Immunotherapy for Treatment of Neuromyelitis Optica (NMO)

Invention Novelty

This invention comprises compositions and methods for treatment of neuromyelitis optica (NMO) spectrum disorders using anti-aquaporin-4 (AQP4) antibody lacking effector function.

Value Proposition

Neuromyelitis optica spectrum disorders (NMOSD) is an inflammatory disease of the central nervous system characterized by severe, immune-mediated demyelination and axonal damage predominantly targeting optic nerves and spinal cord. A defining feature in most NMOSD patients is the presence of serum autoantibodies (NMO-IgG) against astrocyte water channel aquaporin 4 (AQP4). At present, the therapeutics options include only immunosuppressants, plasma exchange and B-cell depletion.

The present invention defining a disease-specific NMO treatment comprises the following:

• A reagent containing anti-aquaporin-4 antibody or an antigen-binding fragment lacking effector functions of an intact antibody.
• A variety of reagent administration options including intraocular, intra-arterial, subcutaneous or intravenous.
• The reagent can be administered chronically.
• Can be used in combination therapy with immunosuppressive or other drugs.
• The method can prevent or reduce the progression of NMOSD.

Technology Description

Researchers at University of California, San Francisco have generated a specific reagent for treatment of NMOSD comprising AQP4 antibody or an antigen binding fragment, lacking effector function. The effector function of the antibody can be abrogated by mutating or chemically modifying Fc region or Fab fragment lacking effector Fc region. The antibody can be optimized for higher affinity AQP4 binding by mutating the Fab region. Treatment with this novel and disease-specific reagent can reduce NMOSD progression leading to visual and motor deficit.

Looking for Partners

To develop & commercialize the technology as a specific treatment for neuromyelitis optica spectrum disorders.

Stage of Development

Pre-clinical

Related Materials

• Tradtrantip, L., H. Zhang, S. Saadoun, P. Phuan, C. Lam, M.C. Papdopoulos, J.L. Bennett and A.S. Verkman (2012). Anti-aquaporin-4 monoclonal antibody blocker therapy for neuromyelitis optica. Ann. Neurol. 71:314-322.

Data Availability

Mouse Model and ex-vivo data

Patent Status