Still, the only approved treatment for AMD in the EU, as well as worldwide, with demonstrated gain of visual acuity is the intravitreal administration of VEGF-inhibiting molecules as ranibizumab (Lucentis®), bevacizumab (Avastin^®) and aflibercept (Eyea^®). To be effective, ranibizumab and relatives require monthly intravitreal injections and are only beneficial in a subset of patients (15-20%) with exudative (neovascular) AMD.

 

The project TargetAMD

• developed protocols, devices and reagents for the transfection of very few somatic cells obtained from a iris biopsy (5,000-10,000 cells),
• developed protocols for the isolation, SB100X-mediated transfection and transplantation of the cells into the same patient within a single surgical session lasting approximately one hour.

 

The simplicity of the treatment protocol opens the door for the application in other genetic diseases amenable to cell transplantation. The clinical study proposed by TargetAMD, apply a novel technology to clinical gene therapy that translates an academic-grade non-viral, transposon-based gene delivery system to a protocol for clinical use in humans to treat AMD by

• developing clinically acceptable gene delivery modalities,
• optimising the transgene expression vector and improving its biosafety profile,
• developing new reagents and devices to be used for this approach, and
• translating the laboratory pre-clinical nucleic acids to clinical practise by an authorised and certified GMP-grade vector production.

 

In the context of the clinical trial all investigational medicinal products (IMP) will be produced to meet the quality requirements for GMP (good manufacturing practice) and GTMPs (gene therapy medicinal product). This will entail (a) consistency of cell isolation and transfection, (b) testing for genotoxicity and tumorigenicity, and (c) preparation of a database for final product specifications, which will be used as standard for future clinical trials and can be adapted for routine clinical use.

TargetAMD ensures conformity with the respective guidelines (GCP, GLP,GMP, and EU directives for standard transplantations) for the whole process of investigational medicinal product development, production, and clinical testing.

The implementation of a non-viral gene therapeutic approach will put European science and industry in a strategically important position.