By SIRION Biotech…
SIRION Biotech Viral Vector Engineering
SIRION Biotech offers highly advanced viral vector technology and vector manufacturing platforms based on lentiviruses, adenoviruses, and adeno-associated viruses (AAVs).
These technologies expedite gene therapy research and advance drug development, making SIRION a partner of choice in these dynamic sectors.
SIRION`s gateway to fast vector production is built on extensive experience in process development (USP & DSP) of AAV, lentiviruses and adenoviruses.
The success of gene therapies depending on delivery of therapeutic genes by viral vectors is boosted by superior transduction efficiency and long-term gene expression in treated patients, made possible by SIRION’s BOOST™ transduction enhancement technology that enhance the efficiency of viral gene transfer into difficult to transduce mammalian and rodent cells, increasing virus uptake without toxicity. They also reduce manufacturing costs by lowering the number of lentiviral vectors needed for production of the cell product.
LentiBOOST™ is a highly effective, non-cytotoxic transduction enhancer that increases lentiviral transduction of difficult cell types, such as T- or B-cells. It increases production of lentiviral vectors in preclinical and clinical applications.
LentiBOOST™ has demonstrated clinical success in improving transduction of therapeutic lentivectors, especially in CAR-T cell experiments and R&D, and has been used in a number of clinical trials from early stage clinical Phases I and Phase II through late stage clinical Phase 3 trials.
For example, NIAID researchers investigating improved durable clinical response in SCID-X1 patients receiving gene therapy by increasing number of integrated genome copies of therapeutic gene into engrafted CD34+ cells have included LentiBOOST™ reagent into standard GMP transduction manufacturing process. This reliably increased transduction efficiencies many-fold, achieving genome copy numbers of up to 1.5 to 3 copies per cell in patients treated with cells manufactured using this new transduction method.
LentiBOOSTTM is currently used in several clinical trials in the USA and Europe up to Phase III and has proven to be safe and clinically effective.
RNAiONE™ customized gene silencing for target validation and drug discovery
SIRION Biotech has developed its base algorithmic identification technology to the point where it can now routinely identify targeted small hairpin RNA (shRNA) sequences with knockdown results of above 90%.
SIRION Biotech now offers RNAiONE™ as a guaranteed path to define the most effective shRNA sequences for target gene identification and validation for further translation into SIRION’s lentiviral vector systems and viral vector manufacturing platforms for adenovirus and AAV vector production.
RNAiONE is readily applicable to gene loss of function studies, gene expression silencing and target identification & validation for early point drug discovery.
Custom inducible vectors
SIRION Biotech’s inducible expression platform is based on the latest 3G TET technology. This guarantees a high sensitivity without leakiness. SIRION offers inducible lentiviral vector systems that can be applied to establish stable inducible cell models, circumvent cell adaption to stable genetic modification, or enable characterization of toxic gene modulations.
The inducible expression platform is based on a TET-inducible All-in-ONE vector backbone, with TET-inducible TWO-vector technology for large constructs that are highly sensitive without leakiness and offer knockdown efficiency of at least 80% on mRNA level when used in combination with RNAiONE.