Richard Fagan
BioPharm, Senior Leadership Team
Advanced Therapies - UCLB Technologies
It's in our genes
UCLB is the commercialisation company of UCL. We bring to market truly world-leading, world-changing technologies and innovations developed at UCL that have a real and positive impact on people’s lives.
Discover UCL Advanced Therapies
AAV Gene Therapy for Wilsons Disease
Wilson’s disease presents an ideal candidate for somatic gene transfer methodologies owing to its singular genetic root, ATP7B gene. By addressing approximately 30% of hepatocytes, the primary liver cells, this approach holds promise in mitigating Cu storage characteristic of Wilson’s disease patients.
AAV Gene Therapy for Diabetes
AAV gene therapy for diabetes, is a novel technology with the potential to treat diabetes type I through a stable endogenous expression of human insulin from the liver after one single administration.
Gene Therapy approach for treating Fabry disease
This technology employs a modified adeno-associated virus (AAV) that delivers a healthy version of the α-galactosidase A (GLA) gene to liver cells, ensuring the production of a functional α-galactosidase A (Gal A) enzyme. This is a one-time treatment approach that aims to cure Fabry disease.
For further information about the technologies here, get in contact with Dr Richard Fagan, Director of BioPharm.
ddPCR for Safety Monitoring in Gene Therapy (MEGA)
“MEGA” enables a quick and unbiased overview of gene editing outcomes after designer nuclease treatment in therapeutically-relevant cells.
LGI1 gene therapy for epilepsy
Gene therapy presents a promising alternative for treating drug-resistant focal epilepsy. LGI1, an extracellular protein is often downregulated in focal epilepsy. Over expression of LGI1 using an AAV vector can reduce neuronal and network excitability seen in epilepsy.
Lentivirally mediated mRNA delivery platform
This technology consists of a retroviral vector that has been engineered to enable direct expression and translation of the RNA vector genome on the target cell, bypassing the need for reverse transcription or integration into the target cell’s genome. This modification leads to enhanced translation of the RNA vector genome.
For further information about the technologies here, get in contact with Dr Caitriona O’Rourke, Senior Business Manager.
Allogeneic Müller-like Cells for the Treatment of Glaucoma
Müller cell therapy is a first in-class allogenic human embryonic stem cell derived therapy for glaucoma with the potential to improve visual function in patients with advanced disease.
For further information about this technology, get in contact with Dr Maria Mastrogiannaki, Business Manager.
Raman-enabled Bioreactor Monitoring
Raman spectroscopy can monitor a wide range of process parameters including metabolite concentrations, cell density and cell viability online. It achieves this by monitoring the cell culture with a laser. Monitoring these parameters in real-time can significantly reduce batch failures and reduce the number of process development runs required for a new therapeutic. It enables automated feed control strategies and could enable real-time product release in the future. Despite its potential, Raman bioreactor monitoring has seen limited application to date, due to performance challenges.
For further information about this technology, get in contact with Dr Alberto Gatta, Associate Business Manager.
LTR – the 4th generation lentiviral platform
The ‘4th -generation’ lentiviral platform developed by Dr John Counsell has demonstrated several advantages over the widely used 3rd -gen tech, with significantly enhanced safety features (in particular reduction of aberrant transcripts underlying potential clonal expansion/mutagenesis), much faster onset of expression, higher expression levels at similar doses, and higher payload capacity.
AAV-based gene therapy for Non-Ketotic Hyperglycinaemia
The AAV-based gene therapy approach to treat NKH, combined with the extensive pre-clinical data package, shows to have efficacy in both the systemic and neurological symptoms of this devastating disease.
For further information about the technologies here, get in contact with Dr Sara Garcia Gomez, Senior Business Manager.