Cancer treatment using antisense oligonucleotides to modulate alternative splicing

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Lung cancer is the leading cause of cancer mortality, accounting for nearly one in five cancer deaths. The most frequent subtype of this cancer is lung adenocarcinoma (40%), for which there is still no effective treatment. An innovative pharmacological approach to tackle tumour progression is a technology based on antisense oligonucleotides, which can correct alternative splicing alterations found on cancer.


  • To bring a new-targeted therapy for lung adenocarninoma to the market, based on antisense oligonucleotides (AON) acting selectively on key cancer related genes, an emerging target for cancer treatment.

Problem to Solve

Lung adenocarcinoma has high incidence and mortality rates, and is an important and challenging objective for innovative treatments. The lack of effective therapies and the resistance that has been acquired to existing treatments are obstacles that have yet to be overcome. To make progress on developing lung cancer treatments, new drugs should be improved in terms of specificity and side effects.


This project uses an innovative AON approach to target lung cancer. The use of a specific set of AON, that correct the alternative splicing of key cancer-related genes, has shown antitumor properties in a mouse model of lung cancer.

Level of Innovation

This is the first therapy for lung cancer that uses splicing-modifying AONs, paving the way for other potential cancer treatments based on this technology.


Project Leader. Postdoctoral researcher

Jordi Hernández

Centre for Genomic Regulation

Project leader

ICREA Professor

Juan Valcárcel

Centre for Genomic Regulation

ICREA Research Professor

Manuel Serrano

Institut de Recerca Biomèdica de Barcelona

Senior Business Development Manager

Silvia Tórtola

Centre for Genomic Regulation

Head of Technology and Business Development Office

Pablo Cironi

Centre for Genomic Regulation


CEO at Aelix Therapeutics

José Luis Cabero

Aelix Therapeutics & Symbiokraft


Obra social “la Caixa”
Caixa Capital Risc
CI.V 17

Scientific Area

Biochemistry and Molecular biology

Business area


Research center

Centre for Genomic Regulation