MTOR-independent activator of TFEB for autophagy enhancement and uses thereof

Overview

The present invention relates to a composition comprising an autophagy enhancement compound. It involves small molecules that activate the gene TFEB, enhancing autophagy and lysosome biogenesis. This can prevent the accumulation of toxic protein aggregates, making it useful in treating neurodegenerative diseases.

Commercialisation opportunities

technology licensing agreement

Problem addressed

To provide a small molecule compound having good blood-brain barrier permeability and potent TFEB-activating effects for the treatment of neurodegenerative diseases.

Innovation

It relates to a composition comprising an autophagy enhancement compound.
A compound that directly binds to and activates TFEB without inhibiting MTOR pathway, thus eliminating possible MTOR-associated complications.

Key impact

The compound of the present invention is a small lipid molecule with good blood-brain barrier permeability and potent TFEB-activating property
The chemical structure of the compound of the present invention is simple and it can be easily synthesized in large scale for pre-clinical and clinical studies
Compound of the present invention activates TFEB without inhibiting MTOR pathway, thus eliminating possible MTOR-associated complications in clinical trials.

Application

The present invention has a wide field of application in the treatment of lysosomal storage disorders and common neurodegenerative diseases.

Patent

China Patent Publication No.: CN113398106A
UK Patent No.: EP3113767
US Patent No.: US9,351,946
Hong Kong Patent Publication No. HK40058789A
Germany Patent NO.: EP3113767

Hong Kong Baptist University (HKBU)

Aspiring to be a leading, research-led, liberal arts University in Asia, Hong Kong Baptist University is committed to conducting world-leading investigations that extends the frontiers of knowledge, tackles global challenges and benefits the community at home and abroad.

Pursuing research excellence is one of the priorities of the University’s Institutional Strategic Plan 2018-2028. The University keeps enhancing its capacity and strength to produce world-class research with an aim to bring significant impact to the society.

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