CSL Behring, with the help of its parent, is developing rIX-FP for the prophylaxis and treatment of bleeds in patients with congenital Factor IX
(FIX) deficiency as part of the PROLONG-9FP clinical study programme.
Global biopharmaceutical group Ipsen (Paris:IPN.pa) (Euronext:IPN) announced today that its partner, Inspiration Biopharmaceuticals Inc, presented pharmacokinetic (PK) data on its lead product, IB1001, a recombinant factor IX
(FIX) for the treatment and prevention of bleeding in individuals with haemophilia B.
The firm said it is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX
gene therapy produced using its proprietary AAV production system.
This haemophilia B gene therapy, administered once, will introduce the functional gene for the Factor IX
protein into the patient's liver cells with the goal of restoring blood clotting functionality long-term.
The company said, "Earlier this month, the Company presented updated clinical data at the 2019 ISTH conference demonstrating that a single administration of AMT-061 resulted in sustained increases in Factor IX
levels up to 54% of normal, with a mean for the three patients in the Phase IIb study of 45% of normal at 36 weeks post administration.
Summary: Injection of coagulation factor IX
is the primary and widely adopted treatment option across the globe, which is further boosting the growth of global coagulation factor IX
[USPRwire, Thu May 09 2019] Coagulation factor IX
isn't made by the body of hemophilia B patient thereby, external injection of coagulation factor IX
is necessary for the treatment of such disorder.
(NYSE: PFE) has initiated a Phase 3 open-label, multi-center, lead-in study to evaluate the efficacy and safety of current factor IX
prophylaxis replacement therapy in the usual care setting, the company and its development partner Philadelphia, Pennsylvania-based gene therapies specialist Spark Therapeutics (NASDAQ: ONCE) said.
Hemophilia B is a congenital bleeding disorder characterised by deficient or defective factor IX
; nearly all affected patients are male.