SQY Therapeutics

What SQY Therapeutics does

SQY Therapeutics is a clinical-stage French biotechnology company developing antisense oligonucleotide (ASO) therapies based on its proprietary tricyclo-DNA (tcDNA) chemistry platform, with a primary focus on Duchenne Muscular Dystrophy (DMD). Founded in 2015 by DMD parent associations and academic researchers, the company's lead asset SQY51 is a tcDNA ASO designed to skip exon 51 of the dystrophin pre-mRNA, restoring a partially functional dystrophin protein and converting severe DMD toward a milder Becker Muscular Dystrophy phenotype. SQY51 is currently in the Phase 2a portion of the AVANCE-1 First-in-Human trial at Hôpital Raymond-Poincaré AP-HP and has received FDA Orphan Drug Designation (May 2024) and EMA Orphan Drug Designation (July 2025). The technology platform incorporates a high-throughput screening cascade and proprietary 3D human DMD engineered muscle models for preclinical evaluation, and a January 2026 publication validated pulmonary administration as a non-invasive systemic delivery route achieving 34.4% bioavailability.

The company operates as a pre-revenue, privately held SASU headquartered in Guyancourt (Saint-Quentin-en-Yvelines), France, with 22 employees as of 2025 and a newly inaugurated 850 m² dedicated R&D laboratory housing the only existing platform for custom tcDNA-ASO synthesis and integrated preclinical evaluation. SQY Therapeutics functions within a co-located Hospital-University Ecosystem (EHU) comprising PHARMAColigo (UVSQ), UMR1179 Inserm-UVSQ (Luis Garcia's research unit), and Hôpital Raymond-Poincaré AP-HP. Future revenue is intended to come from sales of approved pharmaceutical products for rare genetic diseases, supported by 7-year US and 10-year EU orphan market exclusivity upon regulatory approval. The eligible patient population is the approximately 10-15% of DMD patients amenable to exon-51 skipping, within a disease that affects roughly 1 in 3,500 male births.

Disclosed funding is limited to a €300,000 grant from the Île-de-France Region (2025) for laboratory construction; no venture capital or private equity rounds are disclosed. The company was co-founded by Luis Garcia (President) and Christine Saulnier (Managing Director), supported by a specialized leadership team across oligonucleotide synthesis, organic chemistry, biology/preclinical research, and clinical studies.

Name

SQY Therapeutics

Legal name

SQY THERAPEUTICS, SASU

Website

https://sqy-therapeutics.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

11–50 employees

Short description

SQY Therapeutics is a pre-revenue French clinical-stage biotech developing tricyclo-DNA antisense oligonucleotide therapies, with lead asset SQY51 in Phase 2a for exon-51 amenable Duchenne Muscular Dystrophy patients, holding FDA and EMA Orphan Drug Designations.

Ownership category

akta.pro rank

Product category

Antisense Oligonucleotide Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industries

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), In Vivo Discovery & Translational Models Platforms (humanized models, PK/PD, efficacy) (HLAAAIAK)

Keywords

• Antisense oligonucleotide therapy
• Exon-skipping therapeutics
• Duchenne muscular dystrophy
• Tricyclo-DNA platform
• Rare disease biotechnology

Headquarters

HQ city

Montigny-le-bretonneux

HQ country

France

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Infrastructure, Supply Chain

Revenue model

1. Pharmaceutical Product Revenue: Pre-revenue clinical-stage biotech company. Upon regulatory approval of SQY51 and future drug candidates, revenue will be generated through sales of prescription pharmaceutical products for rare genetic diseases. Orphan drug designation provides 10 years market exclusivity in EU and 7 years in US.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

SQY Therapeutics is a clinical-stage biotechnology company developing antisense oligonucleotide (ASO) therapeutics built on its proprietary Tricyclo-DNA (tcDNA) chemistry platform, with lead drug candidate SQY51 designed to treat Duchenne Muscular Dystrophy by skipping exon 51 of the dystrophin gene. SQY51 is currently being evaluated in the AVANCE-1 Phase 1/2a clinical trial at R. Poincaré Hospital AP-HP for the approximately 10–15% of DMD patients eligible for exon-51 skipping therapy, with both FDA and EMA Orphan Drug Designations secured.

Product overview

SQY Therapeutics is a clinical-stage biotechnology company developing a portfolio of antisense oligonucleotide (ASO) therapeutics based on its proprietary tricyclo-DNA (tc-DNA) technology platform. The company's lead product is SQY51, a tc-DNA ASO designed for exon-skipping therapy in Duchenne Muscular Dystrophy patients amenable to exon-51 skipping. The technology platform enables custom synthesis of tc-DNA ASOs and includes preclinical evaluation capabilities using 3D human muscle models. SQY Therapeutics is also developing additional ASO-tcDNA candidates for other DMD subpopulations.

