Tecnologies per llicenciar

VariantX – Potential novel gene therapy for ER-stress-related rare liver disorders


  • Once validated for one indication, it could be expanded to additional pathologies with ER-stress as a major hallmark.
  • This technology may be used alone or in combination with other therapies.
  • Treatment with these variants do not show the deleterious effects observed with the full gene.


The group is looking for collaborators to develop AAV and out-licensing opportunities.

Intellectual Property

PCT patent application filed on June 20, 2024.




IRB Barcelona:
Alba Olivares: alba.olivares@irbbarcelona.org

University of Barcelona:
Inma Íñiguez: iiniguez@fbg.ub.edu

More info: Naonet al. Science 2023


Endoplasmic Reticulum stress plays a key role in the development of a broad number of diseases of different etiologies with genetic and metabolic origins: diabetes, cardiovascular, cancer, aging, neurodegenerative diseases. The effective resolution or amelioration of ER-stress holds significant potential as a single or combined therapy for rare diseases lacking an efficient treatment. This technology has been validated in human in vitro models and in vivo mouse model.

The problem

  • ER-stress is a common hallmark of a broad range of diseases without effective therapeutic solution.
  • Liver is one of the most affected organs by ER stress.
  • AATD (Alpha-1 antitrypsin deficiency) disease is a genetic disease with limited therapeutic options.
  • WD gene replacement requires a boost to tackle the liver ER-stress in patients.

The solution

  • We have identified the therapeutic potential of variants of the Mitofusin 2 (MFN2) gene to restore the normal ER homeostasis.
  • We propose VariantX, a novel monogenic gene therapy based on these MFN2 variants with a primary focus on Wilson Disease and AATD Disease. Other potential indications: NAFLD, NASH, Acute kidney injury, renal fibrosis.

Current stage of development

The therapeutic potential of VariantX has been validated in human in vitro models and murine in vivo models of liver disease.


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Tags: Life Sciences, Salut i ciències de la vida


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