The Need
There is a compelling need for new targets and new approaches in neurodegeneration to deliver truly disease-modifying therapies.
Neurodegenerative disorders are chronic conditions that destroy parts of the nervous system over time, especially the brain. They result in progressive loss of cognitive and motor function and eventually, death. The most common neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS), Motor neuron disease (MND), Huntington’s disease (HD), Spinal muscular atrophy (SMA), and Spinocerebellar ataxia (SCA).
It is estimated that more than 50 million people worldwide are affected by neurodegenerative diseases and this number is increasing as overall life expectancy increases. Most of these conditions are much more likely to happen in people over 65, but some conditions like Huntington’s disease and ALS often appear much earlier.
There are very few effective disease modifying therapies for these diseases.
Historically, research has been focussed on relatively few targets; ones which require down-regulating. However, there are many genetically validated neurodegeneration targets which have not been pursued due to lack of approaches for controlled upregulation.
Many of these targets encompass key neurodegenerative mechanisms, e.g. lysosomal function, lipid metabolism, microglial regulation, RNA metabolism, trophic support.
These targets require approaches which are controlled and with the best possible safety profile to enable treatments which can halt disease progression early. Existing gene therapy and gene editing approaches cannot deliver the specific controlled upregulation required to safely address these targets.