Development of an Industrial Manufacturing Platform for Adeno-Associated Virus (AAV) production to support Regenerative Gene Therapy products

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Gene therapy is becoming an increasingly important method of treatment for a variety of major unmet medicalneeds especially in the areas of inherited and rare diseases and diseases of the eye, conditions which are lifethreatening or significantly diminish quality of life. Adeno-associated virus (AAV) vectors are currently thedelivery vehicle of choice for gene therapy treatments but the advancement of these treatments into clinicaltrials is currently hampered by the lack of scalabilty needed to manufacture these vectors.The proposed collaboration between Cobra and CPI will develop the scientific understanding to allow scalableflexibile process to be developed to manufacture AAV vectors. This will enable the acceleration of morepotential products into clinical testing and ultimately new medicinces. This in turn will increase the chances oftreatment being developed for a whole range of these currently intractable diseases.