“Increased localization of APP‐C99 in mitochondria‐associated ER membranes causes mitochondrial dysfunction in Alzheimer disease” EMBO J. Schon, Ph.D.
Patent Information:
Patent Pending (US20220395486). This technology reduces a patient’s level of endoplasmic reticulum-mitochondrial-associated membrane (ER-MAM) localized APP-C99 in cells using a variety of different pharmacological approaches, including but not limited to γ-secretase activators.
Therapeutic for other neurodegenerative disorders, including Welander distal myopathy (WDM), a rare autosomal dominant disorder caused by a missense mutation in the human TIA1 gene. Small molecules penetrate the CNS and pass through cell membranes (unlike main competitors, which are antibody-based). Most current anti-tau therapies in development are immunotherapies, which have thus far been ineffective, in large part because of poor antibody penetration into the CNS and poor membrane permeability.