Patents

First extracellular vesicles (EVs) as therapeutic vehicles targeting the brain containing engineered miRNA-based silencing sequences to treat Machado-Joseph Disease (MJD) 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

A light-triggerable polymeric nanoparticle (NP) library composed of 160 formulations with physicochemical 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 transfection. 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.

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. Method 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.

A panel of skin allergens and their derivatives exhibit extreme/strong capacity in neuroprotection by targeting the Nrf2 signalling pathway for treating and preventing neurodegenerative diseases, namely Alzheimer's Disease.

Extracellular vesicle-based oral vaccine for the prevention of giardiasis, namely Diarrheal Disease, an infection caused by the protozoa Giardia lamblia (also known as Giardia intestinalis or Giardia duodenalis). Problem to tackle: - ~1.7 billion cases of diarrheal disease are reported globally each year - Diarrheal disease is the 2º leading cause of death worldwide - Loss of worker productivity and direct impact on healthcare systems

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

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

RNA silencing techniques (e.g. RNAi) targeting single nucleotide polymorphisms (SNPs) in the ataxin-3 mRNA in linkage disequilibrium with the Machado Joseph disease-causing expansion. Delivery via minimally invasive routes, including intra-cerebrospinal and intravenous administration, using adeno-associated viral vectors (AAVs).