The opportunity
Genomic therapies are transforming medicine, there are still many challenges to overcome to achieve efficacy, targeting and delivery, manufacturing precision, safety, and cost. The new Centre will harness AI-driven Engineering Biology to design fit-for-purpose advanced genomic therapies for targeted delivery, while simultaneously de-risking their manufacturing.
Our vision
The Advanced Genomic Therapies Centre (AGTC) builds on UCL’s major success in gene editing and brings together our strengths in therapeutic design, delivery, manufacturing and clinical translation.
It will provide a unifying framework and end-to-end translational ecosystem to accelerate the journey from diagnosis and molecular design to first-in-human studies, with patient impact at its core.
We will help address major global healthcare challenges — including brain disorders, cancer, and mental health conditions — driven by advances in AI, Engineering Biology, Advanced Manufacturing, and nucleic acid technologies.
Our work will focus on innovations in tissue and cellspecific targeting, scalable manufacturing, rapid analytics (including diagnostics and biomarkers), and the development of new technologies, models, and AI.
Our focus areas
The centre tackles the key barriers to delivering genetic therapies at scale by connecting the full pathway from discovery to early clinical trials, bringing together expertise in therapeutic design, manufacturing, pre‑clinical testing and regulation to de‑risk programmes earlier.
Modalities - gene and nucleic-acid therapies.
Overcoming barriers in:
• Efficacy and potency
• Targeting and delivery
• Safety and cost
• Supported by analytics, manufacturing and AI
Discovery to delivery
• Discovery to first-in-human trials
• Therapeutic design and development
• Pre-clinical testing and regulation
• Early clinical delivery
• Manufacturing process design and optimisation
• formulation design
• Patient and Public Involvement and Engagement (PPIE)
• Diagnostics development
• Disease model development
Areas of high unmet need:
• Neurodegeneration (dementia, ALS, Parkinson's)
• Brain circuit disorders (epilepsy, schizophrenia)
• Solid tumors
Get Involved
We would love to hear from potential collaborators, partners and funders. Find out more about opportunities to get involved below
- Co-develop and de-risk platforms and programmes through early collaboration and targeted problem solving (delivery, safety, cost, scale up).
- Find out more about routes to partnership including PhDs, KTPs, research contracts, facilities access and translational programmes.
- Opportunities for partnership-building supported through dedicated relationship development and showcase activity as AGTC evolves
- Take part in high-impact, challenge-led programmes, addressing major unmet clinical needs
- Find out more about how our work directly impact patients
- Increased readiness for large collaborative calls through cross-disciplinary convening and translation support.
- Opportunities to accelerate “moonshots” and enable new facilities for emerging technologies as investment grows.
Find out how your gift could support the Advanced Genomic Therapies Centre (AGTC). Get in touch with Sarah Medd-Phillips, Associate Director of Development (Health) at s.medd-phillips@ucl.ac.uk, and visit our website at https://www.ucl.ac.uk/giving to find out more about Here, it will happen, our most ambitious philanthropic and engagement campaign to date.
- An opportunity to work cross-faculty to connect discovery science with developability, regulatory and early clinical translation.
- Take part in challenge-led workshops and match-making
- Access feasibility study resources and expert support.
- Training opportunities (doctoral training, CPD, exchanges, mentoring), open to academics from any discipline seeking advice on aligning their research with end-to-end clinical pathways.
- Get involved in shaping research through patient and public involvement, engagement events and partnership activity.
- Help ensure new therapies are developed with public perspectives, patient needs and real-world benefit in mind.
Leadership and advisory
Advisory
AGTC is one of the strategic opportunity areas identified within UCL’s Health Strategy, supporting UCL’s ambition to deliver meaningful improvements in health outcomes through interdisciplinary, research-led innovation.
AGTC is being shaped by senior academic, clinical and translational leaders from across UCL and partner NHS organisations. An internal advisory group brings together expertise spanning key disciplines and translational enablers, and an external advisory board is being established to provide strategic guidance and alignment with national priorities.
Leadership
Dr Michael Booth, Faculty of Mathematical & Physical Sciences (Chemistry)
Professor Paul Dalby, Faculty of Engineering (Biochemical)
Professor Suzanne Farid, Faculty of Engineering (Biochemical)
Professor Dimitri Michael Kullmann, Faculty of Brain Sciences (Institute of Neurology)
Professor Manju Kurian, Faculty of Population Health Sciences (Institute of Child Health)
Professor Gabriele Lignani, Faculty of Brain Sciences (Institute of Neurology)
Professor Francesco Muntoni, Faculty of Population Health Sciences (Institute of Child Health)
Professor Zoë Waller, Faculty of Life Sciences (School of Pharmacy)
Professor Haiyan Zhou, Faculty of Population Health Sciences (Institute of Child Health)