Correcting Defective Genes
Modern gene therapy approaches necessitate exact gene delivery and expression control, suited to the highly complex in vivo environment of clinical applications. The SIRION technology platform offers versatile chances to identify and optimize the best possible approach for your R&D.
AAV engineering - optimize for clinical trials
Full customization service for Gene Therapy R&D
Optimize AAV tissue specificity and immunogenic profile
Adeno-associated virus are heavily utilized in modern Gene Therapy development. While standard AAV applications are suitable for pre-clinical target validation and proof-of-principle experiments, they can fall short during the big step into clinical application. SIRION Biotech’s AAV optimization platform enables you to address AAV limitations BEFORE you take that crucial step. All elements of your AAV strategy are considered and optimized with your project’s specific goals in mind.
The 3 tiers of successful AAV implementation:
Customized vector design and promotor choices to guarantee optimal expression of your genetic strategy in the targeted tissues.
AAV capsid mutations and surface variant constructions to optimize the tissue tropism.
AAV capsid surface modulation to optimize/minimize the immunogenic profile of your AAV strategy.
SIRION offers full project management capabilities for new AAV optimizations including a firm comprehension of all necessary patent frameworks and options to develop new intellectual properties within the optimization project.
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LentiTHERAPY™ - cell specific transduction technology
LentiTherapy™ is designed to enable cell type specific transduction and improve standard transduction efficiencies. The system is a great candidate for clinical research applications because of its universal applicability and non-toxic chemistry. Proven effective in CD30 and EGFR positive cell types.
The LentiTherapy™ 3 hit strategy to target therapeutically relevant cell types
Step 1: Retargeting antibody fragments on the virus envelope. These single-chain antibody fragments have a high affinity and specificity to peripheral proteins of cell surfaces, allowing targeted precision of viral transduction.
Step 2: LentiBOOST™ transduction enhancer increases the permeability of cell membranes, allowing lentiviruses easy access. The chemical basis for LentiBOOST™ is non-toxic and is used as a standard component in pharmaceutical pill formulation. This unique transduction enhancer is especially potent when used on non-adherent cells such as B-cell and T-cell transduction.
Step 3: Spinoculation increases the number of virusparticles binding to the cellsthrough centrifugal inoculation, enhancing the effectivenessof LentiBOOST™ and the retargeted envelope even further.
Due to its non-toxicity, the process is well suited for basic research as well as early clinical applications. LentiTherapy™ achieves sufficient genetic modification at low MOIs (less or equal to 1).
The figures show transduction experiments in KARPAS-299, SUP-M2 and SUDHL-1 cell lines incubated at MOI 10 or with copGFP-coding lentiviral particles +/- a spinoculation protocol, LentiBOOST (TM) and retargeted scFv-CD30 VSV-G lentivirus.
LentiTherapy™ is effective independent of cell type. The steps of this system influence each other synergistically, as demonstrated in the figure above. The treatment is effective at different MOIs and ideal for treating otherwise hard to transduce,therapeutically relevant cell types.
The LentiBOOST™transduction enhancer has been reported by collaborators from the academic sciences at the NYU Langone Medical Center to increase T-cell transduction significantly.
SIRION Biotech is searching for collaboration partners to help actively develop the application of the LentiTherapy™ Systemin pre-clinical and early clinical stages.
Höfig et al.,Poloxamer synperonic F108improves cellular transduction with lentiviral vectors. J. Gene Med. 14:549-60 (2012)
Höfig et al.,Systematic improvement oflentivirus transduction protocols by antibodyfragments fused to VSV-G as envelope glycoprotein. J. Biomaterials(2014)
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