The commercialization of hemophilia AAV gene therapies in recent years has created a steep learning curve for clinicians, patients, and other stakeholders in the bleeding disorders community. As a novel treatment unlike any that has come before, gene therapy has compelled a series of questions and considerations relevant to its efficacy, safety, patient eligibility, implementation, cost/reimbursement, and more.

NBDF monitors emerging therapies and changes in the therapeutic landscape that may affect people living with bleeding disorders. One of those developments involves BioMarin’s decision to out-license (allowing another company to market and sell) ROCTAVIAN, their gene therapy for adults with severe hemophilia A. 

This statement from BioMarin explains their decision:

The latest Resource Roundup (RR) is dedicated to women, girls, and people with the propensity to menstruate (WGPPM), all historically underserved groups within the inherited bleeding disorders community. While recognition of these populations and their struggles to achieve healthy equity has increased recently, affected individuals continue to face challenges when it comes to timely diagnosis and management. 

Pathway to Cures, the venture philanthropy fund of the National Bleeding Disorders Foundation is focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community.  We carry out our work with volunteer expert advisors on our scientific advisory group, investment committee and board of directors.  Today we spotlight our investment committee, our process for determining what is presented to this committee and the criteria used in making NBDF’s Pathway to Cures investments.