MARTINSRIED, Germany, June 11, 2019 – SIRION Biotech GmbH (“SIRION”) today announced the entry into a license agreement with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, a leading clinical research institute in the gene therapy space, pursuant to which NIAID has licensed SIRION’s LentiBOOST™ technology to improve clinical efficacy of its hematopoietic stem cell gene therapy programs.
X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder that is caused by mutations of a single gene, ILR2G. The mutation causes a 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.
To further enhance the clinical response in this patient group, SIRION licensed its LentiBOOSTTM technology to NIAID. The NIAID will be entitled to apply this 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.
By use of the LentiBOOSTTM technology, it is the expressed goal of the NIAID investigators to further improve a durable clinical response in SCID-X1 patients receiving this gene therapy. A key element to achieve this goal was to achieve a consistently good target level of integrated genome copies for the therapeutic gene into engrafted CD34+ cells. The NIAID investigators found that when they included the LentiBOOSTTM 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.
“Gene therapies stand at a crossroads,” explains Dr. Christian Thirion, CEO of SIRION, a leading provider of viral vector technologies. “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 LentiBOOSTTM helps to reduce manufacturing costs by lowering the amount of lentiviral vectors needed for production of the cell product. 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.”
“SIRION developed a tailored licensing model for research institutions and hospitals providing royalty-free access to its LentiBOOSTTM technology and favorable supply conditions for the GMP material for their clinical trials when entirely financed by non-profit sources. LentiBOOSTTM 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,” says Dr. Sabine Ott, VP, Business Development & Licensing at SIRION.
About SIRION Biotech GmbH
SIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. Now SIRION offers one of the world’s most comprehensive viral vector technology platforms based on lenti-, adeno-, and adeno-associated viruses which expedites gene therapy research and advances drug development. SIRION is becoming a partner of choice in this growing sector. LentiBOOST™ has been used in a number of clinical trials from early stage clinical Phase 1/2 through late stage clinical Phase 3 trials and demonstrated clinical success in improving transduction of the therapeutic vector.
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