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Research 'made in Catalonia' to enamor investors

Increasing deal flow of projects ready for investment in the BioRegion: this is the main goal of the Investment Readiness Series, a new initiative from Biocat that periodically connects Catalan research projects with local and international investors. Some of the scientists participating in the first edition tell us about the projects they hope will enamor these investors.


This year, Biocat has kicked off The Investment Readiness Series, a series of meetings between scientists and investors that hopes to add value to the research projects through one-to-one mentoring and help venture capitalists, corporate investors and venture builders scouting for projects by bringing together top-notch research and establishing a long-term relationship with technology transfer offices (TTOs), leveraging the impact of their work on an ecosystem level.

The first gathering will be held this November, organized by Biocat in collaboration with Barcelona Tech City, Payment Innovation Hub, Catalan Biotechnology Reference Network (XRB), ACCIÓ and Johnson & Johnson, with support from the “la Caixa” Foundation. A second gathering will be held in London in December, where Catalan companies will get the opportunity to pitch their projects to investors from the London, Cambridge and Oxford ecosystem. A new call for healthtech projects will be launched soon. If you don't want to miss out on any of these calls, remember to subscribe to the Biocat newsletters.

Some of the projects that participated in the first The Investment Readiness Series, geared towards biotechnology projects, tell us about their proposal and their current challenges.


The great hope to fight fatty liver

"Hepatic fibrosis is a silent epidemic that is getting on top of us," warns researcher Rafael de la Torre. "An estimated one in four will be affected, becoming the main cause of liver mortality over the coming years, but there is still no drug to treat it." At the Hospital del Mar Research Institute (IMIM), the research group in Integrative pharmacology and systems neuroscience led by Rafael de la Torre is studying non-alcoholic steatohepatitis (NASH), a type of steatohepatitis in which the accumulation of fat in the liver is accompanied by inflammatory changes with fibrosis, which may lead to cirrhosis of the liver or hepatocellular carcinoma. The team has developed two conjugated fatty acids that have proven to be potentially effective in reducing the fat deposits in the liver (commonly known as fatty liver) and could also prevent hepatic fibrosis.

The project is receiving funding from the Carlos III Health Institute through summer 2019. "With these resources, we hope to finish the preclinical phase and get everything ready to start phase I, alone or in collaboration with a company. We've already seen some interest," he explains.


Attacking cancer when it sleeps

Under the framework of The Investment Readiness Series, Héctor G. Palmer, head of the Stem Cells and Cancer group at Vall d'Hebron Institute of Oncology (VHIO), made his first official pitch to potential investors on a new therapeutic target based on modulating latency in tumor cells. His team is developing cancer drugs that target TET2. In the preclinical phases, inhibiting this protein has been effective in eliminating dormant tumor cells, which could be related to resistance to treatment. Now they are focusing on specific hematological cancers that are highly dependent on TET2 function.

"We are in the chemical development phase of the molecules and intend to patent them and set up a spin-off in 2019," explains Palmer. "We are looking for funding, particularly for the regulatory phase."


Inhibiting prostate cancer

The Structural Biology of Human Nuclear Receptors group at the Institute of Biomedicine of the University of Barcelona (IBUB) and the Molecular Basis for Disease group at Hospital de la Santa Creu i Sant Pau Barcelona are working to develop first-line drugs to inhibit androgen receptor dimerization in patients with prostate cancer. As it targets the formation of the 3-D structure of these receptors, it could prevent resistance, as is seen with current treatments.

The project received one of the grants through the fourth edition of the CaixaImpulse program. Eva Estébanez pitched the project to investors at The Investment Readiness Series and has already initiated contact with international investors. "In the short term, we are looking for €500,000 in funding to optimize the molecules in order to apply for a patent. In the middle term, we need €2.5 million for the in vitro and in vivo studies. In 2019, we expect to develop our business model and set up a spin-off with the goal of locking down series A funding."


High-precision alternative to CRISPR

The Translational Synthetic Biology group at Pompeu Fabra University (UPF) has developed Uni-large, a new generation of drugs that combines the precision of CRISPR/Cas9 and the efficacy of clinically established viral vectors. They are developing their first therapy to treat congenital muscular dystrophy type 1A (MDC1A), a condition that affects one in 30,000 people. "It is a highly disruptive technology that allows for precision drug delivery, inserting natural or synthetic genes into the genome precisely," highlights project leader Marc Güell. "This opens up new doors, for example, in muscular atrophy but also in oncological applications that need big genetic changes, where CRISPR has proven difficult in reaching clinical practice."

