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Opportunities and Challenges in Cell and Gene Therapy Development*. 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. First, we talk through the ideal, mediocre, and restrictive labels for our investigational therapy. Revenue will wane naturally as the addressable population is depleted a few years after launch. For Jesse, the novel therapy showed promise of a chance at a new life—one free from ornithine transcarbamylasedeficiency (OTCD), a genetic disease that had plagued him his entire life. Our Company, including a Contract Research Organization (CRO) and Contract Commercial Organization (CCO), is purpose-built to accelerate customer performance to address modern market realities. Genuine potential for success. Perhaps most important, we need to train world-class field teams to work closely with all these stakeholders. By submitting this form, you agree to our Terms & Conditions and Privacy Policy of our website. Mothers like Parvathy, who have children with rare genetic diseases, carry unique medical, psychosocial and economic burdens unfamiliar to many. Again, this may seem early for a company at AVROBIO’s stage. 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. Promise of personalized medicine. 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. I believe that goal is best served by a broad label when the product comes to market. Challenges of Gene Therapy The new genes have to reach the right place. This is a foundational element for all our work. This presentation will highlight some of the existing analytical challenges and provide guidance on development of an appropriate strategy to help overcome these. Potentially curative therapies such as gene therapy are relatively rare among pharmaceuticals, but can possibly enable patients to live functional lives, free caregivers to do the same, lessen the economic burden of care and decrease the strain on healthcare systems. We’re proud of this work. As I look back at these recommendations, I must admit, they seem staggering. Every step along the way needs to be carefully planned. What options do these patients and their parents have for treating these rare conditions? They must truly understand what patients need and move heaven and earth to make that happen — all in an environment where compliance is fundamental. Current Challenges. 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). The “bolus-like” revenue curve associated with a first gene therapy presents a challenge to achieving sustainable growth . If you have any questions, please Contact Us. This is a challenge for every modality, of course, but it’s especially critical for gene therapy, where we have to make decisions early on about whether to include patients with genetic variants in clinical trials — and understand what that might mean for the biomarkers we use to measure efficacy. One of the biggest challenges for AAV gene therapy products is establishing an appropriate analytical strategy to support product manufacture, release, stability, comparability and characterization at different stages of development. Sadly, the treatment Jesse was given led to his death, shaking up the world of gene therapy. Do It Yourself: Make a detailed note on the harmful effects of gene therapy. 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. This website uses cookies to ensure you get the best experience on our website. Optimising manufacturing is still a big challenge for gene therapy. A correct diagnosis takes an average of five years, with a 40 percent error rate. That’s been my philosophy throughout my career, and in the rapidly maturing field of gene therapy, it’s non-negotiable. Syneos Health® (Nasdaq:SYNH) is the only fully integrated biopharmaceutical solutions organization. The rules of engagement in the gene therapy space allow for this type of early conversation because this modality is so new and — let’s face it — because the high prices for the early-to-market gene therapies have payers searching hard for solutions. As my first hire, I plan to bring on a specialist in global market access. That’s why it is vital to start talking about reimbursement, not just at leadership and board meetings, but more broadly among potential stakeholders. Jul-Sep 2017;15(3):369-375. doi: 10.1590/S1679-45082017RB4024. In truth, though, this is just the beginning. You can withdraw your consent at any time. “We were helicopter parents, ready to plot our babies’ lives through college and beyond. These challenges fall into one of five general areas (Exhibit 3). These questions are important to think through early, as the answers can help shape trial design. It is important to pressure test your clinical trial supply chains early, probing the capabilities of existing and potential partners and systematically working through all the implications of scaling the process. These conversations involve post-approval hypotheticals, but they do not involve promoting investigational products. Then, we work backward to identify how we might arrive at each of those outcomes. With just a tiny number of gene therapies approved globally, we don’t have much precedent here. As I tell my teams, you only get one chance to launch, and at launch, you are your label — so it’s essential to get it right. Underlying all the elements I’ve discussed here is the need to understand the patient journey. Despite the tragic results of Jesse’s treatment, exploration of gene therapy continued on for the same reason that Jesse entered his gene therapy trial. Gene Therapy Considerations and Challenges in an Evolving 2020 Market October 12, 2020 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. We need to start having discussions now with commercial and government payers and with the external voices who help shape public policy on pricing. To get people thinking about these issues, I like to run a thought exercise with each program team. Concurrently, the lessons learned from Jesse’s death have led to increased and stricter regulations on gene therapy safety and efficacy. We need to understand what might motivate them to take a leap of faith and enroll in a clinical trial for an investigational gene therapy. This is a challenge for every modality, of course, but it’s especially critical for gene therapy, where we have to make decisions early on about whether to include patients with genetic variants in clinical trials — and understand what that might mean for the biomarkers we use to measure efficacy. But we know that expanding the supply chain to a global, commercial scale — even in rare diseases with relatively small patient populations — is very different. This therapy became possible through … 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. We need to learn more — from them, not from textbooks — about the strengths, limitations, and burdens of the standard of care. In the gene therapy realm, we need all our field teams to be creative, empathetic, and mission-driven problem solvers. In a particularly emotional recount of her experience parenting children with genetic diseases, one mother, Parvathy, shared a heartbreaking story during the SyneosHealth® Rare Disease Day Symposium: “We had it all figured out,” she said. After all, even after we as an industry nail the science, we will face substantial clinical, regulatory, and logistical challenges in bringing these therapies to market. Sometimes targeted cells stop the new genes from entering. The short duration of meaningful revenue Here are the challenges I’m thinking about now: We all want approved therapies to be available to every patient who can benefit. It means implementing rigorous quality assurance protocols, starting with the apheresis centers that collect the patient’s blood products and continuing through every step of vector manufacturing, cell transduction, cryopreservation, and infusion. Confronting the Challenges of Reimbursement. 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