A breakthrough in hemophilia care is now unfolding on the Chinese mainland. The National Medical Products Administration (NMPA) recently approved BBM-H901, a pioneering gene therapy for hemophilia B developed by Shanghai-based Belief BioMed Inc. This one-time injectable treatment is designed for adult patients with moderate to severe symptoms, offering a potentially life-changing option by enabling the body to produce the missing clotting protein internally.
Hemophilia, a rare inherited disorder that impairs the blood's ability to clot, primarily affects males. Globally, more than 270,000 people live with the condition, and over 40,000 cases of hereditary bleeding disorders have been registered in China as of late 2023. Until now, treatment in China predominantly relied on frequent intravenous infusions of clotting factorsāa method that is both burdensome and costly.
With the arrival of BBM-H901, health experts are witnessing a shift toward innovative therapies. By using an adeno-associated virus (AAV) vector to deliver a functional copy of the factor IX gene into liver cells, this gene therapy tackles the underlying cause of hemophilia rather than just managing its symptoms. This approach not only reduces the frequency of treatments but also minimizes the risk of immune reactions associated with conventional methods.
Over the past decade, gene therapies have made impressive strides in treating various rare genetic conditions, including treatments for conditions like spinal muscular atrophy. The approval of BBM-H901 marks a significant milestone in genomic medicine and reflects a broader commitment under the Healthy China Initiative to advance personalized, patient-centered care.
As researchers refine these treatments, patients on the Chinese mainland and around the world can look forward to more sustainable and effective solutions in managing rare diseases. This breakthrough is not only a leap forward in medical innovation but also a beacon of hope for those seeking long-term, accessible treatment options.
Reference(s):
The latest in hemophilia treatment: New techniques, new research
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