We are building a pipeline of transformative investigational therapies for neurodegenerative diseases of significant unmet medical need. Our gene therapies target the genetic drivers, modifiers, or pathways of monogenic and complex neurodegenerative diseases. Our neuroanatomy-led approach to viral vector distribution seeks to deliver our therapies to the right place to maximise their potential.
Frontotemporal dementia (FTD) is one of most common forms of dementia in individuals under the age of 65 (second after Alzheimer’s disease). It is characterized by a rapid decline in behavior, executive function and/or language with an average of onset in the mid-50s. Patients experience a change in personality, apathy, loss of empathy, inappropriate social behavior, and disinhibition. Unfortunately, with time, patients require significant supportive care and survive seven to ten years after disease onset. Currently there are no approved medicines for the treatment of frontotemporal dementia.
FTD predominantly affects the frontal and temporal cortices, but with time spreads to other cortical and sub-cortical structures.
Accumulation of protein inclusions of either microtubule associated protein tau (MAPT) or transactive response DNA-binding protein 43 (TDP-43) in neurons and glia characterize FTD cellular pathology. Approximately 30-40% of FTD cases are familial and linked to autosomal dominant mutations in three genes; GRN (progranulin), MAPT (microtubule-associated protein tau), and a GGGGCC hexanucleotide repeat expansion in C9orf72 (chromosome 9 open reading frame 72).
FTD affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U.