Genosafe S.A.S
Genosafe S.A.S is recorded as a service provider in 19 clinical trials. Explore its recorded services, study phases and therapeutic areas, with trial-level operational learnings where available.
Clinical trial experience
Showing 19 of 19 trials · Latest update first
Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys
Sponsor Genethon
- Start (EU)
- Latest update
- Estimated end
A single-arm, open-label, multi-centre, phase I/II first-in-human study evaluating the safety and clinical activity of QEL-005, an autologous CAR T-regulatory cell therapy treatment targeting CD19, in patients with diffuse cutaneous systemic sclerosis (dcSSc) and in patients with difficult to treat rheumatoid arthritis (D2TRA).
Sponsor Quell Therapeutics Limited
- Start (EU)
- Latest update
- Estimated end
(19429) A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A
Sponsor Bayer AG
- Start (EU)
- Latest update
- Estimated end
A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA, for the treatment of early onset Metachromatic Leukodystrophy (MLD)
Sponsor Orchard Therapeutics (Europe) Limited
- Start (EU)
- Latest update
- Estimated end
- Not reported
Operational learnings available5 trial-level findings · Read insights
- The study was authorized and conducted at a single clinical site in Italy, and all treated subjects received OTL-200 in Italy.
- The planned sample size was based on feasibility in rare MLD rather than statistical considerations.
- Protocol version 6 expanded eligibility to permit enrollment and treatment of early-juvenile MLD at early symptomatic stages before rapid disease progression.
- Protocol version 7 specified arrangements for remote follow-up visits, including assessments by local healthcare professionals or teleconferencing.
- Protocol version 8 reduced required study long-term follow-up to a minimum of 5 years and allowed rollover to a 15-year post-treatment follow-up study.
These findings describe the trial, not the service provider’s performance or responsibility.
A Phase 1-2 multicenter study to evaluate the safety and tolerability of intravenous ATA-100, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I)
Sponsor Atamyo Therapeutics
- Start (EU)
- Latest update
- Estimated end
- Not reported
A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
Sponsor Ultragenyx Pharmaceutical Inc.
- Start (EU)
- Latest update
- Estimated end
CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy
Sponsor Genethon
- Start (EU)
- Latest update
- Estimated end
- Not reported
A phase I/II open label study to assess safety, feasibility and efficacy of ex vivo expanded, autologous haematopoietic stem and progenitor cell populations that contain CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA in children with autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene.
Sponsor Fondazione Telethon Ets
- Start (EU)
- Latest update
- Estimated end
- Not reported
A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT)
Sponsor Orchard Therapeutics (Europe) Limited
- Start (EU)
- Latest update
- Estimated end
A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant
Sponsor Orchard Therapeutics (Europe) Limited
- Start (EU)
- Latest update
- Estimated end
- Not reported
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa.
Sponsor Gensight Biologics
- Start (EU)
- Latest update
- Estimated end
An open-label, phase 2 trial to evaluate the efficacy and safety of a single intravenous administration of GNT0003 (an adeno-associated viral vector expressing the UGT1A1 transgene) following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar syndrome requiring daily phototherapy and presenting pre-existing anti-AAV8 antibodies.
Sponsor Genethon
- Start (EU)
- Latest update
- Estimated end
- Not reported
A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Participants with Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa
Sponsor Aavantgarde Bio S.r.l.
- Start (EU)
- Latest update
- Estimated end
A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
Sponsor Ultragenyx Pharmaceutical Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy
Sponsor Orchard Therapeutics (Europe) Limited
- Start (EU)
- Latest update
- Estimated end
- Not reported
A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson's Disease
Sponsor Vivet Therapeutics
- Start (EU)
- Latest update
- Estimated end
ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients
Sponsor Astellas Gene Therapies Inc.
- Start (EU)
- Latest update
- Estimated end
An open label, non-randomized trial to evaluate the safety and efficacy of a single infusion of OTL-200 in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD)
Sponsor Orchard Therapeutics (Europe) Limited
- Start (EU)
- Latest update
- Estimated end
- Not reported
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression
Sponsor eyeDNA Therapeutics
- Start (EU)
- Latest update
- Estimated end
- Not reported
Questions about this trial experience
What services are recorded for Genosafe S.A.S?
The 2026-10-09 snapshot links Genosafe S.A.S to 19 distinct CTIS trials. Recorded services include Laboratory services, VSV-G RNA PCR testing for RCL evaluation, Humoral and cellular immune response, Humoral immunomonitoring analysis, among 6 recorded service labels. These are study-specific roles, not a complete or independently verified capability catalogue.
Which therapeutic areas and study phases are recorded for Genosafe S.A.S?
The most frequently recorded therapeutic areas are Genetic and Congenital Disorders, Musculoskeletal, Neurology, Blood Disorders. Recorded phases include Phase 1, Phase 2. Use the filters to inspect the supporting studies; missing tags are not evidence of absent experience.
In which countries are trials linked to Genosafe S.A.S conducted?
The linked trials record locations in 8 countries, including France, Italy, Germany, Spain, Netherlands. These are trial locations, not confirmed office locations or countries where Genosafe S.A.S delivered services.
What can this page tell me about Genosafe S.A.S's trial experience?
Browse 19 distinct trials with recorded roles, sponsor names, available study dates and links to CTIS source records. 1 trials have operational findings that can inform further due diligence. Trial counts and findings do not measure the provider’s performance or establish responsibility for study outcomes.
About this data & company names
One current approved profile per CTIS trial. Services reflect recorded roles, not a complete capability catalogue or a performance ranking. Missing tags mean the information was not recorded. Country tags describe trial locations, not the provider’s service territory.
Start is the reported EU start date; latest update is the CTIS publication date; estimated end is the reported global estimate, which may be in the past. Missing dates are shown as “Not reported”. Operational learnings describe the trial as a whole and do not establish the provider’s responsibility.
Clear service synonyms are grouped; otherwise the source label is retained. Service tags show the recorded roles for each trial. This page groups 1 recorded name variant under Genosafe S.A.S. Legacy brands remain separate unless explicitly matched in the name registry.
View recorded company names
- Genosafe S.A.S.