Differentiator

Problem solved

Functional benefit

Products and services

• SQY51 Antisense oligonucleotide drug candidate based on tricyclo-DNA (tc-DNA) chemistry, designed to treat Duchenne Muscular Dystrophy by skipping exon 51 of the DMD gene to restore a functional reading frame via the exon-skipping process. Administered intravenously (with preclinical feasibility of pulmonary route), suitable for approximately 10–15% of DMD patients eligible for exon-51 skipping therapy. Currently in Phase 1/2a (AVANCE-1) clinical trial at R. Poincaré Hospital AP-HP with FDA and EMA Orphan Drug Designations.
• Tricyclo-DNA (tc-DNA) ASO Technology Platform Proprietary technological platform enabling custom synthesis of next-generation antisense oligonucleotides from the tricyclo-DNA class, combined with a high-throughput screening cascade and 3D human DMD engineered muscle (mini-muscle) models for preclinical evaluation. Underpins the company's pipeline including SQY51 and additional ASO-tcDNA candidates for other DMD subpopulations.

Quantifiable outcome

• 34.4% pulmonary bioavailability demonstrated in preclinical studies
• +3 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered core.

• PHARMAColigo (UVSQ)coreStrategic or Co-development Partner · 30 January 2026Translational research unit at Université Versailles Saint Quentin (UR 20261 UVSQ) led by Professors Helge Amthor and Marcel Bonay. Collaborates with SQY Therapeutics to develop therapies for neuromuscular diseases using tricyclo-DNA chemistry. Forms unique Hospital-University Ecosystem (EHU) together with SQY Therapeutics and Raymond-Poincaré University Hospital (APHP-UVSQ).
• UMR1179 Inserm-UVSQcoreStrategic or Co-development PartnerResearch unit at UFR Simone Veil – Santé of University of Versailles Saint-Quentin-en-Yvelines (Université Paris-Saclay). SQY51 drug candidate is result of collaboration with this unit led by Luis Garcia.
• Université de Versailles Saint-Quentin-en-Yvelines (UVSQ)coreStrategic or Co-development PartnerUniversity hosting SQY Therapeutics' research activities at UFR Simone Veil – Santé until 2025. President Loïc Josseran attended laboratory inauguration. SQY Therapeutics benefits from the scientific and hospital-university environment and is located within the 17,000-company ecosystem of Saint-Quentin-en-Yvelines.
• Hôpital Raymond-Poincaré AP-HPcoreStrategic or Co-development PartnerUniversity hospital (APHP-UVSQ) where AVANCE-1 Phase 1/2a clinical trial is conducted. Part of the Hospital-University Ecosystem (EHU) with PHARMAColigo and SQY Therapeutics.

Scale indicators7 records

Recent moves7 records

Expansion highlights6 records

Emerging players

• Capricor Therapeutics: Late-stage biotech developing cell therapy and exosome-based treatments for DMD, addressing the same patient population through complementary rather than directly competing mechanisms.
• Avidity Biosciences: Develops antibody-oligonucleotide conjugates (AOCs) including programs for DMD, representing a next-generation ASO delivery platform that could compete with or complement SQY's tricyclo-DNA approach.
• Solid Biosciences: Clinical-stage gene therapy company targeting Duchenne Muscular Dystrophy with alternative modalities, competing for the same DMD patient population though with a different therapeutic approach.

Direct peers

• PTC Therapeutics: Develops and commercializes DMD therapies including ataluren (Translarna) for nonsense mutations, competing in the same Duchenne Muscular Dystrophy treatment market with overlapping pediatric rare disease infrastructure.
• Sarepta Therapeutics: Commercial-stage leader in DMD exon-skipping ASO therapies with FDA-approved products (eteplirsen, golodirsen, casimersen) targeting the same patient population as SQY51, representing the most direct competitive threat.
• Wave Life Sciences: Clinical-stage company developing stereopure antisense oligonucleotides including DMD exon-skipping programs, directly comparable as a smaller, oligonucleotide-focused biotech targeting the same disease space.
• Dyne Therapeutics: Clinical-stage biotech developing oligonucleotide therapeutics for DMD and other neuromuscular diseases using a conjugate-based approach, competing for the same exon-skipping patient population and investor capital.
• Nippon Shinyaku (NS Pharma): Markets viltolarsen (Viltepso) for DMD exon-53 skipping and is developing additional exon-skipping ASOs, directly competing in the same rare-disease ASO space as SQY Therapeutics.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biopharma with DMD development history (and broader neuromuscular pipeline) that competes for the same prescribers, patients, and payer budgets targeted by SQY51.
• Ionis Pharmaceuticals: Pioneer and leader in antisense oligonucleotide technology, holding foundational IP in the ASO modality that SQY's tricyclo-DNA platform builds upon, and partnering with multiple rare disease programs.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/sqy-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about SQY Therapeutics