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THE PHARMA REVIEW (JULY – AUGUST 2019)

Applications and the Limitations of Real-World Data in Gene Therapy Trials

QUANTICATE

Introduction: There are approximately 7,000 distinct rare diseases that exist, affecting 350 million people worldwide, and approximately 80% of those rare diseases are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system1 have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies especially for single gene mutation disorders. The FDA has more than 700 active INDs for gene and cell therapies. In 2017, the FDA approved two cell-based gene therapies (Kymriah and Yescarta) utilising chimeric antigen receptor T-cells (CAR-T) and also approved the first gene-therapy product (Luxturna) to be administered in vivo, which in addition was the first to target a specific rare disease genetic condition. Collins and Gottlieb, of the NIH and FDA respectively, have stated that “it seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases”2.

 

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