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Press Release
*SIRION Biotech and Acucela Set Sights on Gene Delivery Tools for Ocular
Gene Therapy*
MUNICH, GERMANY (January 8, 2018) - SIRION Biotech GmbH ("SIRION"), a world
leader in virus based technology innovation and gene delivery expert
providing custom services to commercial partners worldwide, announced today
that it signed a two year development agreement with Acucela Inc.
("Acucela"), a clinical-stage ophthalmology company and wholly-owned
subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596)
committed to translating innovation into a diverse portfolio of drugs and
devices to preserve and restore vision for millions of people worldwide, to
establish optimized AAV vectors for clinical applications in ocular gene
therapy. With this cooperative effort, Acucela bolsters its move into the
fast growing gene therapy market to find a genetic cure for retinitis
pigmentosa (RP). Key terms of the agreement include milestone payments to
SIRION, with additional royalties to be paid on net sales from resulting
products or therapies.
RP is the most common form of inherited retinal degeneration. Onset is
usually in childhood and progresses to complete blindness by around age 40.
It is estimated that approximately 1 in 4000 people are affected in their
lifetime(1), and approximately 1.5 million people worldwide are affected by
the disease(2). RP describes a family of slowly progressing diseases that
are caused by a variety of autosomal dominant, autosomal recessive and
x-linked mutations that lead to photoreceptor degeneration. It is a complex
indication with over 100 pathogenic mutations identified(3). To date, no
pharmacological treatment is available.
Recombinant adeno associated virus (rAAV) vectors are considered the best
and most promising gene delivery system for therapeutic applications and
have been shown in preclinical experiments to successfully deliver human
rhodopsin (hRho) to the retina. Acucela, together with SIRION and an
academic partner consortium, aims to develop the next generation of rAAV
vectors. The goal is to secure new and modified AAV capsids that will ensure
that the therapeutic viral particles exhibit a safe product profile with
improved specificity for therapeutic protein delivery, over wild type
vectors, that is needed to effectively restore light sensitivity to
patients.
SIRION relies on 10 years of experience in viral vector engineering and
leverages close ties to leading academics in Europe that will play a pivotal
role in this project. World renowned experts in AAV biology, Prof. Grimm
from the Unversitätsklinikum Heidelberg and Prof. Büning of the Medizinische
Hochschule Hannover as well as PD Dr. Michalakis from the
Ludwig-Maximilians-Universität München will contribute their individual
expertise to this program.
"The academic acumen of our partners, together with our strong viral vector
specialization and experience will empower our client to enter clinical
trials with an efficient, safe and scalable product. For us it is the chance
to participate in a fundamental step for gene therapies that can help
millions of patients worldwide win back their eyesight. This is what we
started SIRION Biotech for," said Dr. Christian Thirion, founder and CEO of
the company.
Additionally, Dr. Ryo Kubota, MD, PhD, Chairman, President and CEO of
Acucela stated, "We are pleased to advance our optogenetic genetherapy
program in partnership with SIRION Biotech to bring a new therapeutic
approach to RP patients suffering from this devastating disease."
(1) Genetics Home Reference, retinitis pigmentosa.
https://ghr.nlm.nih.gov/condition/retinitis-pigmentosa. Retrieved Nov 7,
2016.
(2) Vaidya P, Vaidaya A. Retinitis Pigmentosa: Disease Encumbrance in the
Eurozone. Int J Ophthalmol Clin Res. 2:030 (2015).
(3) National Human Genome Research Institute. Leaning About Retinitis
Pigmentosa. https://www.genome.gov/13514348/. Retrieved Nov 7, 2016.
*About SIRION Biotech GmbH*
SIRION Biotech provides custom engineering services of viral vector
strategies for research and development in the life sciences and industry.
