- Date: March 18, 2020
Read any scientific journal and you’ll quickly come across articles about gene editing, personalised medicine, and antibiotic resistance. The first two are increasingly transforming healthcare, allowing us to understand the complexities of, and thus treat, a variety of diseases once thought unsolvable. Meanwhile, the third is threatening to render many relied-on therapeutic options ineffective, and placing an unprecedented burden on healthcare systems around the world.
These are the key topics that are innovatively explored by the four projects selected in the inaugural edition of CaixaImpulse Consolidate. All of them offer solutions to unmet biomedical needs and will receive up to €300,000 and essential non-financial support to move their assets closer to the market and patients. Although not a prerequisite of the Consolidate call, the four projects had been selected in a previous edition of CaixaImpulse Validate, and are now ready to bring their projects to an investment-ready stage. Discover the first cohort:
Curing Friedreich’s ataxia with gene therapy
Friedreich's ataxia (FRDA) is an incurable neurodegenerative disease that normally starts during childhood, affecting approximately 1 in 50,000 people. The objective of the FRATAXAV project, led by Antoni Matilla from the Germans Trias i Pujol Research Institute (IGTP), is to develop a curative treatment for this devastating disease. Their novel drug will deliver frataxin, a protein that is reduced in FRDA, to patients’ cells via a recombinant adeno-associated virus vector. We spoke to Antoni Matilla last year on Rare Disease Day to discuss the progress of the project, and the importance of spreading awareness. Read the interview here.
The team behind the FRATAXAV project, led by Antoni Matilla (right)
Gene editing to combat type 1A muscular dystrophy
Current gene editing technologies have limitations. For example, it is challenging to administer gene-replacement therapy using viral vectors when the target protein is too large. Marc Güell and his team at Universitat Pompeu Fabra (UPF) hope to overcome this with their project Uni-Large. Their approach, which combines precision of CRISPR with the effectiveness of viral vectors, will be used to treat congenital type 1A muscular dystrophy (MDC1A), an incurable rare disease that begins at birth.
The Translational Synthetic Biology group at UPF, who are developing the gene editing platform Uni-Large
Enabling personalized medicine in cancer
Cancer is already notoriously difficult to treat, which is often further complicated by the different responses of patients to certain drugs. This is the case with PARP inhibitors, a family of drugs used to treat certain types of breast and ovarian cancer. Alba Llop, a researcher at the Vall d’Hebron Institute of Oncology (VHIO), leads the project RAD51predict. Their goal is to develop a diagnostic test to determine which patients will benefit from PARP inhibitors, thus delivering a more personalised treatment.
Alba Llop (third from left) is the leader of RAD51predict, a project from VHIO
Fighting antibiotic resistance with an immunity-boosting drug
Hospital-acquired infections are a huge problem in healthcare systems around the world. Gram-negative bacteria are responsible for up to 30% of these infections, and are rapidly becoming resistant to available antibiotics. The project RemAb Therapeutics, a spin-off from the Institut d’Investigació Biomèdica de Bellvitge (IDIBELL) led by Rafael Mañez, offers an alternative to antibiotics that does not generate resistance. Their drug, RA-01, removes non-neutralizing antibodies thus boosting the immune response of the patient to prevent infections from occurring in the first place. Read our 2019 interview with Rafael Mañez here.
RemAb Therapeutics is a spin-off of IDIBELL, led by Rafael Mañez
More programme highlights
February 28, 2020
On the occasion of World Rare Disease Day, we spoke with Rubén Artero and Beatriz Llamusí, who form part of the former CaixaImpulse 2017 project MBNL.
February 20, 2020
Over five years of the programme, 103 projects have been supported, 20 of which are now spin-offs.
January 28, 2020
While CaixaImpulse Validate supports the earliest stages in health innovative, there are many initiatives aimed at boosting the development of projects at every stage.