Blueprint Genetics
Blueprint Genetics is recorded as a service provider in 17 clinical trials. Explore its recorded services, study phases and therapeutic areas, with trial-level operational learnings where available.
Clinical trial experience
Showing 17 of 17 trials · Latest update first
A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
Sponsor F. Hoffmann-La Roche AG
- Start (EU)
- Latest update
- Estimated end
A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
Sponsor Italfarmaco S.p.A.
- Start (EU)
- Latest update
- Estimated end
A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants with Dravet Syndrome (ARGUS Trial)
Sponsor Epygenix Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase 3, Parallel-group Treatment, Blinded, Randomized, Placebo-controlled Study to Assess the Efficacy and Safety of Pegtibatinase Administered Subcutaneously in Addition to Standard of Care in Participants with Classical Homocystinuria due to Cystathionine Beta Synthase Deficiency (HARMONY)
Sponsor Travere Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
Sponsor Sarepta Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
Sponsor Solid Biosciences Inc.
- Start (EU)
- Not reported
- Latest update
- Estimated end
A Phase 1/2 Open-Label, Ascending Dose, Multicentre Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)
Sponsor Aviadobio Limited
- Start (EU)
- Latest update
- Estimated end
A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (ASTRA).
Sponsor Splicebio S.L.
- Start (EU)
- Latest update
- Estimated end
A Phase 3, randomized, double-blind, placebo-controlled, multicenter study of mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections.
Sponsor X4 Pharmaceuticals Inc.
- Start (EU)
- Latest update
- Estimated end
A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia
Sponsor Moderna Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058) Administered Subcutaneously in Subjects with Cystathionine Beta-Synthase-Deficient Homocystinuria (COMPOSE)
Sponsor Travere Therapeutics Switzerland GmbH
- Start (EU)
- Not reported
- Latest update
- Estimated end
A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis
Sponsor UCB Biopharma
- Start (EU)
- Latest update
- Estimated end
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD
Sponsor Rocket Pharmaceuticals Inc.
- Start (EU)
- Latest update
- Estimated end
Osprey: An Open-label Study to Investigate the Safety, Tolerability, and Exposure of Single Ascending Doses of the Antisense Oligonucleotide STK-002 in Patients with Autosomal Dominant Optic Atrophy
Sponsor Stoke Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
EMPEROR: A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients with Dravet Syndrome
Sponsor Stoke Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome
Sponsor Denali Therapeutics Inc.
- Start (EU)
- Latest update
- Estimated end
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam - ASCEND
Sponsor Biogen Idec Research Limited
- Start (EU)
- Latest update
- Estimated end
Questions about this trial experience
What services are recorded for Blueprint Genetics?
The 2026-10-09 snapshot links Blueprint Genetics to 17 distinct CTIS trials. Recorded services include Laboratory services, Genetic testing, Auto-antibody testing, DMD genotype analysis, among 8 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 Blueprint Genetics?
The most frequently recorded therapeutic areas are Genetic and Congenital Disorders, Neurology, Metabolic Disorders, Musculoskeletal. Recorded phases include Phase 2, Phase 3, Phase 1. Use the filters to inspect the supporting studies; missing tags are not evidence of absent experience.
In which countries are trials linked to Blueprint Genetics conducted?
The linked trials record locations in 19 countries, including Italy, Spain, Germany, France, Poland. These are trial locations, not confirmed office locations or countries where Blueprint Genetics delivered services.
What can this page tell me about Blueprint Genetics's trial experience?
Browse 17 distinct trials with recorded roles, sponsor names, available study dates and links to CTIS source records. No trial-level operational findings are recorded in this snapshot. 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 3 recorded name variants under Blueprint Genetics. Legacy brands remain separate unless explicitly matched in the name registry.
View recorded company names
- Blueprint Genetics
- Blueprint Genetics Inc.
- Blueprint Genetics Oy