Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…
News
In the first clinical trial of the experimental therapy SIG-001 for hemophilia A, one patient developed scarring in the spheres used to deliver the therapy that rendered the treatment ineffective, its developer, Sigilon Therapeutics, announced. The Phase 1/2 trial was put on hold by the U.S. Food and Drug…
Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…
A partnership between Emmes and MedGenome seeks to accelerate development of genomics-powered therapies for people with rare diseases, such as hemophilia. The companies plan to combine patient data into custom disease registries to improve research design and help with patient recruitment for clinical trials. In addition, they…
Canadian Blood Services (CBS) has awarded a three-year tender — an exclusive bulk purchase agreement aiming to minimize medication cost — to Takeda Canada for the purchase of Adynovate, an injectable replacement therapy for the prevention and treatment of bleeding episodes in people with hemophilia A. This…
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.
Chugai Pharmaceutical is asking regulatory authorities in Japan to extend the use of Hemlibra (emicizumab) in preventing or reducing the frequency of bleeding episodes to people with acquired hemophilia A. Hemophilia A is caused by genetic mutations that make the body unable to produce a fully functional…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525 (giroctocogene fitelparvovec), an investigational gene therapy for hemophilia A. This pause in study recruitment and dosing was taken to give the agency time to review changes to the AFFINE…
Recent Posts
- Why progress in von Willebrand disease treatment finally feels real
- Global survey finds many women with hemophilia face diagnostic gaps
- Learning how to infuse factor gave my family freedom, part 1
- Guest Voice: My sons with hemophilia are blessings after a devastating loss
- Love shows up to navigate hemophilia, distance, and family bonds