Pharma Focus Asia

SiSaf Establishes Strategic Partnership with Avellino Labs USA to Co-Develop Non-Viral, Topical Gene Therapies for Corneal Dystrophy

Thursday, December 19, 2019

SiSaf Ltd, pioneers of Bio-Courier® technology, today announced it has entered into a strategic collaboration with Avellino Labs USA, Inc., a leader in precision medicine, to co-develop topical gene therapies for Corneal Dystrophies resulting from mutations in the TGFβI gene. The collaboration brings together Avellino’s expertise in gene-based diagnostics and therapeutics for eye diseases and SiSaf’s groundbreaking non-viral delivery technology.

Corneal dystrophies are a group of inherited eye disorders, often progressive, in which abnormal material can accumulate in the transparent outer layer of the eye, the cornea, causing significant pain, sensitivity to light and visual impairment. Currently available treatments only address the symptoms of the dystrophy and none are capable of halting its progression, with severe cases requiring a corneal transplant.

Under the terms of the agreement, SiSaf Ltd and Avellino Labs USA, Inc., will co-develop topical non-viral gene therapies that deliver small interfering RNA (siRNA) molecules to the cornea to periodically block the production of mutant TGFβI protein, as well as gene therapies that utilize CRISPR gene editing technologies to permanently correct mutations in the TGFβI gene at the DNA level.

While gene replacement therapy is a validated approach for treating ocular diseases, the adeno-associated virus (AAV) vector-based delivery approach used in most investigational therapies have several clinical and commercial limitations. These include a limited capacity to entrap the therapeutic cargo, the need for injection into the eye, the risk of provoking an immune response, alongside high manufacturing costs.

SiSaf’s proprietary silicon-based Bio-Courier technology overcomes these shortcomings by condensing and encapsulating high volumes of therapeutic gene modifying agents in biologically friendly silicon and lipids to allow cellular permeation and controlled release. Importantly, after delivering their payload, SiSaf’s Bio-Couriers degrade to the bioabsorbable form of silicon, an essential micromineral, eliminating the accumulation of potentially toxic or immune-activating material.

“Our mission is to advance the application of genetic technologies to deliver on the promise of precision medicine for improved healthcare outcomes for all,” said Gene Lee, Chairman and Founder of Avellino. “Our partnership with SiSaf accelerates this mission by delivering Avellino’s gene therapy reagents with the simple application of an eye drop to cure genetic eye disease. This is a breakthrough advancement that overcomes the major challenges of treating genetic diseases, advancing past more painful or complex methods of delivering therapies.”

“SiSaf has demonstrated the power of their Bio-Courier technology platform in a vast range of challenging molecules in multiple industries,” said Suzanne Saffie-Siebert, PhD, Chief Executive Officer at SiSaf. “This partnership with Avellino Labs validates the broad potential of our technology to transform gene therapy by overcoming the restrictions of current viral and non-viral delivery systems. We believe that combining our Bio-Courier technology with Avellino’s pioneering leadership in gene-based diagnostics, data, and therapy will advance our mission to unlock the full power of precision medicine to improve patient care and access.”

“Delivering large, complex therapeutic reagents to the cornea without injection is a real challenge due to the five layers of transparent, densely packed cells protecting the eye alongside regular blinking which minimizes residency time for any therapeutic agent,” said Tara Moore, PhD, Professor of Personalized Medicine at Ulster University and Chief R&D Officer at Avellino Labs USA, Inc. “In our preclinical evaluation, SiSaf’s Bio-Courier technology was the only option to successfully and safely deliver siRNA to all layers of the cornea and produce a significant reduction in gene expression using eyedrop application. This exciting partnership will combine our unique skills and expertise allowing us to further develop a similar ocular delivery strategy for RNA and protein CRISPR gene editing of genetic eye disease.”

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