Adverum Biotechnologies Presents Preclinical Data on Novel Gene Therapy Candidates for the Treatment of Wet AMD at The Retina Society 2016 Annual Meeting
Using the industry standard, laser-induced choroidal neovascularization (CNV) model in a non-human primate study, Adverum demonstrated that a single intravitreal administration of ADVM-022 or ADVM-032 each had comparable efficacy in reducing grade IV CNV lesions to an intravitreal injection of standard-of-care anti-VEGF proteins. To date additional pharmacokinetic studies of the vitreous and retinal tissue have shown durable anti-VEGF protein expression with therapeutic protein levels at least 20 weeks after a single intravitreal administration. Observations at 26 weeks show a favorable safety profile. Minimal vitreous inflammation not requiring steroid treatment was noted four to six weeks after injection but was resolved by week eight and did not recur. No retinal structural changes or consequences of inflammation were detected on optical coherence tomography (OCT) at 12 weeks after injection.
“We are proud of our research and vector optimization capabilities. These capabilities have allowed us to demonstrate in preclinical studies that it is possible to achieve the same degree of CNV inhibition using an intravitreally delivered novel vector with an optimized AAV capsid, expression cassette, and anti-VEGF cDNA as the standard-of-care intravitreally delivered anti-VEGF proteins on the market today,” said
Adverum is a gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline that includes product candidates to treat wet AMD, A1AT deficiency, and hereditary angioedema, among other diseases. We are leveraging our next-generation adeno-associated virus (AAV)-based directed evolution platform to generate product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our focus on the patient is supported by clinical development expertise and core capabilities in vector optimization, process development, manufacturing, and assay development. For more information please visit www.adverumbio.com
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