Dr. Lukas Scheibler, Acucela’s EVP Of R&D, Presents Optogenetics At Cell & Gene Meeting On The Mesa

Published: Oct 06, 2017

SEATTLE--(BUSINESS WIRE)--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, announced today that Dr. Lukas Scheibler, Executive Vice President of Research & Development, presented on human rhodopsin based optogenetics technology as a treatment candidate for retinitis pigementosa (RP) at the Cell & Gene Meeting on the Mesa on October 4, 2017 in La Jolla, California, United States.

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 4,000 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 relevant genes identified(3). To date, no pharmacological treatment is available.

Dr. Scheibler presented the collaboration network Acucela has built with world-renowned experts in synthetic promoter and Adeno-associated virus (AAV) vectors optimization. The company plans to build cell specific and patent-protected vectors to allow for a minimally invasive and highly selective delivery of this potential breakthrough therapy to patients with RP. The presentation covered the goals of the technology and the company’s plans.

“We are excited to have built a novel, collaborative network of experts with the common goal to restore functional vision in blind patients,” said Dr. Scheibler.

Additionally, Dr. Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela, stated, “We are pleased to advance our optogenetic genetherapy program 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, Vaidya 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 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; http://www.kubotaholdings.co.jp/en/

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/) and on the SEC’s website (http://www.sec.gov).

“Acucela,” the Acucela logo and “Kubota” are registered trademarks or trademarks of Acucela Inc. or Kubota Pharmaceutical Holdings Co., Ltd. in various jurisdictions.

Acucela Inc.
Michael Hasegawa, +81-3-6550-8928
Senior Director, Corporate Communications
Investor Relations:
John Gebhart, +1-206-805-3972
Chief Financial Officer

Back to news