SIRION Biotech Licenses Technology to National Institute of Allergy and Infectious Diseases to Boost Clinical Efficacy of Lentivector Gene Therapy for X-Linked SCID

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 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 programs.

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.

“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
LentiBOOST™ 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 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,” says Dr. Sabine Ott, VP, Business Development & Licensing at

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.


Corporate Contact
Dieter Lingelbach, COO
SIRION Biotech
[email protected]

Media Contact USA
Lisa DeScenza
Assistant Vice
President, Integrated Communications
[email protected]

BD & Licensing Contact
Sabine Ott, Ph.D., VP Licensing &
SIRION Biotech
+49 89-700-96199-19
[email protected]

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