This page is a temporary, static list of the latest applications for clinical trials with GMOs.
| EU record number | Company / Sponsor | Title |
| B/BE/26/BVW3 | GENETHON | 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 with Duchenne Muscular Dystrophy |
| B/BE/26/BVW2 |
AAVantgarde Bio UK Ltd.
|
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE) |
| B/BE/25/BVW9 |
VectorY Therapeutics B.V.
|
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002 |
| B/BE/25/BVW8 |
Solid Biosciences Inc.
|
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 |
| B/BE/25/BVW7 | AbbVie | A Randomized, Controlled, Partially Masked, Phase 3b Study to Assess the Injection Burden, Efficacy, Safety, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec (ABBVRGX-314) |
| B/BE/25/BVW6 |
University of Tokyo
|
A phase 1, placebo-controlled, randomised, participant- and assessor-blind, single-centre study to assess the safety and immunogenicity of 2 dosages of Nipah measles vector vaccine (MV-NiV) administered subcutaneously either a single dose or as 2 consecutive doses at 4-week interval, in healthy non-exposed volunteers, aged 18-40 years |
| B/BE/25/BVW5 |
Asklepios BioPharmaceutical (AskBio)
|
GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure
|
| B/BE/25/BVW4 | SparingVision | An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors |
| B/BE/25/BVW3 |
CSL Behring LLC
|
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B |
| B/BE/25/BVW2 | AstriVax NV | A randomised, double-blind, placebo-controlled, single centre, Phase I study to evaluate the safety, reactogenicity and immunogenicity of AstriVax’ investigational therapeutic hepatitis B virus (HBV) vaccine (AVX70371) in healthy adults aged 18 to 40 year |
| B/BE/24/BVW6 |
AstriVax NV
|
A randomised, double-blind, placebo-controlled, multi-centre, Phase I study to evaluate the safety, reactogenicity and immunogenicity of AstriVax’ investigational therapeutic hepatitis B virus (HBV) vaccine (AVX70371) in adult patients with chronic HBV
|
| B/BE/24/BVW5 | Pfizer | Phase 3, open-label, single-arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C ≤2%) (BeneGene-2) |
| B/BE/24/BVW4 |
Sarepta Therapeutics
|
A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4 |