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Spark's Gene Therapy Hemophilia Data Give Investors The Jitters

Executive Summary

Company explains how it will design upcoming Phase III hemophilia A study of SPK-8011 for success, but some analysts still think it's risky business.

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BioMarin, Spark Hemophilia Gene Therapies Progress As Accelerated Approval Favors Their Indications

As updated Phase I/II results for BioMarin's hemophilia A and Spark's hemophilia B gene therapies were presented on May 22, it was revealed that FDA plans to make hemophilia the first gene therapy indication eligible for accelerated approval based on a surrogate endpoint.

Installment Payments For Gene Therapy; From Luxturna To Hemophilia

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