Policy & Regulation
Sirion Biotech Licenses Technology to National Institute of Allergy and Infectious Diseases to Boost Clinical Efficacy of Lentivector Gene Therapy for X-Linked SCID
13 June 2019 - - German biotechnology company Sirion Biotech GmbH has entered into a license agreement with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, a 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, the company said.
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 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 LentiBOOST 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 programmes.
By use of the LentiBOOST 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 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.
Sirion Biotech was founded in 2005 to develop the next generation of viral vector technologies for gene and cell therapy as well as vaccine development.
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.


Related Headlines