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HUNTINGTON'S DISEASE
(HD)
Clinical
Huntington’s Disease (HD) is an
incurable, dementing condition which affects 4-7/100,000
of population.
It is inherited as an autosomal dominant condition with
complete penetrance. Thus 50% of offspring of an affected
patient will also carry the mutant gene and succumb to
the disease. Genetic testing means affected patients are
often identified long before they become symptomatic, although
this hasn’t had a large impact on disease incidence.
There is no treatment for the underlying disease state.
DanioLabs Approach
There are a number of reasons which make
HD an important program for DanioLabs:
1. The treatments developed for HD may
also be relevant to other polyQ diseases.
2. The treatments developed for HD may
also be relevant to other “proteinopathies”,
including Alzheimer’s Disease and Parkinson’s
Disease.
3. HD can be identified presymptomatically,
unlike Alzheimer’s Disease, Parkinson’s
Disease, and indeed most other medical conditions, offering
an excellent
chance of slowing down the disease process long before
patients become symptomatic. In many neurodegenerative
diseases, symptoms do not become apparent until 80% of
neurons are lost. It would be much easier to slow down
disease progression when there are more neurons remaining.
4. The disease is monogenic and highly
penetrant, making the disease process easier to study.
5. A number of strong candidate pathological
pathways have been identified in vitro and in vivo.
DanioLabs approach involves developing
compounds which alter biologically meaningful pathology
and endpoints.
Only compounds showing major biological effects are being
progressed, to facilitate the clinical trials.
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