The compounds, peptides and nucleotides, are useful for the treatment of diseases such as premature aging syndromes, dyskeratosis congenita, neurodegenerative diseases, cancer and other pathologies.

The protein dyskerin forms part of the telomerase complex. A fragment of the dyskerin protein, a genetic supressor element (GSE) termed GSE 24-2 act as a telomerase activity activator. The induction could also be obtained by the administration of the correspondent 55 aminoacids-long peptide. These compounds are useful for the treatment of diseases that alter telomerase activity or with cellular senescence, such as premature aging syndromes, Werner syndrome, dyskeratosis congenita, aplastic anemia and could be possibly applied to vascular aging, cancer, neurodegenerative diseases, Fanconi anemia, atherosclerosis and Bloom syndrome.

The peptide expression reactivates telomerase activity in cell lines derived from dyskeratosis congenita patients and in telomerase-deficient human cell lines. Moreover, the expression of GSE24-2 is able to rescue dyskeratosis congenita derived fibroblasts from premature senescence. These cells are able to grow continuously up to 140 days.

Key features

• This reagent is a peptide or cDNA that can be delivered to cells that harbor telomerase activity defects.
• It is a novel reagent since there is no alternative therapy for dyskeratosis congenita and other syndromes.
• Compounds could be useful for tissue regeneration treatments of epithelial or hematopoietic cells.
• Compounds could be useful for immortalization of eukaryotic cells for research use or biotechnology processes.
• The drug could be approved as an orphan drug.
• The size of the peptide is small and that would facilitate delivery to patients.

Early Stage

Priority patent application filed in Spain (ES 200502511 and ES 200703106)
PCT application filed (PCT/ES06/070152, published as WO 2006/054551)
Patents applications filed in Europe (No. 06820027.8), in USA (No.12/090.215), Canada and Japan.

A patent license agreement with a company able to manufacture and market the product is sought.