Precision Gene Therapy

From gene transfer to precision correction

Conventional gene therapy demonstrated that delivering genetic information can durably modify biological function.

New genome-editing technologies now make it possible to act directly on endogenous genetic information.

This changes the role of the vector.

For many precision-editing applications, the vector does not need to establish persistent expression. It needs to deliver sufficient editing activity, to the appropriate cells, for a defined period of time.

The therapeutic effect can then persist independently of the vector.

Transient editor. Durable correction.

For genome engineering requiring the introduction or replacement of larger genetic sequences, complementary DNA-based strategies may remain necessary.

The objective is therefore not to impose one delivery modality, but to match the vector architecture to the genomic intervention.

RNA-ONE for Precision Gene Therapy

GEG Tech’s RNA-BEV platform for direct in vivo genome-editor delivery

RNA-ONE is GEG Tech’s proprietary RNA-BEV technology for transient delivery of functional RNA.

Its modular architecture combines three dimensions that are becoming central to in vivo precision gene therapy:

Cargo. Tropism. Time.

This combination makes RNA-ONE particularly relevant to the transition from ex vivo genetic manipulation toward direct in vivo precision therapy.

Preclinical proof of concept

GEG Tech has already demonstrated the ability of its RNA-vector technology to deliver genome-editing machinery in vivo.

In collaboration with researchers at the University of California, Irvine, an all-in-one RNA vector carrying a genome-editing system was evaluated in the rd12 inherited retinal disease model associated with RPE65 deficiency.

Following local administration, genome correction was associated with a substantial recovery of retinal function, measured by electroretinography.

This program establishes a key principle for precision gene therapy:

a transient RNA-delivered editor can generate a durable functional consequence in vivo.

It also provides a technological foundation for adapting RNA-ONE to other editors, genetic targets and tissues.

Designed for in vivo development

RNA-ONE is not restricted to a single editor or disease model.

The platform can be adapted around the requirements of a partner program through modulation of the RNA cargo, particle envelope, route of administration and expression window.

Its in vivo profile has also been characterized preclinically.

Following local administration, spreading beyond the injection site remains limited.

Following systemic administration, RNA-ONE shows a controlled biodistribution profile, with limited accumulation in major filtering organs such as the liver.

Combined with an engineerable envelope, these properties provide complementary ways to control which cells receive the biological instruction and where it acts.

For direct in vivo therapies, delivery is no longer simply a formulation step.

It becomes part of the therapeutic design.

From platform validation to partner programs

RNA-ONE has moved beyond the stage of a technology concept.

The platform has already been validated for functional RNA delivery, evaluated in multiple in vivo settings, used to carry genome-editing machinery, and associated with functional rescue in a disease model.

GEG Tech can therefore engage RNA-ONE directly in partner-driven precision gene therapy programs.

Development can start from an existing editing system, genetic target or therapeutic concept and focus on adapting the delivery architecture to the required cargo, cell population, tissue and administration route.

The objective for industrial partners is to build on an existing vector platform and preclinical knowledge base, rather than starting a new delivery technology from discovery.

Technology maturity

A development platform for partners

GEG Tech operates through a collaborative studio-lab model.

For precision gene therapy programs, RNA-ONE can be integrated into industrial development around vector design, cargo adaptation, tropism engineering, preclinical evaluation and translational planning.

The collaboration can begin from a partner’s existing genome-editing asset and progress toward an application-specific RNA-ONE configuration.

Bring the editor. We engineer the delivery strategy around the biological objective.

Evidence & milestones

RNA-ONE development in precision gene therapy has progressed through successive technological and preclinical milestones, from genome-editor vectorization to functional in vivo proof of concept.

Platform milestone

New mRNA vectors for next-generation genome editing

October 2024

GEG Tech announced the development of its RNA-vector technology for advanced genome-editing systems and the initiation of evaluation programs with leading genome-editing research groups such as University of California or The broad institute of MIT and Harvard.

Read the announcement

Preclinical milestone

Retinal gene therapy: visual cycle restored in blind mice

January 2025

GEG Tech and the University of California, Irvine reported in vivo delivery of genome-editing machinery in the rd12 inherited retinal disease model, associated with substantial restoration of retinal function.

Read the press release

In the media

Le Parisien — January 2026

A feature on GEG Tech, RNA-vector delivery and the development of precision gene therapy with the University of California, Irvine.

Read the article