SiSaf Establishes Strategic Partnership with Avellino Labs
Dec. 18, 2019
SiSaf Establishes Strategic Partnership with Avellino Labs USA to Co-Develop Non-Viral, Topical Gene Therapies for Corneal Dystrophy
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 TGFbI 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 TGFbI protein, as well as gene therapies that utilize CRISPR gene editing technologies to permanently correct mutations in the TGFbI 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.
“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.”
SiSaf would like to acknowledge Innovate UK Knowledge Transfer Partnership award (KTP 010283) grant funding support for the early developmental work on nucleic acid formulations. The project final report was awarded an ‘Outstanding’ grade for the developmental work.
SiSaf are pioneers of silicon-based Bio-courier technology that leverages the unique properties of elemental silicon (14Si) to overcome the limitations of current drug technologies. Built upon a decade of research and patented technological innovation, SiSaf’s platform is changing the way therapeutic molecules target disease helping to make tomorrow’s precision medicines a reality. SiSaf is a commercial stage biopharmaceutical company headquartered in Guilford, UK, with fully integrated state-of-the-art research labs, manufacturing and bio-analytical facilities to fast track development. To learn more, please visit www.sisaf.com
SiSaf’s first-to-market Bio-Courier platform technology leverages the unique properties of bioabsorbable silicon, lipids and amino acids to address the significant challenges of stability, solubility, targeting and controlled release of complex therapeutic molecules.
Its customizable design and fabrication provide unparalleled flexibility with respect to therapeutic molecule type and target cell location. Bio-Couriers degrade to the bioavailable form of silicon, eliminating the accumulation of potentially toxic or immune-activating material and supporting cell health. Cost-effective, scaled manufacturing provides unequaled capacity to address both rare diseases and large patient populations.
About Avellino Labs USA, Inc.
Avellino Labs USA, Inc. is a global leader in gene therapy and molecular diagnostics and is at the forefront of precision medicine for eye care. The company is pioneering CRISPR gene editing to manage and potentially cure inherited diseases. Avellino continues to expand its diagnostics capabilities with the recent launch of AvaGen, the world’s first DNA test to confirm the presence of genetic indicators that are positively associated with corneal dystrophies and keratoconus genetic risk factors, providing life-changing information for patient treatment decisions and follow-up care. Avellino is headquartered in Silicon Valley, California, with operations in Korea, Japan, China, and the UK. To learn more, please visit https://www.avellino.com/en/
Glenn Silver, Lazar FINN Partners
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