Advanced Therapies Week 2025: Superplenary

This video transcript captures a plenary session from Advanced Therapies Week 2025 focused on the current state and future directions of cell and gene therapy. Anthony Davies, founder and CEO of Dark Horse Consulting Group, opens the session by framing the field’s progress, challenges, and the critical need for collaboration among patients, physicians, and payers, the “three Ps,” to ensure successful commercial launches and sustained innovation. Key voices include Donavon Decker, a pioneering patient and advocate who underwent early gene therapy trials for limb-girdle muscular dystrophy 2D, Sarah Creviston, Vice President of Patient Advocacy at Novartis Gene Therapies, who highlights the importance of patient engagement and infrastructure for successful therapy adoption, Yuliia Sanko, founder of Timosha’s Smile, a foundation supporting pediatric oncology patients in Ukraine, who emphasizes dignity and access amid war and resource constraints, and Bo Wiinberg, Chief Business Development Officer of the Novo Nordisk Foundation Cellerator, who describes their Cellerator initiative aimed at bridging gaps in early-stage CGT development through infrastructure, knowledge, and partnerships.

The dialogue underscores tremendous progress. The field has advanced from experimental promise to multiple FDA approvals and commercial products, with successes like Zolgensma for spinal muscular atrophy. However, commercial uptake has lagged due to high costs, limited patient access, gaps in physician education, and payer skepticism. Patient voices reveal challenges with one-time therapies that cannot be redosed, the need for more affordable nonviral vectors, and the necessity of early patient and payer engagement. Industry perspectives stress that successful launches require integrated teams, transparent communication, and innovative contracting and reimbursement strategies. The Novo Nordisk Foundation’s Cellerator reflects a strategic effort to improve early clinical readiness and reduce development costs through partnerships and infrastructure, facilitating faster patient access.

The session concludes with a forward-looking discussion emphasizing the urgency of lowering costs, expanding equitable global access, supporting rare and ultra-rare disease communities, and harnessing technological advances such as AI. Patient advocates call for dignity, hope, and the expansion of effective gene therapies beyond current successes. Overall, this Advanced Therapies Week 2025 Superplenary provides a comprehensive snapshot of the field’s evolution, ongoing obstacles, and opportunities for collaborative progress toward a healthier tomorrow.

Highlights

• Cell and gene therapy has transitioned from future promise to multiple approved commercial therapies, but commercial uptake remains a challenge.
• The “three Ps,” patients, physicians, and payers, must be engaged collaboratively to ensure successful therapy launches and market sustainability.
• One-time dosing and lack of redosing options create difficult decisions for patients and limit treatment strategies, driving interest in nonviral gene therapy approaches.
• Patient engagement from early development through commercial launch is critical to build trust, infrastructure, and real-world evidence.
• High therapy cost and payer skepticism threaten investment cycles and equitable global access, requiring innovative pricing and reimbursement models.
• The Novo Nordisk Foundation Cellerator aims to bridge early-stage CGT development gaps by providing infrastructure, knowledge, and partnerships.
• Global equity remains a concern, especially in less wealthy regions, where patient advocacy groups play key roles amid limited resources and conflict.

Key Insights

• Maturation of CGT from Concept to Commercial Reality: The field has evolved from hype to tangible approvals such as Kymriah, Yescarta, Luxturna, and Zolgensma. This validates potential while surfacing real-world challenges in access and infrastructure that must be addressed for sustained impact.
• Patient Advocacy and Engagement are Central: Donavon Decker’s testimony shows why early and continuous patient involvement matters for trial design, risk tolerance, and real-world needs. Groups such as Timosha’s Smile help bridge geographic and systemic gaps, reinforcing that trust is essential for irreversible therapies.
• Economic Sustainability and Pricing Challenges: High development costs and small patient populations strain payers and slow uptake. Clear value stories, outcomes-based contracts, and lower-cost technologies, including nonviral vectors, are needed to align incentives across the “three Ps.”
• Infrastructure as a Bottleneck: Successful deployment depends on building treatment center capacity, training, and logistics in parallel with clinical development. Education for physicians and site readiness are prerequisites for confident adoption at scale.
• Start Market Access Early: Planning for evidence needs, pricing, and reimbursement as early as phase 2 helps align R&D with payers’ requirements. Real-world evidence post-launch is critical for sustained coverage in rare diseases with limited trial datasets.
• Public-Private-Philanthropic Collaboration: Initiatives like the Novo Nordisk Foundation Cellerator illustrate how shared facilities and guidance can reduce early development risk and accelerate clinical readiness.
• Global Equity and Regional Disparities: Access remains uneven across regions. Patient-led organizations can enable cross-border care and education, but durable equity will require tailored, region-specific approaches aligned with local systems and economics.

Conclusion
This Advanced Therapies Week 2025 Superplenary offers a multi-stakeholder roadmap for translating clinical promise into patient impact. By aligning the “three Ps,” investing in infrastructure and education, planning market access early, and leveraging collaborative models, the field can improve affordability, access, and commercial execution for transformative therapies.