Search the database for deliberate release of GM medicinal products

Displaying 1 - 20 of 26

EU record number	Title	Company / Sponsor	Treated organism	Genetic modification
B/BE/25/BVW9	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	VectorY Therapeutics B.V.	Humans	Non-replicating recombinant AAV vector with a AAV5.2 capsid and carrying the transgene corresponding to the single-chain variable fragment that binds to human TDP-43
B/BE/25/BVW8	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	Solid Biosciences Inc.	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human micro-dystrophin (h-µD5) gene
B/BE/25/BVW7	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) in a Real-World Context in Subjects with Neovascular Age-Relate	AbbVie Deutschland GmbH & Co. KG	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV8 carrying the human anti-vascular endothelial growth factor (VEGF) antigen-binding fragment.
B/BE/25/BVW6	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 consecut	University of Tokyo	Humans	A live attenuated virus (LAV)-MV-NiV, including the full genome of a live-attenuated measles virus (MV) strain, genetically engineered to express the glycoprotein G of the Nipah virus.
B/BE/25/BVW5	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	Asklepios BioPharmaceutical (AskBio)	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV2i8 carrying the transgene encoding for the human activated Inhibitor-1c ( I-1c)
B/BE/25/BVW4	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	SparingVision	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV2 carrying the G protein-gated inwardly rectifying potassium (GIRK) channel 1, mutated for an F137S amino-acid substitution (GIRK1(F137S)) gene
B/BE/25/BVW3	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	CSL Behring LLC	Humans	Recombinant adeno associated viral vector containing the codon-optimised human PADUA factor IX (FIX) gene
B/BE/25/BVW2	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	AstriVax NV	Humans	Full genome of the live attenuated yellow fever virus (YFV) strain 17D (YF17D) containing the sequence of the HBV core antigen (HBc)
Only notified under the "contained use" procedure. Dossier submitted on 10 September, 2025 .	A phase 2, Multicenter, Open-Label Study Of CC-97540 (BMS-986353), CD-19-Targeted NEXT-T Chimeric Antigen Receptor (CAR) T Cells, in Participants with Active Systemic Lupus Erythematosus (SLE) (Including Lupus Nephritis) with Inadequate Response to Glucoc	Celgene	Humans	CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 25 March, 2025 .	EXACT 2 is a randomized, multicenter, double-blind, placebo-controlled Phase 2b trial designed to evaluate the safety, feasibility, and efficacy of percutaneous endovascular catheter-facilitated intramyocardial administration of XC001 in patients with ref	XyloCor Therapeutics	Humans	VEGF
Only notified under the "contained use" procedure. Dossier submitted on 20 March, 2025 .	A Phase 3, Randomized, Open-Label, Multicenter Study to Compare the efficacy and Safety of BMS-986393, a GPRC5D-directed CAR-T Cell Therapy, Versus Standard Regimens in Adult Participants with Relapsed or Refractory and Lenalidomide-refractory Multiple My	Celgene	Humans	GPRC5D CAR
B/BE/22/BVW6	A phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)	Sarepta Therapeutics	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
B/BE/22/BVW5	A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchennne Muscular Dystrophy (ENVOL)	F. Hoffmann-La Roche	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the human micro-dystrophin (hMicro-Dys) gene
B/BE/22/BVW4	A Phase I/II, Multicenter, Open-Label Study of Nous-209 Genetic Vaccine for the Treatment of Microsatellite Unstable Solid Tumors	Nouscom Srl	Humans	The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the FSP neoantigens; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the same neoantigens.
Only notified under the "contained use" procedure. Dossier submitted on 2 September, 2022 .	A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant	Quell Therapeutics Limited	Humans	BCMA CAR; shRNA CD3ζ; tCD34
Only notified under the "contained use" procedure. Dossier submitted on 25 July, 2022 .	A Phase I/II study to evaluate the feasibility, safety and preliminary efficacy of point-of-care manufactured anti-CD19 CAR T in subjects with relapsed or refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) (CP0101-CLL) – Eup	CellPoint B.V. (a Galapagos company)	Humans	CD19 CAR
Only notified under the "contained use" procedure. Dossier submitted on 7 June, 2022 .	A phase I/II open label, multicenter study evaluating the feasibility, safety and efficacy of point-of-care manufactured anti-BCMA CAR T cells (BCMACP03) in subjects with relapsed/refractory Multiple Myeloma (r/r MM) (Papilio-1)	CellPoint B.V. (a Galapagos company)	Humans	BCMA CAR
B/BE/20/BVW6	Dossier withdrawn by the notifier	Pfizer	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene
B/BE/20/BVW5	Phase 1b Study to Evaluate the Safety, Tolerability, and Anti-Tumor Activity of Nous-PEV, with pembrolizumab, in Patients with Unresectable Stage III / IV Cutaneous Melanoma and with Stage IV NSCLC	Nouscom Srl	Humans	The study involves two GMOs: (i) A replication-incompetent adenovirus (GAd20 with deletions of the viral E1, E3 and E4 coding regions) isolated from a gorilla and encoding the PEV neoantigens gene; (ii) an attenuated, replication-defective orthopoxvirus (Modified Vaccinia virus Ankara), encoding for the neoantigens gene.
B/BE/20/BVW4	A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy	Pfizer	Humans	Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene