RESEARCH PROGRAM

Gene Therapy and Regulation of Gene Expression

"Thanks to gene therapy, we expect to obtain in the coming years a clear clinical benefit in patients with rare diseases."

DR. CRISTIAN SMERDOU PICAZO
DIRECTOR. GENE THERAPY AND REGULATION OF GENE EXPRESSION RESEARCH PROGRAM

The Gene Therapy and Regulation of Gene Expression Program at Cima aims to develop new therapeutic strategies for diseases that lack treatment or whose treatment is unsatisfactory.

We investigate to improve therapies developed for genetic diseases and cancer in order to reach their clinical translation, the implementation of new gene therapy technologies such as gene editing, the use of non-viral vectors to transfer DNA or RNA in vivo, or the development of new expression regulation systems, as well as the identification of new therapeutic targets based on lncRNAs or UPR-related processes.

The research team is fundamentally basic, but with a very translational vision and maintains close collaborations with teams from the Clínica Universidad de Navarra. In addition, the Gene Therapy and Regulation of Gene Expression program is a transversal program that collaborates very actively with the other programs of the Cima Universidad de Navarra. 

Currently, the Program has numerous national and international research projects, actively participates in European calls and maintains collaborations with biotechnology companies that represent a unique opportunity to continue advancing.

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Gene Therapy Research
Program Objectives


Development of new therapeutic strategies based on gene transfer for inherited and acquired diseases for which there is no cure or for which current therapy is not satisfactory.


Analysis of the function and mechanisms of action of long non-coding RNAs in the development of pathologies and their application in the development of new therapies.


Analysis of the role of UPR and post-translational modifications of proteins in the regulation of gene expression and their implication in the development of diseases and new therapies.

Cima gene therapy clinical trial

Our translational vocation moves us to investigate therapeutic alternatives that reach patients. This is the case of VTX-801, a drug originated at Cima for the treatment of Wilson's disease, with technology licensed to the biotechnology company Vivet Therapeutics.

Currently, the pharmaceutical company Pfizer Inc. and Vivet Therapeutics have the GATEWAY study underway, a Phase 1/2 clinical trial evaluating the efficacy of the TX-801 vector for the treatment of patients affected by this rare disease.

Our research groups

We seek to improve therapeutic alternatives for currently incurable diseases

Gene Therapy of Liver Diseases

Gene Therapy of Congenital Diseases with Neurological Affectation

 

Gene therapy of renal diseases and study of N-terminal acetylation of proteins

 

Long Non-Coding RNAs and Cancer Regulation

Gene Therapy for Cancer

Non-Coding RNAs in Hepatocarcinoma and Other Liver Diseases

Endoplasmic Reticulum Stress Response in Neurodegenerative Diseases

FROM THE LABORATORY TO THE PATIENT

Cutting-edge translational research

The research team of the Gene Therapy Program is fundamentally basic, but with a very translational vision and maintains close collaborations with teams from the Clínica Universidad de Navarra.


Clinical
translation

Improvement of therapies developed for genetic diseases and cancer to provide solutions for patients.


Transfer DNA or RNA in vivo

Implementation of new gene therapy technologies using gene editing or non-viral vectors.


Long non-coding RNAs (lncRNAs)

Development of new expression regulation systems, as well as the identification of new therapeutic targets.

<p>Fachada del Centro de Investigaci&oacute;n M&eacute;dica Aplicada (CIMA)</p>

Would you like to help us?

Thanks to the generosity of many people, the Cima Universidad de Navarra is a reality that strives to offer therapeutic solutions to achieve personalized medicine for patients.

Meet the research team

Scientific activity of the Gene Therapy and
and Regulation of Gene Expression Program