Spark Therapeutics, Inc., a gene therapy leader,  and Clearside Biomedical, Inc., a clinical-stage biopharmaceutical company, have entered into an option agreement under which Spark acquired exclusive rights to license Clearside's microinjector technology to deliver gene therapies to the back of the eye.

Under the agreement, the companies will explore the feasibility of using Clearside's microinjector technology to deliver viral vectors to the choroid and the retina through the suprachoroidal space (SCS).

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 Jeffrey D Marrazzo, co-founder and chief executive officer of Spark.

"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. If successful, the microinjector technology may provide an alternative to current routes of administration, provide ease of delivery for physicians and further expand on our leadership in the ocular gene therapy field."

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 Daniel White, chief executive officer and president of Clearside. "We believe this agreement highlights the important potential of precision delivery to propel the field of ocular gene therapy forward. By reaching ocular tissues that we would otherwise not reach, we are creating the potential to treat a range of debilitating ocular conditions."

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.

Clearside Biomedical, Inc, headquartered in Alpharetta, GA, is a clinical-stage biopharmaceutical company developing first-in-class drug therapies to treat chronic, blinding diseases of the eye.