SITE-SPECIFIC BRAIN THERAPEUTICS

Abstract: the present disclosure relates to an approach, referred to as regionally activated interstitial drugs (raid), that does not require viral vectors, but allows for tunable, noninvasive, long-term neuromodulation with small molecules. raid utilizes noninvasive delivery of an engineered protein enzyme into the brain, which then binds to the brain parenchyma and can locally convert a blood-brain-barrier (bbb)-permeable inert prodrug into an active neuromodulatory drug. as long as the raid enzyme is present in the parenchyma, localized neuromodulation can be achieved with systemic administration of the bbb-permeable prodrug even in the absence of the opened bbb. alternatively, gene delivery encoding the raid enzyme offers prolonged expression and precise spatial control, enabling long-term and adaptable neuromodulation.

Inventor(s): Jerzy Olgierd Szablowski, Zhimin Huang

CPC Classification: A61K38/51 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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