Our Pipeline

The most common genetic form of ALS, caused by the expansion of a six base pair
sequence in the gene C9ORF72 that is toxic to motor neurons

• 4,000 C9ORF72 ALS patients in the US/UK/EU
• Typical survival is 3-5 years after symptom onset

No specific treatment available for C9ORF72 ALS and approved drug treatments provide

limited benefit and do not stop the underlying neurodegeneration

Excise the expanded repeat from the C9ORF72 gene, eliminating the underlying cause of the disease

Halt the progression of the disease and provide a functional cure to C9ORF72 mutation carriers

The most common genetic form of FTD, expansion of the C9ORF72 hexonucleotide repeat results in the death of pyramidal neurons in the cortex of the brain.

• 12,000 C9ORF72 ALS patients in the US/UK/EU
• Typical survival is 7-13 years after symptom onset

There are no FDA-approved disease-modifying treatments or cures available for frontotemporal dementia (FTD)

Excise the expanded repeat from the C9ORF72 gene, eliminating the underlying cause of the disease

Halt the progression of the disease and provide a functional cure to C9ORF72 mutation carriers

The more common, sporadic form of ALS is marked by a characteristic TDP-43 and the inappropriate processing of downstream genes important to the survival of motor neurons.

• 80,000 ALS patients in the US/EU, 17,000 new patients every year.
• Typical survival is 3-5 years after symptom onset

Approved drug treatments provide limited benefit and do not stop the underlying neurodegeneration

Edit UNC13A, a downstream target of TDP-43, so it is properly processed and produces functional protein to protect motor neurons.

Halt the progression of the disease and provide meaningful life extension to ALS patients

The more common, sporadic form of ALS is marked by a characteristic TDP-43 and the inappropriate processing of downstream genes important to the survival of motor neurons.

• 80,000 ALS patients in the US/EU, 17,000 new patients every year.
• Typical survival is 3-5 years after symptom onset

Approved drug treatments provide limited benefit and do not stop the underlying neurodegeneration

Edit STMN2, a downstream target of TDP-43, so it is properly processed and produces functional protein to protect motor neurons.

Halt the progression of the disease and provide meaningful life extension to ALS patients

Ultra-rare genetic condition characterized by the build up of oxalate in the kidneys leading to progressive kidney damage and ultimately life-threatening kidney failure

• 1,000-2,000 patients in US/EU
• Roughly 30% of children and over 50% of adults already have complete kidney failure at time of diagnosis. There is a 28% mortality rate by the age of 50.

Currently approved therapy requires lifetime injections as frequently as once a month

Permanently knockout HOA1, a key component of the oxalate synthetic pathway, resulting in a reduction in oxalate formation

Provide a lifelong, durable cure for all PH1 patients