Podcast recap: Richard Wilson on the current state of gene therapy
In this week’s episode of The Genetics Podcast, we spoke with Richard Wilson, Senior Vice President and Primary Focus Lead for Genetic Regulation at Astellas, about the current state of gene therapy. Richard offered an insider’s view of the opportunities and challenges facing the field, from groundbreaking patient outcomes to manufacturing bottlenecks, regulatory scrutiny, and the commercial realities of bringing gene therapies to market.
Breakthroughs amid challenges
Richard began by reflecting on how far gene therapy has come in a relatively short time. Patients with spinal muscular atrophy and sickle cell disease are now leading lives that would have been unimaginable just a decade ago. These successes prove that the technology can fundamentally alter the course of disease.
However, alongside this progress are persistent challenges. Safety concerns, particularly immune responses and dosing-related toxicities, continue to spark debate. Commercial models remain unsettled, as payers struggle to accommodate therapies priced in the millions of dollars per treatment. Manufacturing is still a bottleneck, with limited capacity, high costs, and variable product quality slowing the pace of development.
Manufacturing: Moving beyond scale
A large portion of the conversation focused on the state of manufacturing, which Richard described as one of the biggest constraints on gene therapy’s future. He emphasized that the field must move beyond the notion that reactor size equals progress. Instead, the priority should be reproducibility, yield, and quality.
At Astellas, the team has made significant investments in process development and new manufacturing facilities. Improvements in productivity are being paired with advances in capsid engineering, which enable therapies to deliver payloads more efficiently at lower doses and with reduced immunogenicity. These advances are critical not just for scientific reasons but for commercial viability as well.
Collaboration across the industry
Richard also emphasized the need for collaboration in areas where companies are not directly competing. One example is immunosuppression strategies in AAV trials, which are currently developed independently by each sponsor. Sharing best practices in such areas could save time, reduce risks, and improve patient safety across the field.
Collaboration must also extend to regulators and payers. Therapies need to be reimbursed and integrated into health systems in ways that are sustainable. Rare diseases will remain the proving ground for gene therapy, but broader applications will require new access and payment models that balance innovation with affordability.
A global perspective
The conversation also highlighted how different regions are approaching gene therapy. The FDA continues to lead with flexible regulatory pathways, but Europe and Japan are eager to maintain competitiveness and avoid delays in access for their patient populations. The European Union’s Biotech Act and Japan’s efforts to simplify regulatory navigation are examples of how regions are adapting to attract and support gene therapy innovation.
These global shifts underscore the importance of alignment. Divergent requirements around endpoints, natural history controls, and reimbursement create additional complexity for sponsors, especially those working in rare diseases where time and resources are already stretched.
Beyond AAV
While AAV remains the workhorse of in vivo gene therapy, Richard acknowledged that alternative delivery systems are gaining traction. Lipid nanoparticles are proving effective for liver-targeted applications, while other viral vectors offer greater cargo capacity and could expand the range of treatable conditions.
At the same time, Richard sees enormous potential for AAV in neurology. Advances in capsid technology and new delivery methods are beginning to make conditions like Rett syndrome, Huntington’s disease, and Parkinson’s disease tractable. He described this as the beginning of a possible renaissance in neuroscience, with AAV opening doors to diseases once considered unreachable.
Keeping patients at the center
Throughout the conversation, Richard returned to the central role of patients. Long-term follow-up requirements, trial design, and manufacturing decisions all matter because they determine how quickly and effectively patients can access therapies. For him, the key to gene therapy’s future is to keep patients at the center of every decision, ensuring their needs guide both scientific and commercial strategies.
Looking ahead
Richard’s perspective is realistic and optimistic. The field of gene therapy is navigating serious challenges, but it is also delivering undeniable results. The next chapter will depend on how quickly developers, regulators, and payers can adapt to streamline the pathway in ways that ensure access.
Listen to the full episode below.