Spark Therapeutics, Inc. (NASDAQ: ONCE) and Clearside Biomedical, Inc. (Alpharetta, Georgia, USA) have announced the execution of an option agreement to facilitate a potential acquisition by Spark of exclusive rights to license Clearside’s microinjector technology. The technology may be used for the delivery of gene therapies to the back of the eye. Established in January 2012, Clearside focuses on targeted drug delivery through a proprietary microinjection platform that allows compartmentalization of a pharmaceutical ingredient within a specific area of the eye. According to a press release from Spark, the Clearside technoogy “could create a differentiated alternative to sub-retinal or intravenous administration for future gene therapy applications, potentially broadening the range of conditions treatable through gene therapy”.
Clearside’s proprietary platform was developed from an academic collaboration between the Georgia Institute of Technology and Emory University School of Medicine. The technology developed from the work of Mark Prausnitz, Ph.D., Regents’ Professor and Love Family Professor of Chemical & Biomolecular Engineering at the Georgia Institute and Henry Edelhauser, Ph.D., Professor in the Department of Ophthalmology at Emory University School of Medicine. The targeted delivery approach is focused on the use of a microneedle to deliver agents to the supra-choroidal space (SCS) between the choroid and the sclera for the purpose of more efficiently reaching tissues impacted by chorioretinal disease. The company. announced a favorable end-of-Phase II review with the U.S. FDA regarding a clinical study entitled, an “Open-Label, Safety and Tolerability Study of Suprachoroidal Triamcinolone Acetonide Via Microneedle in Subjects With Non-Infectious Uveitis”. According to Clearside, the company has reached agreement with the FDA on an overall development plan for a single pivotal Phase III clinical trial for the microinjection delivery of triamcinolone.
Under the Spark Therapeutics / Clearside Biomedical Inc. option agreement, the companies will test the feasibility of using the microinjector technology to deliver viral vectors to the choroid, and to the retina, via the suprachoroidal space(SCS). Commenting on the deal, Jeffrey D. Marrazzo, CEO of Spark stated, “We believe gene therapy has tremendous potential to provide one-time, life-altering treatments to patients with debilitating, monogenic blinding conditions, and development of an alternative delivery approach that broadly covers the retina would be an important step in further unlocking this potential. As we build a pipeline of gene therapies to treat inherited retinal dystrophies, we believe Clearside’s proprietary technology and intellectual property estate for delivery of therapeutics to the SCS may allow us to target new diseases.” It is understood that if the companies convert the option into an exclusive licensing deal, Spark will obtain exclusive, worldwide rights to use Clearside’s microinjection technology and related intellectual property in the field of gene therapy, in return for which, Spark will pay an upfront licensing fee, development related milestones and commercial royalties on sales of Spark’s products covered by the licensed technology.