SIRION Biotech and Acucela Set Sights on Gene Delivery Tools for Ocular Gene Therapy

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DGAP-Media / 2018-01-08 / 20:00 
 
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: [phone] 
Email: [e-mail] 
 
*Media Contact SIRION:* 
MC Services AG 
Katja Arnold, Jessica Breu 
Phone: [phone] 
Email: [e-mail] 
 
*Investor Relations Contact Acucela:* 
Acucela Inc. 
John Gebhart 
Chief Financial Officer 
Phone: [phone] 
Email: [e-mail] 
 
*Media Contact Acucela:* 
Acucela Inc. 
Michael Hasegawa 
Senior Director, Corporate Communications 
Phone: [phone] 
Email: [e-mail] 
 
End of Media Release 
 
Issuer: Sirion Biotech GmbH 
Key word(s): Research/Technology 
 
2018-01-08 Dissemination of a Press Release, transmitted by DGAP - a service 
of EQS Group AG. 
The issuer is solely responsible for the content of this announcement. 
 
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643703 2018-01-08 
 
 
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January 08, 2018 14:00 ET (19:00 GMT)