A decade of innovation: perspectives from the Cell and Gene Therapy Insights Editorial Advisory Board
Cell & Gene Therapy Insights 2025; 11(9), 1187–1190
DOI: 10.18609/cgti.2025.137
A decade of innovation
Perspectives from the Cell & Gene Therapy Insights Editorial Board
To celebrate the 10th anniversary of Cell & Gene Therapy Insights, members of our expert Editorial Advisory Board share their reflections on the remarkable progress of the past decade, the innovations shaping the field today, and their predictions for the future. From CRISPR-based therapies to in vivo engineering and decentralized manufacturing, their perspectives offer a snapshot of where the field stands and where it is heading next.
(Left to right) Jason Acker MBA PhD FCAHS, Professor at the University of Alberta, Senior Scientist at Canadian Blood Services, and Senior Director at PanTHERA CryoSolutions; Lee Buckler MBA LLB, Managing Director of Cell Therapy Group, Partner at HighMont Advisors, and Senior Vice President of Advanced Therapies, Blood Centers of America; Gary C du Moulin MS MPH PhD, Executive Director of Product Development, Quality, and Regulatory Affairs at EASCRA Biotech; Philip D Gregory DPhil, Senior Vice President and Head of Regeneron Cell Medicines; Clare Hague PhD LLM, Founder, Oncology Access Solutions Ltd
(Left to right) Tamara Laskowski PhD, Head, Biotech Partnerships & Strategy Head, Biotech Partnerships & Strategy, CTMC; Nate Manley PhD, Senior Principal and Head of Nonclinical at Dark Horse Consulting Group; Aby J Mathew PhD, Executive Vice President and Chief Scientific Officer at BioLife Solutions; Claudio Mussolino PhD, Group Leader, Transfusion Medicine and Gene Therapy at the University Medical Center Freiburg; Pilar Redondo, Site Head at Takeda Madrid (Tigenix)
What has been the single most pivotal development in CGT over the past decade?
Across responses, two milestones stood out: the regulatory approval of CAR-T therapies, which proved that highly personalized treatments could be scaled and commercialized, and the rise of CRISPR/Cas9-based editing, which has revolutionized therapeutic design and enabled the first genome-editing approvals. Several editorial board members also noted advances in in vivo engineering and regulatory collaboration as key drivers of progress.
Clare Hague For me, as a health economist, the regulators’ decision to fast-track CAR-T approvals, even with single-arm data, was pivotal. They struck a careful balance between unmet need and robust evidence, particularly for pediatric patients, and their willingness to work alongside manufacturers throughout the process set a strong precedent.
Philip D Gregory Two milestones stand out to me. On the technology side, CRISPR-based gene editing not only enabled the approval of Casgevy™ for sickle cell disease but also transformed our therapeutic toolbox for future therapies. On the commercial side, the approval of Kymriah™, the first CAR-T in the US, proved these treatments could become viable products and built confidence in their potential beyond B-cell malignancies.
Gary C du Moulin The broader adoption of rapid microbiological methods has transformed manufacturing. It has done so by enabling real-time contamination monitoring, strengthening quality control, streamlining regulatory review, and raising standards across the industry.
What are the most exciting recent advances in CGT?
Many contributors pointed to the rapid momentum behind in vivo therapies, new delivery technologies (viral and non-viral), and the expansion of CGTs into solid tumors and autoimmune diseases. Progress in automation, decentralized manufacturing, and quality standards is making therapies more accessible globally, while landmark case studies highlight the transformative potential of precision medicine.
Claudio Mussolino In a medical first, clinicians at CHOP and Penn treated an infant with a fatal urea cycle disorder using CRISPR base editors delivered via lipid nanoparticles. After three doses, the child showed significant health improvements, providing powerful proof-of-concept for in vivo therapies in rare genetic diseases.
Nate Manley Gene editing and in vivo therapies are advancing rapidly, with increasingly precise viral and non-viral delivery systems and new approaches for larger genetic payloads. Point-of-care manufacturing, bridging ex vivo and in vivo approaches, could ease cost and safety challenges while raising new regulatory considerations. Meanwhile, cellular immunotherapies are pushing into solid tumors, often in combination with checkpoint inhibitors and other modalities.
Jason Acker Closed-system, integrated bioprocessing platforms are making distributed manufacturing a reality, paving the way for advanced therapies to reach patients even in low- and middle-income countries.
What advice would you offer to researchers entering this field?
There was broad agreement that success in CGT demands early strategic planning, strong regulatory awareness, and cross-functional collaboration. Many emphasized the importance of mentorship and a focus on scalability and sustainability, alongside innovation.
Gary C du Moulin Adopt GMP principles early and embed a robust CMC strategy from the start. Building a culture of quality early on accelerates development and better equips teams for the challenges of scale-up.
Pilar Redondo “Begin with the end in mind.” Ensure your therapy meets a real unmet need and is designed for long-term sustainability in both manufacturing and delivery.
Aby J Mathew Learn from the challenges others have faced in this space and surround yourself with experienced colleagues who have successfully brought therapies to market. Their expertise is invaluable.
Clare Hague Develop your pricing and market access strategy as early as your clinical plan. Innovation has little impact if patients cannot access it.
How do you see CGT evolving over the next 5–10 years?
Experts predict a decisive shift toward in vivo therapies, greater automation, and AI-driven innovation. Therapies will move beyond rare diseases to common conditions, with decentralization and manufacturing advances making treatments more accessible. Regulatory strategies are also expected to evolve to support this growth.
Claudio Mussolino In the coming decade, AI and synthetic biology will enable the design of safer, smarter therapies, including programmable immune cells equipped with logic-gated circuits that can dynamically respond to disease signals.
Aby J Mathew We are approaching a time when CGTs will move into frontline use. Advances in biopreservation and local delivery will expand accessibility, but vigilance will be essential to protect patients from unproven interventions.
Lee Buckler Within five years, we will see multiple autologous therapies manufactured in closed, automated systems. By the end of the decade, cell therapies will be more affordable, widely available, and delivered in outpatient or community settings rather than specialized centers.
Tamara Laskowski The future of CGT lies in expanding beyond rare diseases to common conditions, with purpose-built therapies tailored to individual patients, designed for durability, and increasingly capable of delivering cures rather than disease management.
Celebrating the journey and envisioning the future
Taken together, these reflections capture a field that has made extraordinary strides, from the first CAR-T approvals to the dawn of in vivo gene editing. As highlighted by our Editorial Advisory Board, the next era of progress will be shaped by AI, novel delivery platforms, and decentralized manufacturing, moving CGTs beyond rare diseases and into mainstream medicine. Marking the 10th anniversary of Cell & Gene Therapy Insights, these perspectives remind us that the challenge now is not only to innovate but to make these therapies more accessible, affordable, and impactful for patients worldwide.