gene therapy

In a notable advancement for hemophilia treatment, Pfizer Inc. (NYSE: PFE) has received U.S. Food and Drug Administration approval for BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy aimed at adults suffering from moderate to severe hemophilia B. This innovative treatment could significantly alter the treatment landscape for patients traditionally reliant on regular factor IX (FIX) prophylaxis.


A Potential Game-Changer in Hemophilia Care

BEQVEZ is tailored for individuals who are on routine FIX prophylaxis, have a history of life-threatening bleeding, or experience recurrent serious spontaneous bleeding episodes, and have no neutralizing antibodies to the AAVRh74var capsid, as detected by an FDA-approved test.

According to Dr. Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program, BEQVEZ represents a potential paradigm shift, offering patients the possibility to significantly reduce their long-term medical and treatment burdens.


Efficacy Demonstrated in Clinical Trials

The FDA endorsement follows compelling evidence from the pivotal BENEGENE-2 study. This Phase 3 trial revealed that BEQVEZ could lower the annualized rate of bleeding significantly in comparison to traditional prophylaxis, with some patients experiencing zero bleeds over three years of follow-up.


Global Implications and Next Steps

Following its approval in Canada and the ongoing review by the European Medicines Agency (EMA), BEQVEZ has the potential to impact over 38,000 individuals worldwide living with hemophilia B. This therapy promises to ease the constant treatment regimen associated with the disease.

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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