Aletheia Neuroenergetics
harnesses the power of gene therapy to treat neurodegenerative diseases
First-in-Class Gene Therapy
Aletheia's neuroenergetic gene therapies address impaired bioenergetics, which are the common denominator across the entire clinical spectrum of neurodegenerative disease.
Lead Indication: ALS
Aletheia's gene therapy is designed to target motor neurons and augment energy demands to slow the progression of Amyotrophic Lateral Sclerosis (ALS) & increase lifespan.
About ALS
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease caused by the loss of upper motor neurons of the neocortex and the lower motor neurons of the spinal cord.
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Patients suffer gradual loss of muscle mass, strength, and function in bulbar, respiratory, and voluntary muscle. Decline is inevitable, with death from respiratory failure typically 2 to 5 years after diagnosis for most patients. ​​
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There is currently no cure for ALS, and approved treatments provide limited benefit on disease progression or survival for a very finite portion of the clinical population.
Neuroenergetics
In ALS, there is universal disruption of metabolism in diseased neurons leading to loss of energy production, which contributes to neuronal dysfunction & degeneration.
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The augmentation of neuroenergetics has utility for ALS as well as numerous other neurological disorders.
Innovative Platform for CNS Diseases
Aletheia has the potential to leverage its leading technology platform to develop and commercialize products targeting other neurological disorders which require uninterrupted supply of energetic currency for metabolic homeostasis.
