ASKLEPIOS BIOPHARMACEUTICAL, INC. — Department of Health and Human Services STTR Phase I: Abstract We propose to initiate preclinical development of a gene therapeutic targeted to

ASKLEPIOS BIOPHARMACEUTICAL, INC. — STTR Phase I award from Department of Health and Human Services.

Amount
$270,008
Agency
Department of Health and Human Services · National Institutes of Health
Program / Phase
STTR · Phase I
Solicitation
PA14-072
NAICS
Place of performance
NC
Period

Description

Abstract We propose to initiate preclinical development of a gene therapeutic targeted to Alzheimer's Disease. The World Health Organization's and Alzheimer's Disease International's report on Dementia (2012) recognizes that the global burden of AlzheimerDisease is forecast to worsen significantly with prevalence predicted to double every 20 years. Interventions that could provide a modest delay in progression would provide a significant reduction in the high level care required later in the disease. ThisPhase I proposal describes an Adeno-associated viral vector-based metabolic intervention strategy to combat age-related cognitive decline in the 5xFAD model of familial Alzheimer's disease. A broad range of neurodegenerative disorders manifest abnormalities in the metabolism of the abundant amino acid derivative N-acetyl-L-aspartic acid (NAA). Fluctuations in NAA concentration within the brain are generally accepted as an index of oxidative metabolic integrity in neurons. NAA synthesis and catabolism are p