Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…
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Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important events related to hemophilia. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a summary of…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related serious adverse event in one patient. The event concerns a preliminary diagnosis of hepatocellular carcinoma (HCC), a form…
Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing factors like total health care costs and emergency hospital visits allow the company to better evaluate and distinguish…
Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by recurrent bleeding in the joints. The private investment firm Gurnet Point Capital also will support other aspects of TRM-201‘s…
A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing bleeds and the need for replacement therapies in patients with hemophilia B, according to updated data from a Phase 1/2…
Dosing has been resumed in ongoing clinical trials in the U.S. testing the investigational medication fitusiran in adults and adolescents with hemophilia, Sanofi announced in a press release. The company had placed a voluntary dosing hold on its full clinical development program for fitusiran at the end of…
When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…
Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients with hemophilia B, according to the therapies’ maker, uniQure. The company announced in a press release that…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…
