The Critical Role of Academic Translational Centers in Advancing Cell and Gene Therapies
As the field of cell and gene therapy (CGT) matures, academic translational centers remain indispensable in driving innovation and bridging the gap between early research and clinical application. Recent efforts in the UK, Japan, Australia, Spain, the USA, Germany, and Ireland illustrate how dedicated translational frameworks, such as the RISE model, are shaping sustainable CGT ecosystems (Naresh et al., 2025). However, these efforts are now facing significant financial headwinds, in the United States, where drastic cuts to NIH indirect funding threaten to undercut decades of translational research momentum.
The RISE Framework: A Global Lens on CGT Translation
The RISE model (Resource sharing, Interdisciplinary collaboration, Sustainable financial models, and Education) underscores the importance of structuring academic environments to support the full translational pipeline of CGTs (Naresh et al., 2025). This approach is evident in:
United Kingdom: Institutions like UCL lead CGT innovation, leveraging partnerships with the Cell & Gene Therapy Catapult to scale new therapies. Lonrú’s interactive dashboard highlights how UK-based academic centers are leading in CGT trial sponsorship and development phases (Lonrú Consulting, 2025).
Japan: The PMDA's progressive regulatory framework, coupled with translational research centers, has propelled Japan to the forefront of regenerative medicine.
Germany and Spain: Public funding initiatives have strengthened hospital-integrated manufacturing capabilities, increasing trial accessibility.
Australia: Investments in non-viral vector platforms are accelerating local innovation.
Ireland: The recently proposed all-island CGT strategy signals the country’s commitment to fostering a unified research, clinical, and commercial environment (Lonrú Consulting, 2025).
Without vital support, translational momentum is at risk—academic labs remain the backbone of cell and gene therapy innovation, but funding challenges threaten to slow progress from discovery to patient impact.
Threats to Academic Research: The US Funding Crisis
While academic medical centers have historically been the foundation of CGT innovation, recent proposals in the US to cap indirect cost reimbursements represent a direct threat to translational research (NIH, 2025). These funds support vital infrastructure, including laboratories, safety measures, IT systems, and core personnel.
Johns Hopkins University, among others, has sounded the alarm (JHU, 2025):
“These abrupt and sweeping cuts in NIH funding pose an extraordinary challenge to the important and lifesaving work of our faculty, staff, and students. They jeopardize the longstanding and remarkable research partnership that was forged between the federal government and higher education at the conclusion of WWII and put at risk the future of the American research enterprise as a whole.”
The repercussions extend beyond academia—clinical trials reliant on NIH support, particularly in oncology, rare diseases, and regenerative medicine, now face an uncertain future. The loss of indirect funding could delay or halt up to 600 ongoing clinical trials at institutions like Johns Hopkins alone (JHU, 2025).
How Lonrú Supports Academic Translational Efforts
In an era where public funding faces unprecedented change, technology transfer offices (TTOs) must adapt. Lonrú Consulting provides strategic tools to enhance translational readiness in CGT, ensuring academic programs can efficiently navigate regulatory, funding, and commercialization hurdles.
Tools for Translational Readiness: AI-driven tools to de-risk preclinical to clinical translation for academic labs.
Market Positioning for Translational Centers: Interactive tools to identify funding opportunities and investor alignment, optimizing commercialization potential.
Sustainable Financial Models: Advisory support for securing diversified funding, including philanthropic partnerships, venture investments, and alternative grant strategies.
As the impact of policy changes over funding are realised, Lonrú remains committed to supporting the academic innovation ecosystem—helping institutions turn research into real-world therapies and ensuring that promising CGT advances do not languish in the 'Valley of Death.'
Final Thoughts: Translational Research as a Pillar of CGT Sustainability
As global governments and research institutions refine their CGT strategies, translational research must remain a priority. Whether through RISE-inspired frameworks or adaptive funding models, securing the future of CGT requires sustained investment and infrastructure support. Institutions, industry partners, and policymakers must work together to uphold the critical role of academic translational centers.
References
JHU. (2025). Our essential research partnership with the NIH. Office of the University President and Office of Johns Hopkins Medicine. Email communication
Lonrú Consulting. (2025, January 29). UK Cell and Gene Therapy Clinical Trials To Date. Interactive Dashboards. https://www.lonruconsulting.com/interactive-dashboards
Lonrú Consulting. (2025, February 5). Building an All-Island CGT Strategy: Ireland’s Opportunity to Deliver Cell and Gene Therapy. Lonrú's Lens. https://www.lonruconsulting.com/blog-1-1/building-an-all-island-cgt-strategy-irelands-opportunity-to-deliver-cell-and-gene-therapy
Naresh, N. U., Hammill, D., Kessel, M., Hajjar, R. J., & Yozwiak, N. (2025). As new cell and gene therapies emerge from academia, we must RISE to the opportunity. Nature Biotech, 43(January 2025), 143-146. https://www.nature.com/articles/s41587-024-02520-9
NIH. (2025, February 7). Supplemental Guidance to the 2024 NIH Grants Policy Statement: Indirect Cost Rates. Office of The Director, National Institutes of Health. https://grants.nih.gov/grants/guide/notice-files/NOT-OD-25-068.html
