A Major Player in a New
Field of Medicine
With a strong expertise in neuromuscular diseasefield, SQY Therapeutics is a clinical-stage biotechnology company and technological platform designed to create, develop, and produce antisense oligonucleotide molecules to correct certain genetic mutations through the “exon skipping” approach.
The laboratory’s flagship activity involves the development of a new generation of antisense oligonucleotides (ASOs)
based on the tricyclo-DNA (tc-DNA) class. Tc-DNA are third-generation non-natural nucleotide analogs with higher affinity to their targeted RNA than existing options.
SQY Therapeutics’ technological platform enables the custom synthesis of tc-DNA ASOs and conducts preclinical evaluations dedicated to this type of molecule.
Our candidate ASO-tcDNA products are designed for exon-skipping therapies
and correct genetic defects of mutated genes at the mRNA level, allowing production of the missing protein.
The ASO-tcDNAs are compatible with intravenous administration and reach with high efficacy the entire skeletal and respiratory musculatures as well as the heart, primary targets in Duchenne Muscular Dystrophy.
Several ASO-tcDNA candidates are currently under development for Duchenne Muscular Dystrophy, a rare and fatal genetic disease.
Innovators in ASO Therapies
For over 20 years, the founders of SQY Therapeutics have been driven by a continuous commitment to develop and deliver a life-changing treatment for patients affected by a progressive, disabling and fatal genetic disease, Duchenne Muscular Dystrophy.
SQY Therapeutics was created to develop, investigate and transition new molecules to patients.
A decade of preclinical development led to the definition of a new generation of antisense oligonucleotides (ASOs) of the tricyclo-DNA (tc-DNA) class. This unique ASO chemistry exhibits unprecedented pharmacokinetic and pharmacodynamic properties and effectively interferes with dystrophin splicing throughout skeletal, respiratory, smooth, and cardiac musculature after systemic administration.
While tc-DNA persistance in muscles allows treatment to be spaced out over time, these AONs are gratudally cleared, ensuring the potential reversibility of the treatment.
The first candidate molecule, SQY51, entered in clinical phase.
SQY Therapeutics has designed several candidate molecules for Duchenne Muscular Mystrophy and other genetic disorders.
Our Missions and Values
SQY Therapeutics is a unique scientific and human adventure born from
the collaboration of researchers and parents of children affected by Duchenne Muscular Dystrophy, a rare and fatal disease.
Surrounded by a passionate team dedicated to the cause of patients, the company is the culmination of a long-standing fight to identify and promote the development of an effective treatment.
SQY Therapeutics’ mission is to achieve therapeutic treatments through the potential of its technology.
Inclusion
At SQY Therapeutics, we place patients at the heart of the research and drug development process.
Innovation
At SQY Therapeutics, we strongly encourage creativity and to explore new avenues.
Multi-Stakeholder Partnership
At SQY Therapeutics, we aim to adress the social and economic burdens of access to medicine for rare genetic diseases.
L'équipe de direction
Luis Garcia, PhD
Co-fondateur et Président
Christine Saulnier
Co-fondatrice et Directrice Déléguée
Laurent Mairot
Secrétaire Général
Micheline Joly Enyegué
Directrice Etudes Cliniques
Simon Guiraud
PhD, Directeur Biologie – Préclinique
Christophe Blaszykowski
PhD, Directeur Chimie Organique et chaine d’approvisionnement
Cyriaque Beley
Directeur Synthèse Oligonucléotides