The Germany-based technology expert provides small to large-scale projects
to customers worldwide, over 200 a year. SIRION Biotech's unique focus on
gene delivery is why it is the only company able to master customization of
all three major virus systems that are used regularly for therapeutic and
research purposes, to genetically modify mammalian cells. The company has a
strong customer base in cancer research, neurosciences, regenerative
medicines, gene therapy and immuno-oncology. www.sirion-biotech.com [1]
*About Acucela Inc.*
Acucela Inc., is a wholly-owned subsidiary of Kubota Pharmaceutical Holdings
Co., Ltd. (Tokyo 4596) committed to translating innovation into a diverse
portfolio of drugs and devices to preserve and restore vision for millions
of people worldwide. Acucela's development pipeline include drug candidates
and therapeutics for the treatment of retinitis pigmentosa, proliferative
diabetic retinopathy, diabetic macular edema, AMD, Stargardt disease,
cataracts and presbyopia. The company is also developing a monitoring device
for neovascular retinal diseases, to be used directly by patients.
http://www.acucela.com [2]; http://www.kubotaholdings.co.jp/en/ [3]
*Cautionary Statements*
Certain statements contained in this press release are forward-looking
statements within the meaning of Section 27A of the Securities Act of 1933
and Section 21E of the Securities Exchange Act of 1934 and the Private
Securities Litigation Reform Act of 1995. Any statements contained in this
press release that are not statements of historical fact may be deemed to be
forward-looking statements. These forward-looking statements include
statements regarding our expectations related to our development plans and
ability to successfully develop and commercialize our product candidates and
the potential efficacy, future development plans and commercial potential of
our product candidates. These statements are based on current assumptions
that involve risks, uncertainties and other factors that could cause the
actual results, events or developments to differ materially from those
expressed or implied by such forward-looking statements. These risks and
uncertainties, many of which are beyond our control, include, but are not
limited to: our investigational product candidates may not demonstrate the
expected safety and efficacy; our pre-clinical development efforts may not
yield additional product candidates; any of our or our collaborators'
product candidates may fail in development, may not receive required
regulatory approvals, or may be delayed to a point where they are not
commercially viable; our clinical trials could be delayed; new developments
in the intensely competitive ophthalmic pharmaceutical market may require
changes in our clinical trial plans or limit the potential benefits of our
investigational product candidates; the impact of expanded product
development and clinical activities on operating expenses; adverse
conditions in the general domestic and global economic markets; as well as
the other risks identified in our filings with the Securities and Exchange
Commission. These forward-looking statements speak only as of the date
hereof and we assume no obligation to update these forward-looking
statements, and readers are cautioned not to place undue reliance on such
forward-looking statements. For a detailed discussion of the foregoing risks
and other risk factors, please refer to our filings with the Securities and
Exchange Commission, which are available on Kubota Pharmaceutical Holdings
(Acucela's parent company) investor relations website
(http://www.kubotaholdings.co.jp/en/ir/ [4]) and on the SEC's website
(http://www.sec.gov [5]).
"Acucela" , the Acucela logo and "Kubota" are registered trademarks or
trademarks of Acucela Inc. or Kubota Pharmaceutical Holdings Co., Ltd. in
various jurisdictions.
*Contact SIRION:*
SIRION Biotech GmbH
Dr. Christian Thirion
Founder and CEO
Phone: +49-89-700 961 99-15
Email: thirion@sirion-biotech.de
*Media Contact SIRION:*
MC Services AG
Katja Arnold, Jessica Breu
Phone: +49-89-210 228-0
Email: sirionbiotech@mc-services.eu
*Investor Relations Contact Acucela:*
Acucela Inc.
John Gebhart
Chief Financial Officer
Phone: +1-206-805-3972
Email: jgebhart@acucela.com
*Media Contact Acucela:*
Acucela Inc.
Michael Hasegawa
Senior Director, Corporate Communications
Phone: +81-3-6550-8928
Email: mhasegawa@acucela.com
End of Media Release
Issuer: Sirion Biotech GmbH
Key word(s): Research/Technology
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(END) Dow Jones Newswires
January 08, 2018 14:00 ET (19:00 GMT)
