously treated patients under 12 years of age with Hemophilia B.� The data suggest that IXINITY appears to be safe and well tolerated in this subject population, and is comparable to the results from the overall patient population studied in the pivotal clinical trial of IXINITY.. �We�re encouraged to see data in a pediatric setting demonstrating the safety and efficacy of IXINITY in this important population,� said Scott Stromatt, Senior Vice President and Chief Medical Officer.� �The key efficacy outcomes from this study are comparable to what has previously been demonstrated in our pivotal study of IXINITY, which formed the basis for our licensure in the United ...http://it.tmcnet.com/news/2017/04/10/8525363.htm
Gene Therapy has made a huge progress tackling the hemophilia B problem - Long Short Report
Jay Konduros, the former aerospace engineer who has a blood disorder called hemophilia B, use to sprint home many times succeeding disparate times a year after coincidentally cutting and colliding himself. He would inject himself with refrigerated blood clotting element to avoid interior bleeding and substantial bruising and swelling.. Then he admitted himself in a clinical trial, sustaining an inventive gene therapy at Children's Hospital of Philadelphia. Almost instantly he commenced producing the missing clotting factor. After a few weeks he relinquished a heavy box on his shin, he observed an injury develop and recoil within hours.. Konduras, 53, who operates a bakery and a cafe in southeastern Ontario, is one of the 10 men receiving in an early-stage trial financed by Spark Therapeutics. On Wednesday researchers announced that an individual intravenous infusion of Spark's novel gene therapy empowered patients to securely produce adequate clotting factor to avoid hazardous bleeding ...http://longshortreport.com/2017/12/07/gene-therapy-made-huge-progress-tackling-hemophilia-b-problem/
FDA Approves Biogen Idec's ALPROLIX™, the First Hemophilia B Therapy to Reduce Bleeding Episodes with Prophylactic Infusions...
FDA Approves Biogen Idec's ALPROLIX™, the First Hemophilia B Therapy to Reduce Bleeding Episodes with Prophylactic Infusions Starting at Least a Week Aparthttp://www.tmcnet.com/usubmit/2014/03/28/7750672.htm
Catalyst Biosciences Announces Successful Completion Of First Subcutaneous Dosing Cohort In Ongoing Hemophilia B Clinical Trial...
Catalyst Biosciences Announces Successful Completion Of First Subcutaneous Dosing Cohort In Ongoing Hemophilia B Clinical Trial - read this article along with other careers information, tips and advice on BioSpacehttps://www.biospace.com/article/releases/catalyst-biosciences-announces-successful-completion-of-first-subcutaneous-dosing-cohort-in-ongoing-hemophilia-b-clinical-trial-/
Research and Markets: Germany Haemophilia B Market Highlights - 2014 | Business Wire
Research and Markets (http://www.researchandmarkets.com/research/gt8s8q/germany) has announced the addition of the 'Germany Haemophilia B Markethttp://www.businesswire.com/news/topix/20140611005616/en