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Safety Assessments in
Clinical Trials
At GeneWerk, we bring together gold standard technology and 20+ years of pioneering in the industry to make gene therapies safer for patients. With a focus on vector safety, characterization, and functionality analysis, our dedicated scientists and technicians work in compliance with GCP standards in a BSL-2 classified state-of-the-art genomics and bioinformatics laboratory in Heidelberg, Germany.
Our track record speaks for us – market authorizations obtained by our clients
Our two decades of expertise in vector safety assessment, vector integration profiling, and clonality assessment in clinical gene and cell therapy studies enable us to support our clients to bring their breakthrough gene therapies to successful market authorization.
GeneWerk’s expert team was involved in the safety assessment of these ground-breaking, and often first-in-class, cell and gene therapies.
Enabling safety during gene therapy clinical development
UniQure’s Glybera® – AAV-based gene therapy for lipoprotein lipase deficiency (LPLD), 2012: Although it was withdrawn from the market due to commercial failure, Glybera showed to the world the hidden potential of gene therapies to treat diseases often considered ‘hard-to-treat’ or incurable.
Orchard therapeutics’s Strimvelis® – RV-based gene therapy for treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), 2016: Strimvelis® is an “autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector (RV) that encodes for the human ADA cDNA sequence.” It is the first ex vivo autologous gene therapy approved by the EMA.
Novartis’s Kymriah® – LV-based CAR-T cell therapy for acute lymphoblastic leukemia (ALL) and diffuse large B-cell lymphoma (DLBCL), 2018: Kymriah® was one of the first CAR-T cell therapies approved by FDA. It is a CD19‑directed relapsed, refractory ALL and DLBCL and has received approval in the EU as well.
Bluebird bio’s Zynteglo®– LV-based gene therapy for transfusion-dependent β-thalassemia (TDT), 2019: Zynteglo® is the first gene therapy for TDT and was given conditional marketing authorization in the EU. It consists of an “autologous CD34+ cell-enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene.”
Orchard therapeutics’s Libmeldy®– LV-based gene therapy for metachromatic leukodystrophy (MLD), 2020: Libmeldy® received EU market authorization in 2020. It consists of an “autologous CD34+ cell-enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene.”
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