Thus, gene therapy is understood as the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications that target therapeutic treatment. Additionally, gene therapy is applicable far beyond just rare diseases, and gene therapies are being developed for indications affecting larger numbers of patients, including cancer, ophthalmological conditions, CNS disorders including Alzheimer’s and Parkinson’s, and many more. Those are not issues you can resolve on short notice. Then, we work backward to identify how we might arrive at each of those outcomes. Revenue will wane naturally as the addressable population is depleted a few years after launch. We all know that the supply chain for these therapies is long and complex. That’s why it is vital to start talking about reimbursement, not just at leadership and board meetings, but more broadly among potential stakeholders. And then the scenic highway that was our life turned into a car crash from which we are still recovering.”. There’s a natural tension here: The clinical team may want to tighten trial eligibility to a more homogeneous set of patients who are more likely to show strong benefits. For many patients with debilitating or life-threatening rare genetic diseases, gene therapy is their only hope. I believe that goal is best served by a broad label when the product comes to market. Gene therapy as a field has greatly advanced since Jesse received his treatment, with seven approved gene therapies available in the U.S. or EU market as of July 2020 and hundreds more in development (Figure 1). By Holly May, Chief Commercial Officer, AVROBIO. “We were helicopter parents, ready to plot our babies’ lives through college and beyond. We need to understand, too, the hopes and the hesitations of their loved ones, their physicians, their trusted advocates, and everyone else who might play a role in the anticipated success of our mission. Diagnosis patterns for children with rare diseases are troubling, with approximately 50 percent of rare diseases affecting children, 30 percent of whom will not reach their fifth birthday. With just a tiny number of gene therapies approved globally, we don’t have much precedent here. Perhaps most important, we need to train world-class field teams to work closely with all these stakeholders. Promise of personalized medicine. Will patients ultimately want to have choices at certain stages, such as the flexibility to choose from among several conditioning regimens? These challenges fall into one of five general areas (Exhibit 3). All Rights Reserved. In the gene therapy realm, we need all our field teams to be creative, empathetic, and mission-driven problem solvers. Sometimes targeted cells stop the new genes from entering. It’s not. Gene therapy: advances, challenges and perspectives Einstein (Sao Paulo). At AVROBIO, for instance, we expect our gene therapies, if approved, potentially could save many millions for each patient treated by comparison with lifetime costs for the standard of care in indications such as Fabry disease, Gaucher disease, Pompe disease and cystinosis. We have the opportunity to pioneer our own model to best reflect the value proposition we expect to offer, the cost savings we hope to deliver and the core goal of reaching as many patients as stand to benefit from our therapies. This website uses cookies to ensure you get the best experience on our website. Underlying all the elements I’ve discussed here is the need to understand the patient journey. And we need to understand who among them would be ideal candidates for the therapy if approved, and how to address the many questions that are bound to arise. Two decades ago, as gene therapy was emerging as the next great frontier in modern medicine, the field was forced to reckon with tragedy: the death of Jesse Gelsinger as a direct result of a gene therapy treatment he was given. Terms of Service apply. It even means thinking through and planning for crisis scenarios, such as a natural disaster that might disrupt shipping of a therapy during a crucial window for delivery. Biostatistics and Statistical Programming, Clinical Development Services Project Management, Scientific and Medical Affairs Advisory Group, Risk and Program Management Advisory Group, Accelerating Rare Disease Drug Development: Lessons Learned from Muscular Dystrophy Patient Advocacy Groups, Partnering with Pediatric Health Systems to Advance Innovation, Gene Editing Your Cell Therapy: Key Considerations for Biopharma Companies and Investors. Helicopter parents, ready to plot our babies ’ lives through college and beyond to put our brain. 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