Evaluation licence for muscular dystrophy vector

27 April 2021

4basebio UK Societas
("4basebio", the "Company" or the “Group”)

Evaluation licence for muscular dystrophy vector

4basebio UK Societas (AIM: 4BB), the specialist life sciences group focused on exploiting intellectual property in the field of gene therapies and DNA vaccines, is pleased to announce it has signed research collaboration and evaluation license agreements with Royal Holloway University of London (RHUL). Under the terms of the agreements, the parties will collaborate to develop a HermesTM based non-viral vector incorporating a patented full length dystrophin gene with the view to developing a novel therapy for treatment of Duchenne muscular dystrophy (DMD).

4basebio will fund a two year collaboration with the Translational Genetic Medicine Laboratory at RHUL led by Dr. Linda Popplewell. 4basebio will take charge of the design and development of muscle targeting HermesTM vectors whilst the team at RHUL will be responsible for in vitro and in vivo validation of the vectors and evaluate their efficacy for treatment of muscular dystrophy in appropriate models. The vectors will incorporate RHUL’s patented, highly expressive full length dystrophin gene.

Muscular dystrophies (MD) are a group of inherited genetic conditions that gradually cause the muscles to weaken, leading to an increasing level of disability.  MD is a progressive condition, which often begins by affecting a particular group of muscles, before affecting the muscles more widely. Some types of MD eventually affect the heart or the muscles used for breathing, at which point the condition becomes life-threatening. Successful completion of this project will pave the way to further progress the vector to a potential gene therapy for DMD, the most common of the MDs, through pre-clinical proof of concept.

The project is expected to extend over two years; the terms of a commercial licence have also been agreed between the parties, which the Company has an option to exercise, should this first stage project prove successful. 

Dr Heikki Lanckriet, CEO and CSO for 4basebio noted: “We are delighted to have signed these agreements with Royal Holloway and are very pleased to be working with Dr Popplewell who is a world leading expert in the development of novel treatments for DMD.  Our Cambridge team is looking forward to collaborating with the Popplewell Research Group in developing a non-viral based delivery system capable of delivering full length genes to muscle cells.”

Dr Linda Popplewell of RHUL has said: “Gene therapies approved for DMD are mutation-specific and those undergoing clinical testing deliver shortened versions of the dystrophin gene with limited therapeutic potential. This exciting collaboration provides a fantastic opportunity to develop a therapy applicable to all patients and capable of restoring full length dystrophin protein expression.”

This announcement contains inside information for the purposes of the UK Market Abuse Regulation.

For further enquiries, please contact:

4basebio UK Societas         +44 (0)12 2396 7943
Heikki Lanckriet

Nominated Adviser                 +44 (0)20 7213 0880
Cairn Financial Advisers LLP
Jo Turner / Sandy Jamieson

Broker                            +44 (0)20 7220 0500
finnCap Ltd
Geoff Nash/Richard Chambers/Charlotte Sutcliffe


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