Collaborations and membership
Participating in collaborative projects are excellent opportunities for GenoSafe to strengthen its research and innovation activities.
GenoSafe is a major partner in national and transnational cooperation projects gathering key industrial and academic takeholders developing Advanced Therapy Medicinal Products.
Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome
The CureCN consortium brings together 11 European partners from academia, hospitals, healthcare companies and patient organisations to fight the so far incurable liver disease Crigler-Najjar (CN). Together, they aim to validate a gene therapy within a clinical trial to provide the first curative treatment against CN. GenoSafe is responsible for setting up a part of the Quality Controls, and for the biodistribution – bioexpression.
Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB
More informations will come.
GenoSafe is responsible for the biodistribution, viral shedding and immunogenicity analysis.
Transposon-based, targeted gene therapy to treat age-related macular degeneration
Age-related Macular Degeneration (AMD), a neurodegenerative disease of the retina, is a major cause of blindness in elderly people. Due to the aging population, AMD has been referred to as a “time bomb” in society.
This program comprises 13 partners from 4 universities, 2 hospitals, 3 research institutes and 3 industrial partners from 7 countries, who together will develop protocols, reagents and technology to successfully complete a phase Ib/IIa clinical trial for the treatment of neovascular AMD by collecting, transfecting autologous pigment cells with the PEDF gene using the SB100X transposon system and transplanting the transfected cells to the subretinal space of AMD patients within one surgical session.
Coordinator: Hôpitaux Universitaires de Genève
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD)
This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD).
Net4CGD proposes future trials to achieve (i) effective transduction of hematopoietic cells, (ii) physiological expression of the transgene and (iii) long-term engraftment of gene modified cells.
Clinical trial of gene therapy for MPS VI – a severe lysosomal storage disorder
Mucopolysaccharidosis VI (MPS VI) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement.
The MeuSIX consortium plans to conduct a multicenter phase ½ clinical trial to investigate the safety and efficacy of AAV-mediated gene therapy in patients with PMS VI.
coordinator: Fondazione Telethon
PreIndustrial Gene Therapy Vector Consortium
The consortium is a preindustrial demonstrator for the manufacturing and control of therapeutics for preclinical and clinical studies in gene therapy. The work program includes three objectives: (i) reinforcing the process development activities in manufacturing and controls of gene therapy vectors to increase IP and technical competitiveness; (ii) up scaling manufacture and controls of vectors for preclinical studies, which include proof-of-concept and regulatory toxicology; (iii) translation of large-scale technology and processes to GMP cores in order to facilitate and support academic (and private) investigators for Phase I/II gene therapy trials.
GenoSafe in an active member of:
- ESGCT, European Society of Gene and Cell Therapy
- SFTCG, Société Française de Thérapie Cellulaire et Génique
- ASGCT, American Society of Gene and Cell Therapy
- Genopole, France’s leading biotech cluster for biotechnologies and research in genomics and genetics
- Medicen, cluster for innovative therapies and advanced technologies in healthcare
The ATMPs environment is very innovative. Scientific, technological and regulatory aspects are constantly evolving.
At GenoSafe we believe that we can do more by collaborating than by ourselves, thus we generate added value for our customers. We have established non-exclusive partnerships with the following CROs and biotechs.
CERB is a GLP Tox facility, providing every kind of models and having a unique experience in gene therapy in Europe. Its services are complementary of GenoSafe’s and we are proud to propose a global offer, from the selection of your models to the analyzed data to be included in the clinical trial approval dossier.
Genewerk provides unique worldwide experience in assessing vector safety and efficacy in gene therapy and immune repertoire studies, bioinformatics, Next Generation Sequencing and associated regulatory demands.