A few years ago, the Vall d'Hebron Research Institute (VHIR) introduced an innovation model that enabled it to successfully transmit research results to society, creating new treatments and solutions to unresolved clinical issues. This model has contributed to the Vall d'Hebron Campus consolidating itself as an international benchmark in health innovation.

This latest transaction is the biggest financial profit generated by a State Research Institute for a patent. The amount received by the VHIR is in excess of euros1 million and comes as a result of research conducted by Dr Ramon Martí, Head of the Research Group on Neuromuscular and Mitochondrial Diseases at the VHIR, and Dr Michio Hirano from Columbia University in New York. The profit obtained from licensing the patent will be entirely reinvested in the VHIR's research.

The therapy is for a rare disease, a research area that VHIR is firmly committed to pursuing. Research in this field focuses above all on improving diagnostic capacity for diseases that are often difficult to diagnose and on developing new treatments for those diseases. The fact that VHIR is part of the Vall d'Hebron Campus enhances its research capabilities, as it enables direct contact with patients.

Given that these types of diseases do not affect many patients around the world, obtaining funding for research can be difficult. In this case, however, the treatment was so promising from the early days that it sparked the interest of biotechnology companies and captured international investors from the American market and Israel, among others.

"Beyond the financial profit, the satisfaction for me personally and for my entire team dedicated to pre-clinical research is huge. For the first time, we are able to see the fruit of our research so directly leading to an effective treatment and, what's more, an effective treatment for a rare disease", says Dr Ramon Martí.

From research to patients

In 2013, doctors Ramon Martí (VHIR) and Michio Hirano (Columbia University) discovered a new treatment for a mitochondrial disease caused by TK2 deficiency (TK2d). The TK2 gene codifies the enzyme thymidine kinase 2, a key protein needed for the maintenance of mitochondrial DNA and the correct cell function. They then licensed the patent to the company Modis Therapeutics, based in California in the United States, precisely to develop this therapy.

Modis Therapeutics raised an initial sum of over $30 million to advance development of the new treatment for a mitochondrial disease, already known at this stage as MT1621.MT1621 is a deoxynucleoside combination therapy undergoing the research clinical phase. The purpose of the therapy is to compensate for the underlying biochemical deficit associated with TK2 deficiency.

As a study published in the journal Annals of Neurology showed, the treatment significantly improves the course of the disease. In October 2019, Zogenix, an American pharmaceutical company that leads the development of innovative therapies for rare diseases, acquired Modis Therapeutics, Inc. for euros250 million with the aim of bringing this therapy for patients with TK2 deficiency to the market. A clinical trial involving 40 patients is currently underway.

According to Dr Joan Comella, Director of the VHIR, "we have the science, talent and knowledge to work alongside the best academic organisations to promote the creation of biotechnology companies in the world's leading biomedical clusters, and to engage large specialist companies and investors". "This case study shows that we are competitive in health innovation at an international level and encourages us to continue being competitive", he concludes.