January 17, 2025
Be Biopharma has raised $92 million in series C financing to support the first in-human Phase 1/2 trial of BE-101, the company's B-cell therapy candidate for hemophilia B. The treatment is designed to use a patient's own immune B-cells, genetically modify them, and infuse them back into the patient.
January 10, 2025
The US FDA has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The therapy is a subcutaneously-delivered monoclonal antibody therapy designed to target Protein S as a means of restoring proper blood clotting in people with all types of VWD.
December 13, 2024
Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes almost three years after treatment. The data from the phase 3 AFFINE clinical trial was shared at a presentation given at the American Society of Hematology (ASH) annual meeting.
December 13, 2024
Expression Therapeutics announced phase 1 clinical trial results based on ET3, the company's investigational gene therapy. It is a therapy developed with novel lentiviral vectors. The therapy uses repurposed retroviruses to introduce factor VIII transgene to deliver targeted genetic material.
November 15, 2024
Centessa Pharmaceuticals has discontinued their clinical development of their experimental under-the-skin therapy SerpinPC. The therapy was found safe and well tolerated in a recent trial, but the company decided to focus their investments on other areas after Pfizer's Hympavzi, which used a similar mechanism, was approved by the FDA.
November 8, 2024
A recent study from Italy shows new findings on why bone problems are a common issue among hemophilia patients. The study looked at missing clotting proteins and compared experiments on cells gathered from healthy donors and hemophilia patients.
October 18, 2024
A real world study showed that eptacog beta, known as Sevenfact, was deemed safe and effective at stopping bleeds in patients with hemophilia A or B that also have inhibitors. The bypassing agent was shown to be 96% effective at resolving symptoms and controlling bleeds.
October 11, 2024
The US Food and Drug Administration approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce frequency of bleeding episodes in adult and pediatric patients 12 and over. The treatment is designed to treat both hemophilia A and B as it targets a protein in the blood clotting process.
October 1, 2024
According to a real-world study in Japan, Hemlibra effectively prevented bleeds in patients with acquired hemophilia A. The study compared outcomes of patients with acquired hemophilia A who were treated with Hemlibra, and those who were not, finding those in the Hemlibra group had significantly less bleeding.
September 13, 2024
In a survey of doctors, researchers found that nearly two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce quality of life, even when they are on factor replacement therapy to reduce bleeding. The data showed that joint problems still represent a burden in the care of these patients.
September 6, 2024
A gene editing therapy being developed by Metagenomi for hemophilia A has shown safety and durability in an ongoing pre-clinical study in three non-human primates. The results showed that after a year, two animals had normal or nearly normal factor levels. The third animal had factor levels in the mild hemophilia range.
August 20, 2024
A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and need for prophylaxis for as long as seven years. This is the longest follow-up to date for any hemophilia A gene therapy clinical trial.
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