Background
Social impairment is frequently associated with mitochondrial dysfunc on and altered neurotransmission. Although mitochondrial func on is crucial for brain homeostasis, this has not yet been exploited to develop therapies for neurodevelopmental disorders.
Tech Overview
Using CYFIP1 deficiency, a key interactor in Fragile Mental Retarda on with a drosophila model, researchers have demonstrated altera ons in mitochondrial metabolism that causes observed social behavior deficits. Increased mitochondrial ac vity causes GABA sequestra on in the mitochondria, reducing GABAergic signaling and resul ng in social deficits.
Pharmacological and gene c manipula ons of mitochondrial ac vity or GABA signaling corrects the observed abnormali es. This could be done by modula ng the newly discovered mitochondrial transport, Aralar, that sequesters GABA upon increased mitochondrial ac vity.
Further Details: https://linkinghub.elsevier.com/retrieve/pii/S009286742030221X (Cell, 2020).
Stage of Development
Technology Readiness Level (TRL): 3.
Benefits
This newly discovered GABA transporter has not yet been exploited as a target for drug discovery to treat social impairment. There is tremendous poten al for drug screening using this target.
Applica ons
Therapeu c target for neurodevelopmental disorders such as:
- Autism spectrum disorder
- Schizophrenia
- Fragile X syndrome
Opportunity
PACTT offers to grant exclusive or non-exclusive license to industrial partners able to develop and commercialize the technology.
Patents
WO2021176098
