Technologies

The development of genome editing tools has revolutionized the way we think and deal with genetics. The use of Cas9 or its variants allows modifications of specific sites in the human genome by inducing deletions and insertions in a more or less controlled way. In recent years, a new class of tools for genome editing has emerged: the base editors (BE), which result from the fusion of a modified Cas9, which serves to direct the BE to the target, and an active deaminase acting on the DNA, which mediates the C> T or A> G editing.

Aptamers, short structured single-stranded oligonucleotides binding at high affinity to a given target protein, are selected from large combinatorial libraries through repeated cycles of incubation of the library with the target, recovery and amplification of target-bound oligonucleotides (SELEX technology, Systematic Evolution of Ligands by EXponential enrichment). SELEX can be applied to select aptamers against a known target protein or against a specific cell phenotype, without any prior knowledge of the specific target, leading to new biomarkers discovery.

The technology we participate to develop, called "Zinc-Finger Artificial Transcription Factors (ZF-ATFs)", allows to design, realize and select artificial genes coding for proteins capable of recognizing and binding "potentially" any DNA sequence. We used ZF-ATF technology to reprogram the expression of "beneficial" genes capable of efficiently counteracting the negative effect of mutated genes related to rare diseases.

The present invention relates to the biomedical sector of the treatment of lung diseases and related symptoms. In particular, the present invention provides compositions and methods based on the use of selected polymeric biomaterials, in combination with stem cells and/or their secretome, capable of synergistically improving the development, regeneration and repair of chronic lung injuries and related symptoms.

The herein described technology aims at the development of a platform of injectable hydrogels for application as drug carriers for localized delivery or in the regenerative medicine field. The use of ad-hoc synthesized poly(ether urethane)s (PEUs) as hydrogel forming materials is a common property which characterizes all the systems belonging to this platform.

An innovative approach for the treatment of diabetic and venous ulcers, characterized by a difficult healing process and therefore at potential risk of infection and therefore of hospitalization and amputation of the limb, is represented by the local administration of "bioactive" factors through the use of synthetic and/or biological matrices that allow a gradual and controlled release in order to obtain a better and faster healing.

Recently, nanoparticles and nanovesicles have been investigated as potential approaches for the treatment of neurodegenerative diseases. In particular, in the Biotech sector an increasingly deeper penetration of new treatment models and biological drugs based on cellular, subcellular and vesicle therapies is expected. The patent is based on the production of Myelin-based nanoVesicles (MyVes) produced by microfluidics, starting from myelin extracted from brain tissue. These vesicles find two major fields of applications as potential drugs or as supplements/nutraceuticals.

Therapeutic strategies targeting cell cycle in cancer have in general failed in the clinic since the drugs have lacked the therapeutic index required to achieve a robust response against cancer cells with little or no cytotoxic effect on normal cells. NEK6 kinase, which is implicated in cell cycle control, has recently emerged as an attractive target for the development of novel anticancer drugs with enhanced therapeutic index.

With the advent of senolytic agents, capable of selectively removing senescent cells in “aged” tissues, the perception of age-associated diseases has changed from being an inevitable to a preventable phenomenon of human life. The present invention is part of this research topic with the identification of molecules with potential pro-apoptotic activity, specifically with senolytic activity. The computational approach adopted, is based on combining ligand-base and structure-based virtual screening.

The development of an innovative screening platform of natural marine extracts guided by biological assays represents one of the main products developed within the Antitumor Drugs and Vaccines from the SEA (ADViSE) project which aims to provide a new vision in Drug Discovery processes.

Our treatment demonstrated the ability to kill metastatic human melanoma cells, for which there are very few effective therapeutic approaches. Use of a specific Essential Oil (EO) to inhibit the replication of human metastatic melanoma cells. This EO can be used both for direct application to the skin, and administered by mouth to reach both primary and metastatic melanomas.