Retinitis pigmentosa is the most common inherited eye disease. Mutations in the gene for rhodopsin, the pigment for photoreceptor cells in the retina responsible for the perception of light, account for 15 percent of retinitis pigmentosa cases. CHOP spinout Spark Therapeutics, which has a late stage retinitis pigmentosa gene therapy treatment under development, is collaborating with Ireland-based biopharma company Genable Technologies, according to a company statement. The deal will focus on Genables lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa, called GT038. Under the deal terms, Genable will license certain manufacturing patents from Spark. Philadelphia-based Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in its development of GT038. In exchange, Spark received an initial payment and will receive subsequent milestone payments and royalties on future sales of GT038, and near-term revenue from the manufacture and supply of the product, according to the statement. In response to emailed questions a spokeswoman for Spark Therapeutics said: Generally, the company recognizes that there are a significant number of severe genetic diseases without effective treatments in therapeutic areas where it has significant expertise (for example, inherited blindness, hematologic disorders, neurodegenerative diseases), and … Continue reading →

Wednesday, March 26 12:07:25 Genable Technologies, the venture-capital-backed Dublin-based pharma company specialising in fighting blindness, has entered in to a collaborative deal with Spark in the US to find a therapy against inherited blindness. They both announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038, as well as near-term revenue from the manufacture and supply of product. "We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable's great work, and expand the number of debilitating diseases of the eye that can be addressed through gene therapy," said Jeffrey D. Marrazzo, Spark Therapeutics co-founder, president and CEO. Dr. Jason Loveridge, CEO of Genable Technologies commented, "We have chosen Spark as our partner to advise, … Continue reading →

Genable Technologies founder and director Prof Jane Farrar. Photo: Aidan Crawley Genable Technologies, a Dublin company targeting genetic diseases causing blindness, has announced a collaboration with US biopharma group Spark Therapeutics. The academics behind Genable Technologies have spent over 20 years looking at one form of inherited blindness rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP) in which a genetic mutation leads to damage of the retina and a gradual loss of sight. There is no approved drug treatment on the market for the condition, which affects an estimated 30,000 patients worldwide. In 2011, Genable raised $5 million in an exercise led by Fountain Healthcare Partners and involving existing investor Delta Partners, to develop its lead drug candidate GT038. While Genable will licence certain intellectual property from Spark under the terms of the collaboration, it will retain the full IP on GT038, the company said yesterday. The drug is expected to enter full clinical trials next year. Spark will become the exclusive manufacturer of the drug and will receive milestone payments and royalties on future sales. The collaboration with Spark provides an exciting opportunity to greatly expedite development of Genables novel therapy targeted towards RHO-adRP, said Prof Jane Farrar, founder and director, … Continue reading →