Novel Gene Therapies for Refractory Epilepsy

With TRO support, researchers at UCL’s Queen Square Institute of Neurology and School of Pharmacy are leading the field in developing 3 novel gene therapy approaches to treat refractory epilepsy.

Unmet Need

Epilepsy affects approximately 600,000 people in the UK and despite optimal treatment 30% of these continue to have seizures. Gene therapy is arguably the most promising option to treat this unmet need.   

Solution

Researchers at UCL’s Queen Square Institute of Neurology (IoN) and School of Pharmacy (SoP) are leading the field in developing 3 novel gene therapy approaches to treat refractory epilepsy.    

1. EKC Engineered Potassium Channel Gene Therapy – MRC DPFS FUNDED  
2. DREADD Designer Receptors Activated by Designer Drugs  - UTF funded 
3. ADPD Activity Dependent Promoter Driven Gene Therapy  - TAS, UTF funded 

How We Helped

The TRO worked closely with the PI to develop the EKC strategy and support the application that secured the £ 2.1M award.
  
The EKC project is novel and cutting edge approach and has been extremely informative for the other programmes and the TRO has ensured that these learnings are communicated and acted on. This has necessitated many discussions with the MHRA which the TRO has led, to ensure as much data overlap as possible between the programmes. This is important from a 3Rs perspective and for rapid project progression.  

Where is the project now?

The EKC project is in the late preclinial stages with work completing at the CRO on safety and biodistribution studies and the vector being manufactured ready for the clinical trial which is planned for early 2022. The other 2 projects are at the planning GMP manufacture and biodistribution studies stage. Completion of this work will lead to CTA submission to run 2 clinical trials.