Gene Therapy and Bleeding Disorders: Making Sense of It All

Gene Therapy is definitely a dominant topic in the bleeding disorders community, with new articles being published regularly. Read some of the latest publications addressing the future of gene therapy and how it could impact people and families living with a bleeding disorder.

Making Sense of It All – Gene Therapy
What exactly is a gene? What happens when genes mutate? And can gene therapy research help solve problems related to mutations? Learn about all of this and more from the BioMarin Gene Therapy Learning Academy. Read more.
Source: National Hemophilia Foundation, Hemaware, August 2022

Gene Therapy and Bleeding Disorders: Where Are We Now, and What’s on the Horizon?
The National Hemophilia Foundation’s 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia, held this past November (and which has been convening since 1966), addressed some of the latest findings around gene therapy as well as what the future holds. Read more.
Source: National Hemophilia Foundation

Does Gene Therapy Face an Uphill Battle? Gene Therapies for Hemophilia Treatment Will Face Strong Market Headwinds, Research Suggests
Solid market position of Hemlibra and strong prescriber interest in RNAi and TFPI treatment options, combined with patient concerns regarding gene therapies, are likely to create obstacles to market entry. Read more.
Source: The Communiqué – August 2022, LA Kelley Communications, Inc.

Hemophilia B — An Excellent Candidate for Gene Therapy
Current Hemophilia B treatments offer strong protection from bleeds, but impact patient lifestyle and require consistent infusions to keep protection levels high. Gene therapy is an innovative approach to treating a medical condition by introducing a new fully functioning, or working, gene into the body, or by turning off or changing the gene that is causing the condition. Why hemophilia B? Read more.
Source: National Hemophilia Foundation, HemAware, August 2022

Paper Features Q&A Resource on Shared Decision Making and Gene Therapy

The Q&A resource highlighted here is meant to encompass all stages of a patient’s decision making journey in gene therapy. As investigational hemophilia gene therapies move closer to regulatory approval, community stakeholders have recognized the acute need for people with hemophilia (PWH) and healthcare professionals (HCPs) to be fully engaged in shared decision making (SDM). Read more.
Source: National Hemophilia Foundation, August 2022

NHF Requests Risk Evaluation and Mitigation Strategy for Pending Gene Therapy Treatments
On July 1, the National Hemophilia Foundation (NHF) submitted a citizen’s petition to the Food and Drug Administration (FDA) in relation to the pending approval of two gene therapy treatments. The intent of the requests in this petition are to ensure the health and well-being of people with hemophilia who receive a gene therapy product. Read more.
Source: National Hemophilia Foundation, August 2022


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