Patents

A light-triggerable polymeric nanoparticle (NP) library composed of 160 formulations with physico-chemical diversity and differential responsiveness to light for delivery of RNA-based therapies. Physico-chemical diversity: allows the selection of the best NPs considering the cell type being transfected, diminishing the time required for the transfection to occur. Some NPs presented cell transfection of 10 min or less. Differential responsiveness to light: allows the selection of the more efficient NPs in endolysosomal escape after light-triggering the release.

The first extracellular vesicles (EVs) as therapeutic vehicles targeting the brain containing engineered miRNA-based silencing sequences to treat Machado-Joseph Disease/Spinocerebellar Ataxia Type 3 (MJD/SCA3) upon daily intranasal administration.   Advantages: Packaging cell line to continuously produce Engineered EVs 5x more delivery due to a specific tropism for neurons 2.5-fold enrichment of silencing sequences into EVs due to the presence of ExoMotifs Non-invasive administration: Intranasal route An alternative and efficient delivery method for miRNAs/Silencing sequences to treat MJD and other brain diseases

Optimized oligonucleotide sequences for PCR-based diagnostics of the genetic aetiology of Machado-Joseph Disorder (MJD), the CAG trinucleotide expansion within ATXN3 gene: a) length of the expansion and b) single-nucleotide polymorphisms (SNPs) associated with the mutant allele. This method will allow to diagnose and predict the progression of MJD and to design specific gene therapy strategies. Optimized oligonucleotides and probes to measure the expression of mutant mRNA in MJD to complement the genetic diagnosis and to evaluate the potential of gene-based therapies such as gene editing or silencing.

Device to isolate adherent cells from tissue explants, using a non-enzymatic method Improvements: - design allows the distribution of tissue fragments within pre-determined positions and immobilises them between the device and the cell growth area of a tissue culture container - tissue fragments are immobilized throughout the time in culture, favouring cell migration from the explants to the tissue culture container Application of the device for improving the isolation of cells from tissue explants

This technology comprises methods for reprogramming stem cells or differentiated cells into hemogenic and/or hematopoietic stem cell-like cells by expressing transcription factors in a polycistronic vector. Problem to tackle: - Treatment or diagnosis of blood disorders, cancer (especially haematological tumours) or infectious diseases

A panel of skin allergens or their derivatives, which exhibit extreme or strong capacity in neuroprotection by targeting the Nrf2 signalling pathway, for the treatment and/or prevention of neurodegenerative diseases, namely Alzheimer ́s Disease (AD).

This technology utilizes RNA silencing techniques, such as RNA interference, to target single nucleotide polymorphisms (SNPs) in the ataxin-3 mRNA in linkage disequilibrium with the disease-causing expansion. One of the ways we validated our technology was through its delivery via minimally invasive routes, including intra-cerebrospinal and intravenous administration, using adeno-associated viral vectors (AAVs). Problem to tackle: MJD Disease, or SCA3, is a rare and incurable disease and the most common form of Spinocerebellar Ataxias, affecting up to 2 in 100 thousand adults. MJD is a dominantly inherited autosomal neurodegenerative disorder caused by a genetic mutation in the coding region of the ataxin-3 gene (MJD1/ATXN3 gene), which encodes ataxin-3 protein. This genetic mutation leads to increased repetitions in the CAG segment of the ataxin-3 gene, with the severity of symptoms dependent on the number of extra repetitions. MJD symptoms include slowly progressive clumsiness in the arms and legs, a staggering or lurching gait, difficulty with speech and swallowing, impaired eye movements (sometimes accompanied by double vision or bulging eyes), and lower limb spasticity.