4basebio PLC announced its collaboration with University of Alabama and Teesside University in a research project aimed at developing an effective treatment for Neurofibromatosis Type 1 (NF1). The universities have been awarded almost $1million from the US based Gilbert Family Foundation to fund a three-year research program aimed at developing a life-changing gene therapy with applicability to all NF1 patients and which will use a non-viral vector for targeted delivery of full length NF1 DNA. 4BB will support this project by designing and developing a 4BB non-viral Hermes™ vector and 4BB hpDNA™ sequence as payload for UA and TU.

NF1 is a genetic condition which causes usually benign tumours to develop anywhere in the nervous system, including the brain, spinal cord and nerves.  Complications of NF1 are many and can include learning difficulties, speech difficulties and skeletal malformations, heart and blood vessel (cardiovascular) problems, loss of vision, and severe pain.  NF1 is caused by mutations in the NF1 gene which regulates the production of neurofibromin protein.