Ocular BioFactory™ Platform
Avalanche’s Ocular BioFactory™ platform is our technology for developing new product candidates to treat the cause of ophthalmic diseases and beyond by enabling patients’ own cells to express a therapeutic protein for a sustained period of time.
We use adeno-associated virus (AAV) as a vector to deliver functional genes that promote continuous production of therapeutic protein. AAVs are being widely used for gene therapy in clinical trials to test their safety, stability, and sustained protein expression. Our Ocular BioFactory™ platform produces product candidates that are designed for optimal potency and tissue specificity.
The 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.
Directed Evolution
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 tissues such as 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.
Pipeline
Using our Ocular BioFactory™ platform, we have developed a pipeline of proprietary and partnered programs in gene therapy.
- Product Candidate, Indication
- Indication
- Research
- Preclinical
- Phase 1 & 2
- WCRWorldwide Commercial Rights
- Stage
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WCR: Avalanche
AVA-101 is being developed to provide a safe and effective treatment for wet age-related macular degeneration (wAMD) that is durable and reduces the need for frequent injections. AVA-101 is comprised of the AAV2 vector, which contains a gene encoding sVEGFR-1. When administered in the eye and expressed by the host retinal cells, the sVEGFR-1 protein has been shown to inhibit the formation of new blood vessels and reduces vascular permeability by binding and blocking VEGF activity.
In Phase 1 and Phase 2a studies, AVA-101 met its primary endpoint of safety and no serious adverse events related to AVA-101 were observed. Based on these results, Avalanche has initiated additional studies to inform further development of our wAMD program.
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WCR: Avalanche
AVA-101 is also being developed for diabetic macular edema (DME) and retinal vein occlusion (RVO).
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WCR: Avalanche
AVA-201 is a next generation anti-VEGF gene therapy product candidate, which is being developed for the potential prevention and treatment of wet age-related macular degeneration (wAMD). AVA-201 delivers the same sVEGFR-1 expressing gene as AVA-101 but uses a next-generation AAV vector delivery method. AVA-201 will be administered by an intravitreal injection at a specialist’s office.
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AVA-322L Color Vision Deficiency Color Vision Deficiency PreclinicalWCR: Avalanche
AVA-322L and AVA-323M are next-generation gene therapy product candidates, which are being evaluated as treatments for color vision deficiency (CVD), commonly known as red-green color blindness. CVD is one of the most common genetic diseases for which there currently are no available treatment options, affecting over 10 million people in the US alone.1
AVA-322L carries the gene for L-opsin and is being developed for the treatment of protan defects.
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AVA-323M Color Vision Deficiency Color Vision Deficiency PreclinicalWCR: Avalanche
AVA-322L and AVA-323M are next-generation gene therapy product candidates, which are being evaluated as treatments for color vision deficiency (CVD), commonly known as red-green color blindness. CVD is one of the most common genetic diseases for which there currently are no available treatment options, affecting over 10 million people in the US alone.1
AVA-323M carries the gene for M-opsin and is being developed for the treatment of deutan defects.
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WCR: Regeneron*
AVA-311 is being developed for the treatment of Juvenile X-linked Retinoschisis (XLRS), an inherited retinal disease caused by mutations in the RS1 gene located on the X chromosome, therefore occurring almost exclusively in males. AVA-311 is comprised of an optimized AAV vector to deliver the RS1 gene into the eye via intravitreal injection.
In May 2014, Avalanche signed a collaboration agreement with Regeneron that includes the development of AVA-311.
- WCR - Worldwide Commercial Rights
- PH - Phase
Manufacturing
Our seasoned pharmaceutical development team has decades of gene therapy manufacturing experience. Avalanche’s industrialized manufacturing process—based on our proprietary baculovirus expression system—is highly efficient and scalable, with production yields up to one hundred times greater than those obtained using conventional AAV production systems.
Our process enables us to manufacture commercial grade material for diseases with large patient populations. We have performed multiple single use bioreactor production / purification runs and have the ability to make highly concentrated purified vector.
Collaborations
Avalanche collaborates with leading academic institutions and pharmaceutical companies who can augment our industry-leading expertise in gene therapy.
Please contact us for more information.
References:
- Sharpe LT, Stockman A, Jagle H, Nathans J. Opsin genes, photopigments, color vision and color blindness. In: Gegenfurtner KR, Sharpe LT (eds.) Color Vision. Cambridge UP: Cambridge, 1999.