Newsroom
In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.
Phase 3 Gene Therapy Trial Shows Giroctocogene Fitelparvovec Safe, Effective
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.
Investigational Hemophilia A Gene Therapy Updates Shared at ASH
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.
Potential Hemophilia Treatment SerpinPC Halted by Centessa
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.
Study Finds Missing Clotting Proteins in Hemophilia Patients Can Directly Affect Bone Health
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.
Study Shows Sevenfact Safe, Effective for Patients with Hemophilia A or B with Inhibitors
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.
FDA Approves First Non-Factor, Once-Weekly Treatment for Hemophilia A or B
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.
Study Suggests Hemlibra Effectively Prevents Bleeding in Acquired Hemophilia A
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.
Survey Finds Most Men With Hemophilia Continue to Have Joint Problems Even With Prophylaxis
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.
Gene Editing Therapy Shows Promise in Pre-clinical Study in Animals
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.
Gene Therapy Data for Roctavian Shows Most Remained Off Preventative Therapy After Seven Years
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.
Prophylactic Treatment of Hemophilia Linked to Less Disability-Related Unemployment
August 6, 2024
A study done through the World Federation of Hemophilia shows that hemophilia has a negative effect on a person's employability. The data from the ADVANCE study showed those who received prophylactic treatments had a significantly lower probability of unemployment due to disability.
Pfizer Shares Positive Trial Data for Hemophilia A Gene Therapy
August 1, 2024
Pfizer announced positive data for their phase 3 clinical study of giroctocogene fitelparvovec, an investigational gene therapy for hemophilia A. The AFFINE study is looking at the safety and efficacy of the one time infusion and it's impact on patient's annual bleeding rate through 15 months after treatment.
News Content Disclaimer:
Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.
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