This invention comprises compositions and methods for treatment of neuromyelitis optica (NMO) spectrum disorders using anti-aquaporin-4 (AQP4) antibody lacking effector function.
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:
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.
To develop & commercialize the technology as a specific treatment for neuromyelitis optica spectrum disorders.
Mouse Model and ex-vivo data
|Hong Kong||Issued Patent||1195000B||04/27/2018||2011-096|
|United Kingdom||Issued Patent||2699256||09/27/2017||2011-096|
|Germany||Published Application||2 699 256||02/26/2014||2011-096|
|Rep Of Korea||Published Application||2011-096|
Additional Patent Pending
NMO, NMOSD, AQP4, Water channel, Neuroinflammation, Autoantibodies, Immunotherapy, Antibody engineering