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

基因治疗正在成为一种越来越重要的治疗方法，用于治疗各种主要的未满足的医疗需求，特别是在遗传性疾病和罕见疾病以及眼病领域，这些疾病危及生命或显著降低生活质量。腺相关病毒（AAV）载体目前是基因治疗的首选运载工具，但由于缺乏制造这些载体所需的可扩展性，这些治疗进入临床试验的进展目前受到阻碍。Cobra和CPI之间拟议的合作将发展科学理解，允许开发可扩展的灵活工艺来制造AAV载体。这将使更多潜在产品加速进入临床试验，并最终成为新药。反过来，这将增加为一系列目前难以治疗的疾病开发治疗方法的机会。

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.