PRIORITY INDICATIONS
Motor Neuron Disease (NMD), also known as Amyotrophic Lateral Sclerosis (ALS), is a severe neurodegenerative disease with no disease-modifying treatment options.
This devastating disease leads to progressive loss of brain & spinal motor neurons, paralysis, and death, typically within 2-5 years from symptom onset.
FTD involves degeneration of frontal and temporal lobes of the brain leading to altered behavior, language, and executive function with death usually occurring within 8-10 years from symptom onset. There are no disease-modifying treatments available for FTD.
Genetic mutations in the form of repeat expansions in the C9ORF72 gene (i.e hexanucleaotide repeats or ‘rogue RNA’) are the most common genetic cause of ALS and FTD. Approximately 5,500 ALS and 10,000 FTD patients are living with the disease (USA, EU, Japan) with ∼1,500 and ∼2,000 new ALS and FTD cases per year, respectively.
This devastating disease leads to progressive loss of brain & spinal motor neurons, paralysis, and death, typically within 2-5 years from symptom onset.
FTD involves degeneration of frontal and temporal lobes of the brain leading to altered behavior, language, and executive function with death usually occurring within 8-10 years from symptom onset. There are no disease-modifying treatments available for FTD.
Genetic mutations in the form of repeat expansions in the C9ORF72 gene (i.e hexanucleaotide repeats or ‘rogue RNA’) are the most common genetic cause of ALS and FTD. Approximately 5,500 ALS and 10,000 FTD patients are living with the disease (USA, EU, Japan) with ∼1,500 and ∼2,000 new ALS and FTD cases per year, respectively.
UNMET MEDICAL NEED
The approved NMD/ ALS treatments have only a modest effect on life expectancy or progression of disability. Better treatment options are therefore urgently needed.
We are developing a highly differentiated approach to treating ALS and FTD that indirectly targets two types of rogue RNA strands (sense and antisense RNA). Others have effectively targeted and reduced one rogue RNA strand (sense only), but this approach was not efficacious in the clinic.
Our goal is to develop a disease-modifying treatment that halts neurodegeneration and enables patients to recover motor function and cognitive function in ALS and FTD, respectively.
Alongside our therapeutic development efforts, we are implementing a comprehensive biomarker strategy that will allow us to monitor target engagement in the clinic and could serve as an early signal of efficacy in our clinical trials.
We are developing a highly differentiated approach to treating ALS and FTD that indirectly targets two types of rogue RNA strands (sense and antisense RNA). Others have effectively targeted and reduced one rogue RNA strand (sense only), but this approach was not efficacious in the clinic.
Our goal is to develop a disease-modifying treatment that halts neurodegeneration and enables patients to recover motor function and cognitive function in ALS and FTD, respectively.
Alongside our therapeutic development efforts, we are implementing a comprehensive biomarker strategy that will allow us to monitor target engagement in the clinic and could serve as an early signal of efficacy in our clinical trials.
