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Sustained Therapeutic delivery for the eye

The Ocular BioFactoryTM

Avalanche's Ocular BioFactoryTM platform is designed to treat the cause of ophthalmic diseases by enabling patients' own cells to express a therapeutic protein for a sustained period of time. We use an adeno-associated virus (AAV) as a vector to deliver and express, or transduce, a functional gene to the cells of the eye to promote continuous protein production. Although AAVs are widely used for gene therapy due to their safety, stability and sustained protein expression, our Ocular BioFactoryTM platform produces product candidates that are designed to have distinct mechanisms of action from other gene therapy technologies using AAVs as well as other viral and non-viral vectors.

Our Ocular BioFactory platform features two key proprietary components: a novel vector screening and optimization system referred to as directed evolution, and an industrialized manufacturing process. Through directed evolution, we generate a diverse library of millions of AAV variants and subsequently screen the variants in multiple in vitro and in vivo tests to identify the optimal variant for a specific disease. Our directed evolution technology allows us to create proprietary vectors and optimize them to target cells in different layers of the retina. Each of these cell layers constitutes a potential therapeutic target for currently unmet medical needs, providing us with multiple opportunities to apply our directed evolution technology. Our industrialized manufacturing process, based on our proprietary baculovirus expression system, is highly efficient and scalable. Production yields are up to one hundred times greater than those obtained using conventional AAV production systems. Therefore, we are able to manufacture commercial grade production for diseases with large patient populations.

The results of a Phase 1, 52-week trial of Avalanche Biotechnologies’ rAAV.sFLT-1 (AVA-101) gene therapy for the treatment of wet age-related macular degeneration (wAMD) have been published online by The Lancet. The study sought to evaluate the safety of rAAV.sFLT-1 via a single subretinal injection. Results of the study demonstrate that rAAV.sFLT-1 was safe and well tolerated, and support ocular gene therapy as a potential long-term treatment option for wAMD.

“The use of gene therapy to reprogramme genetically normal cells of the retina to take on additional functions opens a new chapter in potential AAV applications.”
– Dr. Robert MacLaren, Moorsfield Eye Hospital

The complete article and editorial commentary by Dr. MacLaren, are available from The Lancet.

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