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.

New Data for BioMarin's Gene Therapy for Hemophilia A Shared at ISTH

July 19, 2021

BioMarin announced new data for their investigational gene therapy, valoctocogene roxaparvovec currently in a phase 3 study to treat hemophilia A. Results included positive data for the 134 participants who have been involved in the study for more than a year.


FDA Puts Hold on Sigilon's Hemophilia Cell Therapy Trial

July 9, 2021

Sigilon Therapeutics' cell therapy trial for hemophilia A is on hold due to an FDA hold after a patient developed inhibitors. Three patients have undergone treatment and one developed antibodies to inhibit factor VIII. 


ASC Therapeutics to Begin Phase 1/2 Gene Therapy Clinical Trail for Hemophilia A

July 9, 2021

The US Food and Drug Administration approved ASC Therapeutics' request to open a clinical trial in the US for the safety and efficacy of ASC618, a second-generation gene therapy for hemophilia A. The trial will begin in July 2021 and will test the one time therapy in up to 12 adults. 


Study Analyzes Risk of Inhibitors in Non-Severe Hemophilia A

June 18, 2021

A new study found that those with non-severe hemophilia A were also at risk for developing inhibitors. The findings highlight the importance of closely monitoring these patients in order to prevent or delay the onset of inhibitors. 


Prophylactic Hemlibra May Lower Treatment Costs for Hemophilia A Patients

June 14, 2021

A study published in the journal Haemophilia shows that switching or starting prophylactic treatment Hemlibra may lead to a significant reduction in treatment related expenses. The study found there was about 27.5% drop in expenses over a six month period. 


Book Contest for Hemophilia B Patients Ends June 30

June 9, 2021

The Coalition for Hemophilia B is hosting a contest for those with hemophilia B, their family members, or caregivers. Participants can enter through creating a comic or coloring book and are judged in various age categories. Prizes are $500 for each category and submissions are due by June 30, 2021. 


Gene Therapy Trial Data Suggests Efficacy in the Presence of Antibodies

May 25, 2021

Updates from the Phase III HOPE-2 clinical trial of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B patients, were shared at the ASGCT Virtual meeting in May. The data found that AAVs appear to be safe and effective in patients with and without pre-existing neutralizing antibodies. 


Roctavian, Gene Therapy for Hemophilia A, Shows Potential from Early Data

May 19, 2021

BioMarin releases key findings in gene therapy study of Roctavian, showing no signs of tumors, malignancy, or permanent integration to the DNA of mice. 


First Patient Dosed in Marzeptacog Alfa (MarzAA) Phase 1/2 Study

May 18, 2021

Catalyst Biosciences announced the first dosing of their Marzeptacog Alfa, also known as MarzAA, in a phase 1/2 clinical trial of hemophilia A patients with inhibitors who are being treated with Hemlibra.  


Jivi May Prevent Bleeds in Minor Surgery for Hemophilia A Patients

May 7, 2021

Severe hemophilia A patients who were treated prophylactically with Jivi showed the treatment was highly effective in preventing bleeds during and after minor surgeries. 


Dosing Begins for Efanesoctocog Alfa Trial in Boys with Hemophilia A

April 16, 2021

A first patient has been dosed with Efanesoctocog Alfa, formerly known as BIVV001, in a phase 3 clinical trial evaluating its safety and efficacy in boys with severe hemophilia A. 


Graphic Novel Shares Life With Hemophilia, Dungeons and Dragons

April 14, 2021

Hemophiliac Jonathan Hill created a graphic novel based on his memoir and characters and adventures he came up with as a way to cope with his bleeding disorder. The graphic novel is available to order free through 

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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