The Challenges of Gene Therapy

For any emerging biotech company with a novel platform technology, establishing trust in that platform is essential to building momentum and long-term success. Trust must be established with multiple stakeholders, including potential licensing partners, investors, regulators, clinicians and, foremost, with patients. In the gene therapy and genome editing space, these concerns are heightened by the unprecedented manipulation of the body’s basic building blocks, where failures of past programs effectively delayed progress for decades. Building a meticulous plan to establish evidence of safety and of clinical utility in a stepwise manner is thus of heightened concern for companies advancing technologies into this arena.