Duchenne Muscular Dystrophy

Treatment by Exon skipping

Antisense oligonucleotides

Tricyclo-DNA

 

Our mission

To implement therapeutically aimed R&D programs for Duchenne Muscular Dystrophy and other genetic diseases for which no satisfying treatment yet exists

 

 

 

Our values

Research

SQY Therapeutics conducts innovative R&D projects with a therapeutic aim

Engagement

A passionate team of researchers devoted to the patient cause

Integrity

The social purpose of our company takes precedence over its commercial valuation

The Patients

To broaden the field of application of our innovations to other pathologies

The story of our battle against Duchenne Muscular dystrophy

 

SQY Therapeutics is a singular scientific and human enterprise born from the meeting of researchers and parents of children affected by an incurable disease: Duchenne Muscular Dystrophy.

 

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At first there was the disease that burst into our lives, Duchenne Muscular Dystrophy, and the absence of treatment for our boys, Paul, Julien, Adrien, Alexander and many others… Then came the time to fight against the unacceptable. First by creating associations to aid in this struggle, then by pooling our resources to promote the implementation of effective treatments. The solidary alliance of two parents, Luc Pettavino, president of the Assoctiation Monégasque contre les Myopathies, and Christine Saulnier, president of Duchenne Parent Project France, has allowed the assembly of an international collaborative network of Duchenne Muscular Dystrophy research and the creation of two biotechnology companies; Synthena in 2012 and SQY Therapeutics in 2015, thanks to Luc Pettavino’s exceptional fundraising for the last 20 years.
SQY Therapeutics is a technological platform intended to design, develop and produce antisense oligonucleotide molecules for the correction of certain mutations using “exon skipping”. These are personalized treatments (all DMD patients are not eligible for the same drug) and several molecules are under development, for other pathologies as well, through collaborations and/or partnerships.
The majority of the capital of SQY Therapeutics is held by the Association Monégasque contre les Myopathies which raises most of the operating funds coming from charity resources. All profits made by SQY Therapeutics (sale of manufactured products for other neuromuscular diseases, licensing, etc.) are reinjected into the company’s activity in service of its cause and to remain economically viable. The company is managed by parents of patients affected by Duchenne Muscular Dystrophy.

About the Team

Equipe SQY THERAPETUCIS

 

A few questions about the SQY Therapeutics team

Today the company employs a dozen highly qualified collaborators, doctors of biology and chemistry, research engineers, as well as many expert consultants in the domains of organic chemistry of nucleic acids, molecular genetics, preclinical evaluation of candidate compounds and their clinical development.
Highly competent in neuromuscular disease research, the team is mainly dedicated to two research areas: (i) the development of therapeutic approaches based on targeted modulation of mRNA splicing through antisense oligonucleotides in the tricyclo-DNA family, and (ii) the conception of new gene transfer systems.
SQY Therapeutics is the only tricyclo-DNA class oligonucleotide manufacturing synthesis platform that also integrates a preclinical evaluation activity dedicated to this type of molecule.
The SQY Therapeutics headquarters is based in Noisy le Roi, in the Yvelines, and its R&D activity is located in the UFR santé Simone Veil at the Université de Versailles in Montigny-le-Bretonneux where it works alongside the U1179 Inserm-UVSQ mixed research unit.

The Avance 1 clinical trial project

  • Phase 1/2-a Clinical trial: DMD children and adults, eligible for exon 51 skipping
  • Objective: Security, SQY51 Pharmacokinetics and Pharmacodynamics
  • Phase 1 duration: 16 weeks
  • Phase 2a duration: 33 weeks
  • Administration: intravenous injections
  • Monocentric: Hôpital Raymond Poincaré, Garches (92380)

  • Launch : 2022
  • Trial coordinator : Adeline Vulin, SQY Therapeutics
  • Trial promotor : SQY Therapeutics.
  • Entity responsible of the trial: Garches Clinical Investigations Center.
  • Main investigator : Professor Helge AMTHOR (Service Pédiatrie/Centre de référence des maladies neuromusculaires, Hospital Raymond Poincaré of Garches, University of Versailles Saint-Quentin-en-Yvelines).
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sqy therapeutics projet avance 1
sqy projet avance 1
SQY therapeutics projet avance 1

The SQY51 candidate compound

Developed by SQY Therapeutics, it is an antisense oligonucleotide (15-mer) of the tricyclo-DNA class.

SQY51 is designed to hybridize with exon 51 of the dystrophin pre-messenger RNA to restore an operational reading frame by "exon skipping" in Duchenne Muscular Dystrophy patients with an appropriate deletion pattern (i.e., ≈10% of the DMD population).

Preclinical data of the Avance 1 project

Preclinical work in non-human primates and mice confirms the strong potential of this compound: (i) the preclinical safety profile is very favorable, (ii) the candidate compound is efficiently distributed throughout the musculature (skeletal muscles, in particular respiratory muscles, and the heart - priority targets in DMD), (iii) the levels of exon 51 skip in all tissues analyzed are such that they suggest the possibility of a real clinical benefit in DMD patients.

The designers of the Avance 1 trial

SQY Therapeutics

A human and scientific adventure