Spark Therapeutics and Clearside Biomedical Announce Exclusive Option to License Technology for Potentially Differentiated Delivery of Gene Therapy to the Eye
If successful, this technique 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.
"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," said
Using a proprietary microinjector, Clearside's technology delivers therapeutic agents to the retina and choroid through the SCS and without substantial diffusion to the vitreous of the eye. Clearside is currently conducting a Phase 2 clinical trial in macular edema associated with uveitis and a Phase 2 clinical trial in macular edema associated with retinal vein occlusion (RVO). In a recently concluded Phase 1/2 trial, subjects with non-infectious uveitis treated with a single suprachoroidal injection of a commercially available formulation of triamcinolone acetonide, using Clearside's proprietary microinjector, showed improvement in best corrected visual acuity ranging between one and five lines (or up to 25 letters). The procedure was generally well tolerated in this trial and no meaningful intraocular pressure (IOP) increases were observed in any subjects through the six month duration of the trial.
"Clearside is delighted to be working with Spark, a clear leader in applying gene therapy to treat blinding diseases," said
If Spark exercises its option, in return for exclusive, worldwide rights to use Clearside's microinjection technology and related intellectual property in the field of gene therapy, Spark will pay to Clearside an upfront licensing fee, development related milestones and commercial royalties on sales of Spark's products covered by the licensed technology.
Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating orphan diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, is in a fully enrolled pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark is leveraging the experience and technology utilized in the development of SPK-RPE65 to address a broad spectrum of blinding conditions, starting with the development of SPK-CHM for the potential treatment of choroideremia, currently in a Phase 1/2 clinical trial. Spark also is establishing a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative diseases, including through a global collaboration with
About Clearside Biomedical
Cautionary Note on Forward-looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Spark's and Clearside's plans with regard to future product candidates, and the expected uses and potential benefits of alternate delivery vehicles to the eye, including Clearside's microinjector. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of Spark's other risks and uncertainties, and other important factors, any of which could cause Spark's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in Spark's most recent Annual Report on Form 10-K filed with the
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Spark Therapeutics, Inc. - Stephen W. Webster, 888-772-7560, Stephen.Webster@sparktx.com; Clearside Biomedical, Inc. - Charles Deignan, 678-270-4005, firstname.lastname@example.org