New vectorized antibody treatment for HD
Our program in Huntington’s Disease (HD) has the potential to significantly delay disease progression and preserve quality of life in HD patients by targeting the underlying cause of the disease in a novel way.
What is Huntington’s disease
Huntington's disease is a rare, inherited neurodegenerative disease characterized by the progressive loss of neuronal cells in the brain. Disease symptoms include movement, cognitive and psychiatric problems. Over time symptoms worsen and eventually lead to death. There is no cure for HD and current treatments aim to reduce disease symptoms but do not address the cause of the disease.
The role of huntingtin (HTT) in HD
The huntingtin (HTT) protein is essential for neuronal cell development and health. In people with HD however, one of the huntingtin gene copies is mutated and causes mutant HTT protein to misfold and build up (aggregate) in the brain. This process is toxic and leads to loss of neurons and the onset of HD. A potential medicine for HD should remove toxic mutant HTT but leave enough normal HTT protein to perform its essential function.
Vectorized antibodies to treat neurodegeneration in HD
We believe vectorized antibodies hold great promise for treating HD. A vectorized antibody consists of two parts. The first is the transgene that carries the instructions for a cell to make the therapeutic antibody. The second part is the vector into which the transgene is packaged and delivered to the brain cells. We use AAV vectors, the same delivery method used for gene therapies.
What is VTx-003
VTx-003 has the potential to significantly delay disease progression and preserve quality of life in people with HD. VTx-003 is a vectorized antibody that consists of a vector and the transgene that carries the instructions for a cell to make the therapeutic antibody. The therapeutic antibody selectively binds mutant HTT (and not normal HTT) with the aim to clear toxic mutant HTT and restore neuronal health. VTx-003 is currently in discovery phase.