The project received one of the grants through the fourth edition of the CaixaImpulse program. "With this grant, we expect to complete the bulk of the preclinical phase, but we will need funding to tackle the regulatory phase and reach the clinical phase," explains Güell. "Within the year, we can have intellectual protection tied down, more consolidated preclinical and in vitro data, and a more developed business model. The goal then will be to get series A funding."


New approach to ALS: Neuroresolving Therapeutics

At the Autonomous University of Barcelona Neurosciences Institute, the Group of Neuroplasticity and Regeneration is researching the contribution of a new family of lipids with a disruptive mechanism of action that emphasizes resolution of inflammation and not an anti-inflammatory mechanism with significant side effects and limited benefits. Dr. Rubèn López and his team have developed this new treatment for amyotrophic lateral sclerosis (ALS), multiple sclerosis and spinal injuries to find more effective therapies for these degenerative neuromuscular diseases. Their results so far show that these compounds help slow the onset of the disease and protect the functioning of the nervous system. Their goal, in addition to stopping the progression of ALS, is to find new biomarkers to predict the evolution of the disease. Riluzole is currently the only drug approved for the European market to treat ALS and, according to KOL, is limited in its therapeutic benefits and ability to extend the life of ALS patients.

 "We have funding for three years of non-regulatory preclinical study for ALS. We are looking for investors to help us, after, move into the regulatory phase," explains head researcher for the project Rubèn López. The team, which pitched the therapy to investors at The Investment Readiness Series, has already initiated contact with international investors.


New hope for an ultra-minority disease

Mitopath, with spin-offs already set up in Spain and Ireland, is based on research conducted at the Vall d’Hebron Research Institute (VHIR) on mitochondrial neuro- gastrointestinal encephalopathy (MNGIE), a very rare disease that, above all, affects the digestive and nervous systems and is seen in only one in 9 million people. Between 20 and 40 new cases are estimated to be diagnosed each year in Europe.

Those who suffer from MNGIE have a mutation in the thymidine phosphorylase gene (TYMP), which breaks down some substances (thymidine and deoxyuridine nucleosides) that, in excess, are toxic to the cell. The researchers have shown that gene therapy using adeno-associated viruses can maintain safe levels of these nucleosides without undesirable side effects. "In the short-term, we are looking for €1.5 million to tackle production of the vector and toxicology testing. The commitment of a local investor would help us attract attention from international investors," highlights Alan Belicha, CEO of Mitopath.


New strategy for broad-spectrum antivirals

The Molecular Virology Research Group at Pompeu Fabra University (UPF) is developing therapeutic tools using RNA that can be used as broad-spectrum antivirals to treat emerging viruses, such as Dengue, Zika and Chikungunya, among others. Plus, this strategy prevents resistance to drugs. "It is a highly novel strategy," says group leader Juana Díez, who pitched the project to investors at The Investment Readiness Series. “We are looking for funding to finish in vitro, in vivo and delivery studies and expect to create a spin-off in 2019."


New target for lung cancer

The AON project led by Jordi Hernández, entrepreneur in residence at the Center for Genomic Regulation (CRG), is based on correcting a genetic alteration in lung cancer that is key to cell proliferation. The new drug they are developing corrects defects in alternative splicing, a process that is necessary for proper protein formation. The treatment has been tested in mice and shown to reduce the rate of tumor growth.

"We've already completed in vivo proof of concept and are looking for €500,000 to optimize the compound and see how we can improve it before moving into the preclinical phase. The problem is finding funding in such an early stage," Hernández admits.


Breaking the tumor shield against chemotherapy

Tumors defend themselves with DNA repair mechanisms, and that explains why some do not respond to chemotherapy. Furthermore, the survival of tumor cells is often dependent on these repair pathways, making them an excellent therapeutic target to fight cancer. "We are looking for inhibitors of this repair mechanism so that tumors will once again be sensitive to chemotherapy. After many years of research, we've identified some molecules we think could be useful in cancer treatment," explains UAB professor Jordi Surrallés. This expert leads a group at the UAB, Hospital de la Santa Creu i Sant Pau Barcelona and the Biomedical Research Networking Center on Rare Diseases (CIBERER) that is studying tumor-prone syndromes like hereditary breast cancer and rare diseases, in which the cells have DNA repair defects that make them highly sensitive to chemotherapy. Studying these repair pathways has allowed them to identify inhibitors that could work as a new cancer drug.

Now they are working on the medical chemistry to make the molecule effective at low doses. "So far, we have worked with public funding and competitive grants. But we are moving into a drug development phase that requires involvement from the business sector," he notes. Within the year, they expect to begin the preclinical phase in mice, alone or in collaboration with one of the pharmaceutical corporations that have already begun to show interest in the project.


With the support of*:

* With the support of the Department of Enterprise and Knowledge and the co-financing of the ESF