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.

EQUPE SQY THERAPEUTICS BANDEAU 2023

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

Our specificity

 

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.

It is the result of many years of struggle by parents to identify and promote the development of an effective treatment to save their children.

Founded in 2015, SQY Therapeutics is a technological platform intended to design, develop and produce antisense oligonucleotide molecules for the correction of certain mutations using “exon skipping”. Several molecules are under development, for other pathologies as well, through collaborations and/or partnerships.

The company is managed by parents of patients affected by Duchenne Muscular Dystrophy.

 

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SQY Therapeutics team

Today the company employs 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 the development of therapeutic approaches based on targeted modulation of mRNA splicing through antisense oligonucleotides in the tricyclo-DNA family.
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 phase 1/2A "Avance1" clinical trial has begun

During Phase 1 of the trial, each of the twelve participants (boys with Duchenne Muscular Dystrophy eligible for exon 51 skipping of the DMD gene) will receive an intravenous dose escalation of SQY51 (an antisense oligonucleotide from the tricyclo-DNA family) in six steps, with each administration spaced at least two weeks apart.

The first participant received his first dose of SQY51 on June 14, 2023 at the Raymond Poincaré Hospital in Garches (near Paris, France).

The other eleven participants will be integrated sequentially, at least one week apart. Phase 1 is scheduled for completion in the second quarter of 2024.

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The Avance1 clinical trial

The Avance1 clinical trial application filed by SQY Therapeutics received an approval from the ANSM (National Agency for Medicinal Safety and Health Products), number EU CT 2022-500703-49-01. The study is referenced in the ClinicalTrial.gouv database. Click here to find it.

Title of the study :

  • Phase 1/2-a Clinical trial : DMD children and adults with a genetically confirmed diagnosis of Duchenne Muscular Dystrophy, eligible for exon 51 skipping
  • Objective : open label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of SQY51
  • Phase 1 duration : 13 weeks multiple dose escalation
  • Phase 2a duration : 32 weeks
  • Administration : intravenous injections
  • Monocentric : Hôpital Raymond Poincaré, Garches (92380)
  • Launch : 2023
  • Trial coordinator : Lucia Echevarria-Zamora
  • 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).
SQY therapeutics projet avance 1
SQY THERAPEUTICS AVANCE 1 2023 v1

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 clinical trial

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

"SQY51 is a very promising molecule for the treatment of boys with Duchenne Muscular Dystrophy who are eligible for exon 51 skipping. I have been involved in the development of this new drug for 15 years, which is the result of a close collaboration between parents of boys suffering from DMD, researchers and doctors. The integration of the SQY Therapeutics laboratory within the UFR Simone Veil - Santé, and the strong link with the Hospital Raymond Poincaré in Garches, have created a privileged environment in which we were able to build our first clinical program, the AVANCE 1 trial, which will begin soon. I am very proud to be part of this collaborative effort."
Professeur Helge Amthor
Centre de référence des maladies neuromusculaires - Hôpital Raymond-Poincaré Garches – Coordinating Investigator of the Avance 1 trial
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As “compagnon de route” of Duchenne-Parent Project France and the Association Monégasque contre les Myopathies, I helped create SQY Therapeutics, a company born out of the meeting of researchers and parents motivated by the need to be fully engaged in the search for therapeutic solutions for their children. It has been a great privilege for me to witness their unwavering commitment to the cause of patients and I am at their side more than ever as they prepare to evaluate a first drug candidate for Duchenne Muscular Dystrophy, the compound SQY51."
Luis Garcia
CNRS Research Director - Director of the U1179 Inserm-UVSQ research unit - Co-Director of LIA-BAHN UVSQ-Centre Scientifique de Monaco – Scientific Counsellor of SQY Therapeutics
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SQY Therapeutics

A human and scientific adventure

Contact Team