For many of the 400 million people worldwide affected by one of more than 7,000 types of rare diseases, gene therapy is the only option that provides a glimmer of hope for diseases that are currently poorly treated.
Gene therapy as a field has greatly advanced over the past two decades, with several approved gene therapies currently available and hundreds more in development – reinforced with increased and stricter regulations on safety and efficacy.
In this article published in PharmaVoice, Pete Robinson, head of the Syneos Health Cell & Gene Therapy Consortium, explores the challenges inherent in bringing a gene therapy through development and into market, and provides insights on considerations to account for in order to succeed in this fast evolving landscape.
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