News

U.S. doctors caring for people with hemophilia are more likely to make treatment decisions based on patient preferences than doctors in the U.K., who tend to be more influenced by their colleagues and government policies, according to a study comparing decision-making in both…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…

ASC Therapeutics has gotten exclusive rights to Expression Therapeutics‘ technology to develop liver-targeting hemophilia A gene therapies. “We have a common mission with Expression Therapeutics — to develop novel therapies that will dramatically improve the lives of persons with hemophilia around the world. Hemophilia A is a…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

A single infusion of AMT-061, an investigational gene therapy for hemophilia B, continues to maintain clinically significant increases in clotting factor IX (FIX) up to six months after its administration, as demonstrated in all three patients enrolled in uniQure…

Trio Health has launched a novel database with real-time clinical data of patients with hemophilia which combines information from medical doctors and pharmacies. The main aim of the database is to provide information on the treatment regimen to physicians in order to improve patients’ outcomes. Access to the database…

A new collaboration between St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) is aimed at helping blood disorder patients in countries where they may not receive regular treatment. The partnership’s centerpiece will be a program to conduct a gene therapy clinical trial in…

Hemophiliacs in Europe have never had so many reasons to feel hopeful about the future, thanks to significant recent scientific advances. But “vested interests, entrenched systems, and blatant, outright corruption limit access” to even basic healthcare in some Eastern European countries, says Amanda Bok, CEO of the Brussels-based European…

A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…

A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…