4basebio enters neurofibromatosis collaboration with two universities

The AIM-traded firm said the universities had been awarded almost $1m from the US-based Gilbert Family Foundation to fund a three-year research programmed, aimed at developing a “life-changing” gene therapy with applicability to all NF1 patients, and which would use a non-viral vector for targeted delivery of full length NF1 DNA.

It said it would support the project by designing and developing a non-viral ‘Hermes’ vector and ‘hpDNA; sequence as payload for UA and TU.

4basebio described NF1 as 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 could include learning difficulties, speech difficulties and skeletal malformations, heart and blood vessel problems, loss of vision, and severe pain.

NF1 is caused by mutations in the NF1 gene, which regulates the production of neurofibromin protein.

“We are very pleased to be able to support this research project and are delighted that this exciting collaboration between academia and industry has been recognised with the award from the Gilbert Family Foundation,” said chief executive and scientific officer Dr Heikki Lanckriet.

Reporting by Josh White at Sharecast.com.