Researh & development

Development of a New Drug for Cancer Therapy


Our R&D program focuses on the activities required to develop a novel drug for cancer treatment, based on the original work of Dr. Luis Burzio and his team, and to obtain FDA approval to start clinical studies in humans. These activities are carried out under a Technology Transfer Agreement between the start up company Andes Biotechnologies, located in the Science and Business Park, and Fundación Ciencia & Vida.

We completed the bulk GMP manufacturing of the drug substance Andes-1537, and the chemical characterization and their controls.

Formulated injectable Andes-1537 drug product in vials have been prepared, from the bulk drug, and acute and chronic toxicology studies in two animal species have been completed.

We also completed, in collaboration with Drs. Pamela Munster and Marc Shuman from UCSF, a Clinical Protocol describing the studies to be carried out at the University of California in San Francisco (UCSF) with patients with advanced unresectable solid tumors that are refractory to standard therapy.

By the middle of 2015, all of the above studies and results were summarized in an Investigational New Drug application sent to the FDA and approved in September 2015. The FDA approved Clinical Protocol together with the required Informed Consent and Investigators Brochure documents were also reviewed and approved by the UCSF Institutional Review Board composed of cancer clinical investigators from the university.

Phase-I clinical studies started at the Mount Zion Cancer Center Hospital in December 2015.


Valenzuela P., Hager G., Weinberg F. & Rutter W.J. The molecular structure of yeast RNA polymerase III. Demonstration of the tripartite transcriptive system in lower eukaryotes. Proc. Natl. Acad. Sci. 73, 1024. (1976) | PDF | Abstract

Valenzuela P., Venegas A., Weinberg F., Bishop R. & Rutter W.J. Structure of yeast phenylalanine tRNA genes. An intervening DNA segment within the region coding for the tRNA. Proc. Nat. Acad. Sci. 75, 190 (1978) | PDF | Abstract

O’Farrell P.Z., Cordell, B., Valenzuela P., Rutter W.J. & Goodman H.M. Structure and processing yeast precursor tRNAs containing intervening sequences Nature 274, 438-445 (1978) | PDF | Abstract

Valenzuela P., Gray P., Quiroga M., Zaldivar J., Goodman H.M. & Rutter W.J. Nucleotide sequence of the gene coding for the major protein of hepatitis B surface antigen. Nature 280, 215 (1979) | PDF | Abstract

Valenzuela P., Medina A., Rutter W.J., Ammerer G. & Hall B.D. Synthesis and assembly of hepatitis B virus surface antigen particles yeast. Nature 298, 347 (1982) | PDF | Abstract

Brake A.J., Merryweather J.P., Coit D.G., Heberlein U.A., Masiarz F.R., Mullenbach G.T., Urdea M.S., Valenzuela P., & BarrP.J. Alpha-factor directed synthesis and secretion of mature foreign proteins in S. cerevisiae. Proc. Natl. Acad. Sci. USA 8l, 4642-4646 (1984) | PDF | Abstract

Chien D., Arcangel P., Medina M.A., Coit D., Baumeister M., Nguyen S., George-Nascimento C., Gyenes A., Kuo G. and Valenzuela P. Use of a novel Hepatitis C major epitope chimeric polypeptide for the diagnosis of HCV viral infection. J. Clin. Microbiol., 37 (5), 1393-1397 (1999) | PDF | Abstract

Aguayo, C., Riquelme, J., Urrejola, C., Valenzuela, PDT. & Silva, E. Bchex a virulence gen of Botrytis cinerea: characterization and functional analyses. Journal of General Plant Pathology 77: 230-238 (2011) | PDF | Abstract

Villota, C., Campos, A., Vidaurre, S., Oliveira, L., Bocardo, E., Burzio, V.A., Varas, M., Villegas, L., Villa, L.L., Valenzuela, P.D.T., Socías, M., Roberts, S. y Burzio L.O. Expression of ncmtRNA is modulated by high risk HPV oncogenes. J. Biol. Chem. 287: 21303-21315 (2012) | PDF | Abstract

Barriga GP, Cifuentes-Muñoz N, Rivera PA, Gutierrez M, Shmaryahu A, Valenzuela PD, Engel EA. First detection and complete genome sequence of Deformed wing virus in Chilean honeybees. Virus Genes. 45(3):606-9. (2012) doi: 10.1007/s11262-012-0791-0 | PDF

Leroch M, Kleber A, Silva E, Coenen T, Koppenhöfer D, Shmaryahu A, Valenzuela PD, Hahn M. Transcriptome profiling of Botrytis cinerea conidial germination reveals upregulation of infection-related genes during the prepenetration stage. Eukaryot Cell. 12(4):614-26. (2013) doi: 10.1128/EC.00295-12 | PDF

Lolas M, Valenzuela PD, Tjian R, Liu Z. Charting Brachyury-mediated developmental pathways during early mouse embryogenesis. Proc Natl Acad Sci U S A. 111(12):4478-83 (2014). doi: 10.1073/pnas.1402612111 | PDF

Shmaryahu A, Carrasco M, Valenzuela PDT. Prediction of bacterial microRNAs and possible targets in human cell transcriptome. J Microbiol. 52(6):482-9 (2014). doi: 10.1007/s12275-014-3658-3 | PDF

Varas-Godoy M, Lladser A, Farfan N, Villota C, Villegas J, Tapia JC, Burzio LO, Burzio VA, Valenzuela PDT.In vivo knockdown of antisense non-coding mitochondrial RNAs by a lentiviral-encoded shRNA inhibits melanoma tumor growth and lung colonization. Pigment Cell Melanoma Res. 31(1):64-72 (2018). doi: 10.1111/pcmr.12615. Epub 2017 Oct 21.PMID: 28707763 | PDF

Principal investigator

Pablo Valenzuela, PhD

+56 2 2367 2004