ALS, which also known as Motor Neuron Disease or Lou Gehrig’s disease, is a fatal condition that leads to progressive muscle weakness, paralysis, and eventually respiratory failure. Most people are diagnosed around age 55 and survive only 2–5 years. Current treatments offer minimal benefit, often extending life by just a few months.
A mutation in the C9ORF72 gene is the leading inherited cause of ALS and a related disorder, FTD. While 90% of cases arise randomly, nearly all (97%) involve toxic TDP43 protein aggregation that is damaging to motor neurons. These aggregates disrupt nerve signals, causing muscle wasting and loss of mobility.
Crucible is developing a novel treatment that targets the root of this damage by blocking toxic proteins and protecting neurons. Our approach could help up to 97% of ALS patients and may also benefit those with other neurodegenerative diseases. Our goal is to stop disease progression, and the loss of motor neurons restore motor function.
Frontotemporal dementia (FTD) is the leading cause of dementia in people under 60, causing gradual decline in behaviour, language and thinking. Life expectancy after symptoms appear is 7–13 years.
Around 10,000 people live with FTD in the US, EU, and Japan, with 2,000 new cases yearly. There are no treatments to slow the disease – care focuses only on easing symptoms.
FTD often runs in families, with C9ORF72 mutations being the most common genetic cause. Crucible is developing a therapy to block the harmful effects of these mutations, aiming to slow or stop the disease and protect brain function.
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