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Regeneron, Avalanche to Partner on Eye Disorder Gene Therapy

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5 May 2014. Regeneron Pharmaceuticals in Tarrytown, New York is licensing gene therapy technology from Avalanche  Biotechnologies in Menlo Park, California to develop and commercialize new treatments for eye diseases. The value of the deal is at least $640 million, but not all payment amounts were disclosed.

Regeneron is a biopharmaceutical company developing therapies for inflammation, cardiovascular and metabolic disorders, cancer, and pain. The company is also devising a treatment for eye diseases involving the growth of abnormal blood vessels and leakage behind the macula, the part of the retina that governs central vision. That therapy, under an agreement with Bayer, is an injection that controls production vascular endothelial growth factor-A, a protein that stimulates growth of blood vessels, but excessive production of the protein is a key factor in diseases of the retina.

Avalanche’s technology delivers therapeutics with adeno-associated viruses, benign (non-disease causing) viruses carrying genes encoded with a protein that treats age-related macular degeneration, a common condition and the leading cause of vision loss for people age 50 and over. The company says adeno-associated viruses can treat a variety of cells in the retina and remain stable, providing long-term expression of the proteins being delivered.

Stability of the proteins, says Avalanche, makes it possible to deliver the therapeutics over an extended period of time, at least 18 months based on preclinical studies. The company has a clinical trial underway in Australia of 40 patients with age-related macular degeneration testing various doses of its gene therapy against ranibizumab, a standard-of-care medication, given by injection into the retina about once a month. The trial is looking primarily at the safety of the treatments, but also reporting any improvements in vision among the patients.

The agreement covers up to eight therapy targets, with Regeneron gaining exclusive worldwide rights for each product it advances into clinical trials. Avalanche will receive an undisclosed upfront payment, plus up to $640 million payments keyed to certain development and regulatory milestones. In addition, Avalanche will qualify for royalties on net sales of products developed under the collaboration.

The deal also gives Avalanche an option of selecting two therapy targets, and share with Regeneron both development costs and targets. Regeneron receives as well under the deal a time-limited right of first negotiation to Avalanche’s gene therapy product to treat age-related macular degeneration that targets  vascular endothelial growth factor.

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