DYSTRO-SMARTY: A novel nanotechnology platform for delivery of nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy
Awardee: Cecilia Jimenez-Mallebrera
Institution: Hospital Sant Joan de Deu
Grant Amount: $48,876
Funding Period: February 1, 2022 - January 31, 2023
Summary:
Nucleic-Acid based therapies are being developed at a fast pace with 11 currently approved products and many more in the pipeline. However, delivering therapeutic amounts of these nucleic acids to the target tissue remains the major hurdle, particularly for muscle diseases. Here we propose to apply a validated nanotechnology platform, SMARTY, based on non-liposomal lipid-based nanovesicles, called Quatsomes, to deliver nucleic acids to treat COL6-related Congenital Muscular Dystrophy (COL6-CMD). These nucleic acids are antisense oligonucleotides (ASO) that we have designed and tested to correct a common mutation in collagen VI genes. ASO will be conjugated to the Quatsomes and their physico-chemical properties, distribution and integrity inside the cell as well as their specificity and efficacy to correct collagen VI mutations will be systematically investigated in cells from COL6-CMD patients. The Quatsomes platform (patent WO/2020/229469), developed by our collaborators (at VHIR and ICMAB-CSIC), has already been exploited for other applications for effective intracellular delivery of nucleic acids. Moreover, Quatsomes will be produced by a GMP compliant manufacturing process. This will facilitate the future translation and approval of this potential therapy by regulatory agencies bringing COL6-RD closer to Clinical Trial Readiness.