Synthena AG (SYN)
Synthena AG has been established in 2007 as a development stage biotechnology company aiming to develop and commercialize new life saving oligonucleotide therapeutics for the treatment of neuromuscular diseases based on mRNA targeting. Its proprietary tricyclo-DNA technology platform offers broad advantages over competing state-of-the-art oligonucleotide chemistries for new RNA-based therapeutic intervention strategies such as splice modulation and other antisense approaches. Its most advanced preclinical drug development program focuses on Duchenne muscular dystrophy, a genetic disorder which affects 1/5,000 boys and which leads to death in early adulthood.
Due to their properties tcDNA oligonucleotides are particularly well-suited for biological and therapeutic applications where high target affinity and bioavailability are required and where the mechanism of action does not rely on RNase H activity.
In collaboration with the laboratory “Biotherapies of Neuromuscular Disorders” based at the University of Versailles-Saint-Quentin (partner VER in the current proposal), Synthena has demonstrated the unique ability of tricyclo-DNA to cross the blood-brain barrier at low levels and their high therapeutic potential for the systemic treatment of DMD. Both partners have a long-standing collaboration and extensive experience with therapeutic applications of tcDNA AONs [1].
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1. Aurélie Goyenvalle et al. Nature Medicine, 21:270-275 (2015). doi:10.1038/nm.3765
Website
Team
Dr Luis Garcia
Position in Organisation
Board Member
Profile
Luis Garcia received his PhD in 1989 at the University Paris 7 (Diderot). After a postdoctoral at the Rockefeller University (1989-1990) he was appointed by CNRS as a permanent researcher (1991). In 1997, Luis Garcia joined Genethon to initiate the transformation into a research centre on gene therapy, and in 2007 moved to the University Pierre & Marie Curie to develop therapeutic strategies based on mRNA repair. In 2012, he joined the Faculty of Medicine at the University of Versailles St-Quentin to create an Associated International Laboratory UVSQ-CSM in Monaco (2013) and a joint research laboratory UVSQ-INSERM in Versailles (2015), both committed to novel therapeutic approaches for neuromuscular diseases in partnership with University hospitals of Paris-Ile-de-France Ouest.
Role in the project
Luis GARCIA is the lead contact for Synthena AG for co-ordination with project partners.
Dr Dmitry Stetsenko
Position in Organisation
Chief Scientific Officer
Profile
Dmitry Stetsenko received his PhD in Bioorganic Chemistry in 1997 from the Institute of Bioorganic Chemistry, Russian Academy of Sciences. After a postdoctoral in Michael Gait’s group at MRC Laboratory of Molecular Biology in Cambridge (1998-2004), he was working at University of Surrey (2005-2010) and Imperial College London (2010-2011). In 2012 he joined the Institute of Chemical Biology and Fundamental Medicine in Novosibirsk, Russia as a Lead Scientist in the Russo-American Laboratory of Biomedicinal Chemistry under Nobel Prize winner (1989) Prof Sidney Altman and later as the head of Laboratory of Nucleic Acid Chemistry (2014-2019). In 2019 he joined Synthena AG as CSO.
Role in the project
Dmitry STETSENKO will organize research work, oversee experimental design and supervise synthetic part of the project to ensure timely delivery of tcDNA AONs.
