In Vivo Gene Therapy: Personalizing the Ultra-Rare and Scaling-Up for Chronic Disease

Advanced Therapeutics, including gene and call therapies, have emerged as significant FDA/EMA approved therapies for several ultra-rare genetic disorders and advanced malignancies.  In the next couple years, many more will be seeking registration using validated in-vivo and ex-vivo gene transfer and modification platforms (AAV, lentiviral, oligonucleotides, gene editing nucleases, etc.) earning a significant role in the healthcare pharmacopeia.  However, while hurdles such as regulation, manufacturing costs, logistics and reimbursement have limited patient access, pioneers in the gene therapy sector have been reshaping what healthcare delivery looks like for the next wave of innovators. This BioFuture Salon will discuss the state of the art and potential for gene therapies to become more “drug-like” and scalable for either the personalized ultra-rare or more common chronic disorders.