Protein Replacement: Gene Therapy

The immune system recognizes biologic and gene therapies as foreign, and inhibits biologics and gene therapies, which renders them therapeutically suboptimal or ineffective. This immune response is a major clinical challenge for repeated dosing of biologics and gene therapies, essential for continued therapeutic efficacy.

So far, clinicians have relied on co-administration of immune suppressant regimens with biologics and gene therapies to overcome the immune response; however, these immune suppressive regimens have limited usefulness and come with the high cost of severe side-effects and poor quality of life.

COUR is developing CNP-AAVC, a biodegradable nanoparticle encapsulating AAV protein replacement.