Next generation DNA constructs for the rapid and safe manufacture of AAV vectors for Regenerative Gene Therapy

Gene therapy is becoming an increasingly important method of treatment for a variety of major unmet medical

needs especially in the areas of inherited and rare diseases and diseases of the eye, conditions which are life

threatening or significantly diminish quality of life. Adeno-associated virus (AAV) vectors are currently the

delivery vehicle of choice for gene therapy treatments but the advancement these treatments into clinical trials

is currently hampered by the time and expense required to manufacture these vectors.

The proposed collaboration between Cobra and Touchlight will develop a fast and less expensive route to

manufacture of AAV vectors, which will enable the acceleration of more potential products into clinical testing.

This in turn will increase the chances of treatment being developed for a whole range of these currently

intractable diseases.