Proyectos de valorización

2022 Valorization Program

ACRÓNIMO	TÍTULO DEL PROYECTO	IP	CoIP	DESCRIPCIÓN
PHISaureus	Staphylococcus aureus phenotyping for lower respiratory tract analysis	Juan Pablo Salvador	Fernando Albericio, Miriam Royo	Infectious diseases are between the leading causes of mortality worldwide. Among other, Staphylococus aureus is one of the most prevalent bacteria in health-care system. Diagnostic is often limited by the need to previously growth and enrich the bacteria in specific culture media. With this scenario, the necessity for clear interventions to improve diagnostic tool for infectious diseases is evident. This project aims to validated an assay kit developed for the detection of four different strands of S. aureus. The clinical validation using lower respiratory tract samples will allow the clinicians the better understanding of the pathology of S. aureus.
MYODMDCARE	Drug repositioning for the treatment of Duchenne muscular dystrophy	Patricia Rico	NA	This proposal aims to investigate a drug repositioning for the treatment of Duchenne muscular dystrophy (DMD). We describe a new use, as an FDA approved drug, outside the scope of the original medical indication. Our investigations have identified that it is a promising strategy for the treatment of rare diseases, in particular DMD, for which repurposing is key, and often the only route for drug development. Our proposal is based in our previous results. Once optimised, we will advance to pre-clinical in vivo models of DMD following regulatory advice from the Agencia Española de Medicamentos y Productos Sanitarios (AEMPS).

2023 Valorization Program

ACRÓNIMO	TÍTULO DEL PROYECTO	IP	CoIP	DESCRIPCIÓN
FAST-MYOREPAIR	A new approach to accelerate muscle repair after an injury	Patricia Rico	NA	This proposal aims to preclinically validate the efficacy of treatments for various pathophysiological conditions that affect skeletal muscle, preparing the regulatory and organizational requirements for a possible repositioning of the drug and/or describe a new drug. In particular, we plan to collect enough preclinical data to be able to validate the use of these compounds as a new approach for muscle repair in indications of muscular injuries of diverse origin such as sports injuries, aging or traumatisms. In our ongoing work. To advance in the regulatory pathway of the possible use of these compounds for the treatment of muscular injuries, we plan to carry out an in vivo preclinical mouse model to evaluate their efficacy in muscle regeneration after an injury in healthy muscle. Our work plan also includes activities to analyse the technical and regulatory feasibility of the proposed clinical application, in order to maximize the opportunities for transferring the developed technology. FAST-MYOREPAIR proposes a new strategy for acceleration of muscle repair after an injury. Trauma and muscular injuries have a high prevalence and generate enormous and increasing medical costs. Particularly, we have confirmed the interest for new strategies in the sports medicine market. We believe our technology represents a very attractive option for the induction and acceleration of muscle repair after an injury, due to its low costs and safety.
BBBon-a-chip	In vitro drug testing platform using a BBB model on-a-chip culture and integrated monitoring for brain disease applications	Mònica Mir Anna Lagunas	Santiago Grijalvo	Recent breakthroughs in the field of micro- and nanotechnology allowed the development of organs-on-a-chip (OoCs) devices that better mimic the microphysiological environment of organs in vitro. OoCs have become an essential research tool in medicine and engineering, especially in the field of pathophysiological modelling and drug screening. For the specific field of application of brain models, one of the most interesting brain subsystems to be modelled is the blood-brain barrier (BBB). The BBB is a complex structure that acts as a gatekeeper separating the central nervous system from the systemic circulation. The low permeability of the BBB represents a major challenge to deliver drugs systemically to the brain. Here we will take advantage of the experience accumulated in a previous project to build up a highly versatile and integrated BBB on a chip (BBBoC) that physiologically reproduces a brain environment for simultaneous monitoring and stimulation of it. The proposed platform is meant to be easy to use, portable, likely to undergo mass production and automatization. It will be validated with nanocarriers loaded with agents and studies involving the BBB. The platform will be developed following a philosophy that favours optimizing its scalability, performance, and cost while maximizing its ease of use and the targeted end-users.
Radical-NEED	New Metal-free MRI Contrast Agents for Glioblastoma Diagnosis	Vega Lloveras Monserrat	Ana Paula Candiota	The use of paramagnetic Gd-based contrast agents (CAs) in Magnetic Resonance Imaging (MRI) (99% use in hospitals worldwide) is strictly regulated by many drug regulatory agencies like EMA, MHRA and FDA and some of them have been removed from market. They cannot be administered to patients with renal impairment due to the development of potentially lethal nephrogenic systemic fibrosis and it has recently been demonstrated the accumulation of toxic gadolinium deposits in the brain, bones, skin, liver and kidneys of healthy patients. This situation makes it critical to find alternative imaging probes. MRI contrast agents are paramount for the early diagnosis of tumours, as well as following-up response to therapy or relapse in several organs, but it is especially useful in brain tumors. We have focused on the use of metal-free paramagnetic species. We have been able to successfully synthesize up to 18 different CAs fully soluble in water. We have demonstrated in vitro that they can provide even higher contrast enhancement than Gd-based CAs and are not cytotoxic. Moreover, in 1 of them we have performed preliminary in vivo studies in healthy and glioblastoma (GB)-bearing mice, by intravenous (IV) administration, showing very promising results. The proof of concept in vivo suggested that our metal-free CAs are safe, produce consistent and sustained MR-contrast and are excreted in renal pathways, making them a viable alternative to metal-based MRI contrast agents, particularly on MRI analysis of glioblastomas. In summary, this project aims to carry out complete in vivo studies of some of the best candidates from the 18 CAs already tested in vitro.
DRUG4SIGHT	ADME studies and preliminary safety pharmacology of two lead compounds for functional restoration of sight	Pau Gorostiza	Eduardo Fernández	Blindness greatly affects human life and personal destinies. In adults, visual impairment causes loss of personal independence and the ability to work in many cases, often leading to the need for disability pensions, social services, and nursing. Around 3 million Europeans suffer from low vision and almost 700.000 have been legally recognized as blind. There are several inherited and acquired forms of blindness, including retinal degenerative diseases like the rare disease Retinitis Pigmentosa (RP). Options for patients with these retinal blinding disorders are very limited. However, the inner retinal neural circuitry persists until late stages of the degenerative process, which has led to the idea of targeting the remaining retinal wiring using novel therapeutic approaches. In this project, we have been able to identify two lead compounds achieving nanomolar affinity against mGlu6 receptor. These compounds have demonstrated to be active and potent in vivo, restoring the visual acuity of blind zebrafish larvae and restoring functional sight in a mouse model of retinal degeneration. The compounds have been administered topically to the eye without formulation, which emphasizes their potency and permeability By the end of this project, we expect to obtain the toxicological evaluation, the dosage, safety and biodistribution in vivo of the compounds, which are necessary steps to proceed towards clinical trials. through the cornea.