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Amyotrophic Lateral Sclerosis (ALS) & Frontotemporal Dementia (FTD)

About ALS & FTD
ALS is an orphan disease characterized by progressive muscle weakness and loss of ability to speak, eat, move or breathe.

FTD is a progressive frontal / temporal cortex disease associated with behavior and personality changes, emotional problems, difficulty communicating, working or walking.

The median survival for ALS is 3 years and 9 years for FTD. When ALS and FTD present as comorbidities, survival is markedly reduced.

Amyotrophic Lateral Sclerosis (ALS) & Frontotemporal Dementia (FTD)

Why TPN-101?
TDP-43 proteinopathies are found in over 90% of ALS patients and approximately 40% of FTD patients. Loss of nuclear TDP-43 leading to increased LINE-1 expression and reverse transcription is enriched in ALS or FTD patients with a mutation of the C9ORF72 gene and associated with neuroinflammation and induction of Type 1 interferons. Preclinical evidence shows blocking LINE-1 reverse transcription with TPN-101 mitigates neurotoxic pathology.