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Gene Therapy Offers a Glimmer of Hope for Rare Disease Patients

Article

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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