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Sonogenetic therapy for visual restoration : towards a first in human clinical study

Objective

Brain machine interface (BCI) aims at modifying brain activities by activating or inhibiting neurons. BCI based on electrodes have often failed because they generate fibrotic reactions. The partners develop the sonogenetic therapy for the distant activation of neurons by ultrasounds (US) with very high spatiotemporal resolution. It relies on the ectopic expression of a mechanosensitive ionic channel via gene therapy to render neurons sensitive to US. A first application is to restore vision in blind patients by imprinting US images of visual scenes in the visual cortex.
After a first proof of concept (POC) in rodents, the partners have obtained convincing results in their NeuroSonoGene ERC synergy showing the distant activation of neurons in the visual cortex of non-human primates. The clinical transfer is not included in this ERC synergy. Therefore, this SonoClin project aims at developing the path toward a first in human for sonogenetic therapy to restore vision. To avoid a too complex implantation of an ultrasound stimulator, this first in human proposes: 1) to open the skull on the visual cortex for injecting AAV viral vectors to allow expression of the mechanosensitive ionic channels in cortical neurons, 2) To stimulate the cortical neurons with US from an external stimulator applied on the skin through an artificial US translucent skull.
The SonoClin project aims at providing the POC for this external cortical sonogenetic stimulation through an artificial US translucent skull in non-human primates.
In parallel to this scientific objective, we will define all the steps and costs leading to the authorization for such a clinical trial: 1) the AAV manufacturing and safety evaluation, 2) the design of a US probe holder and clinical US stimulating machine, 3) the business plan for the creation of a company carrying the clinical trial.
The SonoClin project will establish the path for the clinical transfer of sonogenetic therapy in the context of visual restoration.

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Programme(s)

Topic(s)

Funding Scheme

HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

(opens in new window) ERC-2026-POC

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Host institution

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Net EU contribution
€ 150 000,00
Address
RUE DE TOLBIAC 101
75654 Paris
France
Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Links
Total cost
No data

Beneficiaries (1)