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   Infocentre

Published: 17 June 2016  
Related theme(s) and subtheme(s)
Frontier research (ERC)
Health & life sciencesBiotechnology  |  Genetic engineering  |  Major diseases
Research policySeventh Framework Programme
Success storiesHealth & life sciences
Countries involved in the project described in the article
Sweden
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ERC grantee and regional government to set up gene therapy centre

ERC grantee Professor Deniz Kirik's spin-off company will join forces with Skåne Regional Council in southern Sweden to build a specialised hospital and a state-of-the-art gene therapy centre, the parties announced on 8 October. The new facilities are expected to be operational by 2020. They will provide researchers unique opportunities for clinical trials, while patients will gain access to the latest treatment methods for Parkinson's disease and other illnesses.

Photo of two hands holding each other
© Ocskay Mark - fotolia.com

Parkinson's disease is a neurodegenerative brain disorder affecting people's ability to regulate their movements, body and emotions. There is currently no definite cure for it. Deniz Kirik, professor of neuroscience at Lund University in Sweden, implemented what the industry calls a disruptive technology: introduce harmless viruses into brain cells to deliver therapeutic genes in a controllable and personalized manner.

In 2009 he received an ERC Starting Grant to collect scientific evidence that such a treatment could reverse the disease’s symptoms and replace the lost brain functions in animals. As his project was making progress, three years later he applied for an ERC Proof of Concept grant to test the market potential of his findings. The grant enabled him to get help from business experts to start a new company, Braingene. He contacted venture capital firms, industry leaders, and he applied for patents.

Hard way from frontier research to clinical trials

Professor Kirik's research findings were promising both academically and commercially, but in this field the success is dependent on the ability to translate the research into clinical trials.

"This translation is much talked about but not as commonly realised," he said. "It is a difficult task and there are so many challenges that it erodes the energy and enthusiasm of those that carry the burden across what is commonly referred to as the “Valley of Death”."

The hurdles are even bigger for non-traditional drug candidates, especially gene therapies that require tests in hospitals. Usually, hospitals are not designed to handle such specialised activities and have no trained personnel to do it.

Partners in regional government

"So my goal was to engage in discussions with the Skåne Regional Council in southern Sweden, which is a self-governing administrative organisation responsible for healthcare provision," said Professor Kirik.

It paid off. In early October 2015 the regional council decided to form a jointly owned company with Braingene to build a specialised hospital dedicated to implementation of gene therapies. It's the first time in Nordic countries that the public sector engages in implementing a future clinical treatment facility with a small spin-off company. The facility will focus on early and later-stage clinical trials leading to new drug approvals, and will ultimately make these future therapies available for clinical practice.

Keeping top researchers in Europe

The regional authorities also gave Professor Kirik a mandate to establish a Centre for Gene Therapy under the direct management and control of the region. The centre will not only conduct pioneering clinical trials, taking cutting-edge technology to the clinics, but it will also train the doctors and nurses to meet the competence requirements of these highly specialized treatments. It has the potential to become a flagship project in brain diseases and in other conditions where gene therapy is becoming a reality, for example genetic forms of blindness or haemophilia.

"Our efforts here will help bring the most promising therapies faster to the patients," said Professor Kirik. “At the same time we hope they will also help keep frontline research in Europe, and encourage more international researchers and entrepreneurs to locate their operations in the region.”

Project details

  • Project acronym:TREATPD
  • Participants: Sweden (Coordinator)
  • Project Reference N° 242932
  • Total cost: € 1 508 940
  • EU contribution: € 1 508 940
  • Duration:November 2009 - October 2014

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