Protein Replacement: Gene Therapy

The immune system recognizes biologic and gene therapies as foreign, and responds by producing neutralizing antibodies against them. Neutralizing antibodies swiftly bind and inhibit biologics and gene therapies, which renders them therapeutically ineffective. Neutralizing antibodies are a major clinical challenge as repeated dosing of biologics and gene therapies, essential for continued therapeutic efficacy, becomes impossible.

So far, clinicians have relied on co-administration of immune suppressant regimens with biologics and gene therapies to overcome neutralizing antibodies; 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.