Phase 3 Trial of Hemophilia A Gene Therapy SB-525 on FDA Hold
WEBNov 10, 2021 · Further trial updates from the companies are expected after the FDA’s review of the proposed protocol amendment. “We are committed to resuming patient dosing in this phase 3 program and believe this gene therapy, if approved, could represent an important treatment option for patients with hemophilia A,” …
Hemophilia A Patient Care Facilitated with Hemlibra Dosing …
WEBMay 24, 2019 · BrightInsight and Roche have launched a web-based tool to help physicians select the optimal loading and maintenance dose of Hemlibra for hemophilia A patients. Hemlibra is a prophylactic treatment that targets both factor IXa and factor X clotting factors and can reduce bleeding episodes.
DalcA Shows Sustained Efficacy in Hemophilia B in Trial
WEBJun 17, 2020 · DalcA is a lab-made version of the blood clotting protein FIX that works as a prophylactic treatment for bleeding episodes in people with severe hemophilia B. A Phase 2b trial found that DalcA provided protective FIX levels after 28 …
For Hemophilia A Without Inhibitors, Hemlibra Superior to Factor...
WEBAug 16, 2019 · The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial” was published in the journal Current Medical Research and Opinion. Hemlibra (by Roche) is a humanized monoclonal antibody approved in the U.S. as a routine preventive ...
First 2 Adults with Severe Hemophilia A Respond Well to Gene...
WEBDec 6, 2019 · The ongoing, dose-establishing Phase 1/2 trial (NCT03588299; 2017-000806-39) is evaluating the safety, tolerability and early effectiveness of three ascending doses of BAY 2599023 in adult men with severe hemophilia A who have been previously treated with FVIII products. It is the first clinical trial to evaluate a gene therapy based on the AAVhu37.
Trial of Hemophilia B Gene Therapy BBM-H901 Begins Dosing in …
WEBJan 5, 2022 · BBM-H901 is designed to deliver a working copy of F9 using a modified, harmless version of an adeno-associated virus (AAV) to liver cells, where blood clotting factors are produced. The gene therapy aims to increase FIX levels to prevent or control bleeding episodes and eliminate the need for regular treatments.
Obizur (susoctocog alfa) for hemophilia - Hemophilia News Today
WEBNov 22, 2023 · Obizur is a recombinant antihemophilic factor that can replace the clotting factor VIII (FVIII) that is inactivated by self-reactive antibodies in acquired hemophilia A patients. Learn how Obizur works, who can take it, how it is administered, and what side effects it may cause.
Hemophilia prognosis and life expectancy
WEBLearn how hemophilia severity, treatment, and complications affect life expectancy and quality of life. Find out the latest research and news on hemophilia care and prevention.
FDA OKs first-in-human study of F9 gene therapy for hemophilia B
WEBFeb 28, 2024 · Intellia and Regeneron are testing a CRISPR/Cas9-based F9 gene-editing therapy for people with hemophilia B, a rare bleeding disorder caused by F9 gene mutations. The therapy aims to insert a working version of the F9 gene into patient cells and restore clotting factor activity.
Mononine Soon Will No Longer Be Available to Treat Hemophilia B
WEBSep 21, 2020 · CSL Behring, the therapy’s manufacturer, said the decision was based on the emergence of next-generation hemophilia B treatments now in greater use. “Over time, patients have transitioned from older therapies to newer, next generation treatment options, and very few patients currently remain on MONONINE in the US,” CSL Behring wrote in a statement published on the Hemophilia Federation ...
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