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Programs

Pipeline

We are developing transformational therapies in the field of oncology,  and other genetically driven disorders.

Our focus in cancer is based on a unique two pronged approach -  TUMOR SUPPRESSOR RESCUETM (TSG-RESCUETM, TSG-RESQTM) and ONCOGENE EDITINGTM (ONCOEDITTM).   These approaches have  been validated by numerous studies in therapeutic cancer models including  lung cancer,  colorectal cancer  pancreatic cancer and osteosarcoma.  

Our focus in other diseases has been validated by studies in therapeutic models including reversal of hemophilia and hypercholesterolemia using our technology.

Therapeutic programs


Our technology opens new perspectives for gene modulation, gene therapy and gene expression control strategies.

We are currently developing several therapeutic programs based on targeted delivery of various nucleic acids.

Therapeutic mRNA with Targeted Peptides

mRNA constitutes a promising new class of therapeutic molecules with the potential to treat a wide variety of diseases, which cannot be addressed with other technologies. mRNAs have highly similar compositions, generated with four different building blocks, but with an unique sequence order to encode a variety of different proteins.

In the last decade, therapeutic mRNA has become an efficient alternative to DNA. mRNA are easier to use and allow the fast development of therapies that are widely applicable for the treatment of many diseases (cancer, infectious diseases, rare diseases). Delivery of specific mRNA into cells directed the production of protein with biological effects, which is impossible with other drug approaches.

CRISPR/CAS9 Gene Editing with Targeted Peptides

Clustered, Regularly Interspaced, Short Palindromic Repeats “CRISPR” is an adaptive immune defense mechanism present in bacteria to degrade foreign genetic material, which is integrated into the CRISPR locus. The CRISPR/Cas9 system uses a combination of 2 types of molecules : a nuclease (the gene editor) and a guide RNA (which helps the nuclease find the right place to edit). CRISPR/Cas9 edits genes by precisely cutting DNA, and then letting natural DNA repair processes to take over. DNA damage is repaired by cellular DNA repair mechanisms, via either the non-homologous end joining DNA repair pathway (NHEJ), which leads to insertions or deletions creating errors or the homology-directed repair (HDR) pathway, which can be used to recombine selected markers at specific sites in the genome.

 

CRISPR/CAS9 gene editing technology has the potential to revise, delete, and replace almost any gene in human cells in highly targeted manner. Hence, advances in this technology will help us to develop awfully specific drugs for people with a wide variety of diseases.

 

Nucleic Acid-based Gene Silencing

Antisense and RNA interference therapies acted upstream by blocking the production of disease causing protein. Over or under production of a protein, or production of a mutated protein, are common causes of many human diseases. To generate a protein, a cell must make a copy of genetic code from DNA to messenger RNA (mRNA). mRNA carries the specific instructions on how to make this specific protein from cell’s nucleus to ribosomes, the cell’s protein-making machinery.

Antisense and interference strategies are designed to search for, bind to and destroy mRNA in a highly specific manner so that the amount of pathogenic proteins is considerably reduced. They are based on the use of short synthetic nucleic acid sequences, DNA or RNA (single- or double-stranded), which bind precisely with target mRNA and induce its degradation. Two different approaches are commonly used : the degradation of the target mRNA by an endogenous RNAse triggered by an antisense oligonucleotide (ASO) or the activation of the natural pathway involved in the regulation of gene expression in mammalian cell by small interfering RNA molecules (siRNA).