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NIAID allergy institute chooses SIRION LentiBOOST™ technology to strengthen SCID-X1 immunodeficiency study
Martinsried, Germany: – Leading viral vector technology specialist SIRION Biotech International has concluded a license agreement for its LentiBOOST™ technology with the US Government’s National Institute of Allergy and Infectious Diseases (NIAID).
NIAID has licensed LentiBOOST™ to improve clinical efficacy of its hematopoietic stem cell gene therapy programs, and in particular its current study of SCID-X1, the X-linked severe combined immunodeficiency disorder caused by mutations of a single gene, ILR2G.
Threat to children
SCID-X1 causes severe loss of T cells, B cells, and natural killer (NK) cells, leading to profound impairment of the immune system of affected children. Infants born with this severe immune deficiency require a life-saving hematopoietic stem cell transplant in infancy. When the transplant is performed without chemotherapy, most achieve only partial immune reconstitution, continue to have significant chronic medical problems, and also require life-long treatment with monthly injections of supplemental immunoglobulin (antibodies).
Until 2016 there was no treatment to improve the partially functioning immune system and associated medical problems affecting these older children and young adults with SCID-X1. In a clinical study at NIH, a small cohort of these older children and young adult patients with SCID-X1 achieved significant clinical benefits including production of their own immunoglobulins when treated with lentivector gene therapy to express healthy ILR2G. This ongoing study is led by Dr. Suk See De Ravin and Dr. Harry L. Malech of the Genetic Immunotherapy Section in NIAID’s Laboratory of Clinical Immunology and Microbiology.
Boosting transduction efficiency
By use of the LentiBOOST™ technology, it is NIAID’s expressed goal to achieve a more durable clinical response in SCID-X1 patients receiving this gene therapy. A key element in achieving this is to increase the target level of integrated genome copies for the therapeutic gene into engrafted CD34+ cells. NIAID investigators have found that by including the LentiBOOST™ technology reagent into their standard GMP transduction manufacturing process, they reliably increased transduction efficiencies many-fold, achieving genome copy numbers of up to 1.5 to 3 copies per cell in the four patients who have been treated recently with cells manufactured with the new transduction method. These early results are highly promising in terms of achieving consistently increased target levels of gene correction.
Applying LentiBOOST™ across the NIAID portfolio
Following this pilot program, the NIAID will be entitled to apply LentiBOOST™ technology for early clinical development of its SCID-X1 trial as well as for its complete gene therapy portfolio including multiple stem-cell and T-cell programs.
“Gene therapies stand at a crossroads,” said SIRION Biotech CEO Dr. Christian Thirion.
“Delivery of therapeutic genes by viral vectors is the most critical part of gene and cell therapy approaches nowadays. Our contribution is the optimization of viral vector gene delivery allowing for superior transduction efficiency and long-term gene expression in treated patients. Furthermore, the use of transduction enhancers like LentiBOOST™ helps to reduce manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product,” said Dr. Thirion.
“It fills us with pride to be working with Dr. Malech and Dr. De Ravin at the NIH to improve a promising treatment option for SCID-X1 patients,” he commented.
Tailored licensing model
Dr. Sabine Ott, SIRION VP for Business Development & Licensing, added:
“We have developed a tailored licensing model for research institutions and hospitals providing royalty-free access to its LentiBOOST™ technology and favorable supply conditions for the GMP material for their clinical trials when entirely financed by non-profit sources. LentiBOOST™ is currently used in several clinical trials in the USA and Europe up to Phase 3 and has proven to be safe and clinically effective.”
About SIRION Biotech
SIRION Biotech International is a wholly-owned subsidiary of SIRION Biotech GmbH providing custom engineering and manufacturing services of viral vectors for the life sciences industry.
Founded at Martinsried, near Munich, in 2007, SIRION Biotech has become Europe’s leading commercial supplier of viral vectors used for genetic research, clinical target validation, gene therapy and vaccination studies. It is SIRION’s mission to change the paradigm for viral vector supplies, with their ability to develop and supply all major viral vector types (adenovirus, lentivirus, AAV, etc.) within a matter of weeks at the concentration titers and quantities needed for preclinical human and animal testing.
Its unique focus on improving transduction efficiencies and safety make SIRION Biotech a valuable technology partner for gene and cell therapy trials. LentiBOOST™ transduction reagent is actively used to improve, among others, hematopoietic cell transductions in clinical trials. NextGen AAV capsid evolution projects aim to improve tissue targeting and immune escape of capsids to usher in a new generation of therapeutics for international gene therapy companies.
SIRION offerings include the transformational RNAiONE™ knockdown validation platform along with a full range of virus related services, ranging from particle production to virus driven cell modelling.
Headquartered at North Bethesda, Maryland, the National Institute of Allergy and Infectious Diseases (NIAID) is one of the 27 institutes and centers that make up the National Institutes of Health (NIH), an agency of the United States Department of Health and Human Services (HHS).
NIAID’s mission is to conduct basic and applied research to better understand, treat, and prevent infectious, immunologic, and allergic diseases, with in-house laboratories in Maryland and Montana. The institute also funds research conducted by scientists at institutions in the United States and throughout the world and works closely with partners in academia, industry, government, and non-governmental organizations in multifaceted and multidisciplinary efforts to address emerging health challenges such as the pandemic H1N1/09 virus.
NIAID traces its immediate origins to October 1948, when the Rocky Mountain Laboratory and the Biologics Control Laboratory were joined with the NIH Division of Infectious Diseases and Division of Tropical Diseases to form the National Microbiological Institute. In 1955, Congress changed its name to the National Institute of Allergy and Infectious Diseases to reflect the inclusion of allergy and immunology research.
Further information at: https://www.niaid.nih.gov/