OLIGONUCLEOTIDE BASED THERAPEUTICS

Treatment of genetic neuromuscular diseases
by using its tricyclo-DNA molecular technology

ABOUT US

Synthena’s mission is to develop first in class drugs in the field of Myopathy with a focus on the Duchenne Muscular Dystrophy disease, based on its proprietary tricyclo-DNA technology.

Its proprietary tricyclo-DNA technology offers broad advantages over state of the art oligonucleotide chemistries for new RNA intervention strategies like splice switching, and other antisense approaches. Its most advanced preclinical drug development program focuses on Duchenne Muscular Dystrophy, a genetic disorder which affects 1 in 3,500 boys and which leads to death in early adulthood.

Modulation of RNA for therapeutic purposes holds the promise of opening up entirely new concepts for pharmaceutical intervention, however, current oligonucleotide chemistries to modulate RNA do not meet the requirements for efficient delivery, pharmacokinetics and tolerability.

 

LEADERSHIP

MANAGEMENT TEAM - DIRECTORS & FOUNDERS

Pascal Ferré

Board Member

Pascal Ferré is the parent of a boy with Duchenne muscular dystrophy. He is a chartered accountant and ...

MORE

statutory auditor in France.

To find a cure for this disease, he helped found SYNTHENA AG with other families, DPP France, the Association Monégasque contre les Myopathies and researchers.

TcDNA, developed and patented by SYNTHENA AG, is currently used by other companies to design AONs targeting Duchenne disease and many other diseases of genetic origin.

LESS

Terrence Partridge

Chairman

Terrence Partridge, a renowned researcher in the field of myology, the science of muscle. He is a professor at...

MORE

the Children’s National Medical Center in Washington DC and a leader of the Center for Genetic Medicine Research.

LESS

 

Luis Garcia

PhD and Board Member

Luis Garcia received his PhD in 1989 at the University Paris 7 (Diderot) in the laboratory of Michel Fardeau (INSERM U153/UA614 CNRS) on the role of the L-type Ca2+ channel in excitation-contraction coupling in skeletal muscle. After a Post-doctoral experience...

MORE

(1989-1990) at the Rockefeller University in the Laboratory of Prof. Gerald M. Edelman, he was appointed by the CNRS (1991) as a permanent researcher. In 1995, Luis Garcia integrated the INSERM Unit «Neuroplasticity and Therapeutics», headed by Marc Peschanski (INSERM U421) to explore raising fields such as cell or gene therapy. In 1997, he joined Olivier Danos at the Genethon/UMR8115 CNRS laboratory to initiate its transformation into a Research Centre on gene therapy, and later on in 2007, Luis Garcia moved my team to the University Pierre & Marie Curie (INSERM U974/CNRS UMR7215) with the aim of developing therapeutic strategies based on mRNA repair. In 2012, he was invited to join the new Faculty of Medicine of the University of Versailles St-Quentin to create an Associated International Laboratory UVSQ-CSM in Monaco (LIA-BAHN - 2013) and a joint research laboratory UVSQ-INSERM in Versailles (U1179 - 2015), both committed to investigating novel therapeutic approaches for neuromuscular disabilities in relationship with the University hospitals of “Paris-Ile-de-France Ouest”.

LESS

 

Markus Ruegg

PhD and Board Member

Markus Rüegg is a Swiss neurobiologist and professor at the Biozentrum of the University of Basel. Markus Rüegg studied biochemistry at the University of Zurich and graduated with a PhD in the field of Neurobiology. In 1989...

MORE

he went as a postdoctoral fellow to conduct research at the Department of Neurobiology at Stanford University School of Medicine.[1] In 1992 he was appointed as Assistant Professor to the Biozentrum, University of Basel. Since 1998 he is a Professor of Neurobiology and teaches and conducts research at the Biozentrum of the University of Basel.[2] Based on his research findings he co-founded the first spin-off company (MyoContract Ltd) of the Biozentrum in 2000,[3] which merged in 2004 to become Santhera Pharmaceuticals Ltd.[4] As of August 2021, he co-founded SEAL Therapeutics Ltd. and acts as its CEO. SEAL Therapeutics Ltd. aims to develop a gene therapy for the severe LAMA2-related muscular dystrophy (LAMA2 MD).
Rüegg studies the molecular principles that are essential for the development and the maintenance of the neuromuscular system. The major achievements of his earlier work include the isolation and functional characterization of proteins involved in axonal pathfinding,[7] synapse formation[8][9][10][11] and in mediating changes in synapse structure upon learning.[12] Furthermore, for the last 20 years his laboratory is interested in understanding the disease mechanisms involved in congenital muscular dystrophies and recent findings of his laboratory have led to the development of a novel therapeutic strategy.[13][4] In addition, his research group has recently demonstrated that the multi-protein complex mTORC1 is essential for muscle homeostasis and is associated to precocious sarcopenia, the loss of muscle mass and function at advanced age.[14][15] This knowledge may help to counteract pathological muscle degradation and to develop new therapeutic strategies.

LESS

 

SCIENCE

Antisense Technology

Synthena AG is a development stage biotechnology company aiming to develop and commercialize new life saving drugs for the treatment of severe neuromuscular diseases based on the modulation of RNA. Its proprietary tricyclo-DNA technology platform offers broad advantages over state of the art oligonucleotide chemistries for new RNA intervention strategies like splice switching, and other antisense approaches. Its most advanced preclinical drug development program focuses on Duchenne Muscular Dystrophy, a genetic disorder which affects 1/3,500 boys and which leads to death in early adulthood.

 

PIPELINE

CONTACT

Synthena AG
Holderstrasse 5, 3011 BERN
Switzerland

Mail   info@synthena.com

Scroll to top