GluGene Therapeutics announced the presentation of four independent studies at the Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) held in Spain. The presentations highlighted the company’s data-driven AAV engineering platform, built on large-scale experimental datasets and designed to expand the therapeutic scope of in vivo gene therapy while addressing critical biological and translational limitations.
The studies covered a broad spectrum of AAV engineering strategies, including approaches to expand the range of disease indications amenable to AAV-based gene therapies and to enhance key vector properties, such as evasion of pre-existing neutralizing antibodies, through machine-learning–guided design. These findings support a central conclusion: although payload design remains an essential component of gene therapy development, the ultimate success of in vivo gene therapies is primarily determined by the ability to engineer tissue-, cell-, and indication-specific AAV capsids.
According to the company, data presented at ESGCT demonstrated that distinct therapeutic areas impose fundamentally different performance requirements on engineered AAV capsids, highlighting the inherent limitations of one-size-fits-all vector designs. These results emphasize the importance of data-driven capsid engineering in improving in vivo delivery efficiency, safety, and manufacturability–key determinants of translational success and commercial viability.
A key highlight of the presentations was the disclosure of newly developed precision-targeted AAV vectors for neuromuscular indications. Based on these data, GluGene Therapeutics reported that its proprietary ATLAS (AAV Targeting through Library-driven Advanced Selection) and AIMING (AI-Mediated, Innovative, and de-Novo Gene Vector) platforms have demonstrated broad applicability across pulmonary arterial hypertension, cardiovascular diseases, retinal disorders, hearing loss, and neuromuscular systems.
Moon Sue Lee, Ph.D., Chief Executive Officer of GluGene Therapeutics, stated that the ATLAS and AIMING platforms enable the development of customized novel AAV capsids optimized for specific therapeutic indications and target cell populations. “By simultaneously advancing safety profiles and manufacturing productivity, we aim to strengthen our global competitiveness in engineered AAV capsids and accelerate the development of clinically viable gene therapies,” the CEO said.
Through its continued emphasis on data-driven vector design and translational engineering, GluGene Therapeutics is positioning the ATLAS and AIMING platforms as scalable and versatile foundations for next-generation in vivo gene therapy development.
