Gene therapy for hemophilia A and B now exists as a licensed, prescribable treatment option for patients in certain countries; however, many questions and challenges remain. Senior advisor Dr. Donna DiMichele and patient-host Patrick James Lynch speak to global KOLs about the current state of hemophilia A and B gene therapies, both commercially and investigationally.

 

Contributors:

Lindsey A. George, MD

Margareth Ozelo, MD

Steven Pipe, MD

 

Senior Advisor: Donna DiMichele, MD

 

Hosted & Written by: Patrick James Lynch

 

Featured Advertiser:

Sanofi

 

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Notes and References:

 

Nathwani: Hematology Am Soc Hematol Educ Program . 2022 Dec 9;2022(1):569-578.

 

The current state of this exciting and rapidly evolving field, as well as the challenges that need to be overcome for the widespread adaptation of this new treatment paradigm, is the subject of this review.

 

Pipe: N Engl J Med. 2023 Feb 23;388(8):706-718

 

The annualized bleeding rate decreased from 4.19 (95% confidence interval [CI], 3.22 to 5.45) during the lead-in period to 1.51 (95% CI, 0.81 to 2.82) during months 7 through 18 after treatment, for a rate ratio of 0.36 (95% Wald CI, 0.20 to 0.64; P