Huntington’s disease
Research activity in Huntington’s disease focuses on gene therapy and HTT gene lowering strategies for adults with early manifest disease, with attention to striatal delivery and neurological degeneration.
- HTT gene lowering
- Striatal delivery
- Neurodegenerative disease
The clinical work in this area is centered on central nervous system targeting for movement disorder pathology associated with Huntington’s disease.
Amyotrophic lateral sclerosis
In amyotrophic lateral sclerosis, the sponsor is involved in genetic medicine approaches for SOD1-ALS, with interest in therapies directed at motor neuron disease and neuroprotection.
- SOD1-ALS
- Motor neuron disease
- Neuroprotection
This area reflects a therapeutic focus on intrathecal administration for disorders affecting motor function and neuromuscular decline.
Central nervous system gene therapy
The sponsor’s portfolio includes gene-based therapeutics designed for central nervous system disorders, with emphasis on targeted delivery to affected neural structures.
- Gene-based therapeutics
- Neural targeting
- Inherited neurological disorders
These studies are aligned with treatments aimed at modifying the underlying biology of rare neurological disease.
