本帖最后由 凤凰涅盘 于 2012-7-1 11:22 编辑
June 29, 2012 – The clinical development of two promising retinal disease gene therapies — StarGen™ for Stargardt disease and UshStat® for Usher syndrome type 1B — has received a major boost. Sanofi, a large international pharmaceutical company, has licensed the treatments from Oxford BioMedica, a leading gene-therapy company.
Under the terms of the agreement, Oxford BioMedica will receive an initial $3 million payment from Sanofi, and will be eligible for future royalty and milestone development payments. The companies will continue to work together to move the treatments through human studies and out to the marketplace.
“We are very excited to see a major pharmaceutical company make such a strong commitment to gene therapies for inherited retinal diseases,” says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. “Sanofi’s investment demonstrates the company’s confidence in the potential for these treatments to save vision, and at the same time, it reinforces the commitment to getting the therapies through the clinical development process and out to the people who need them.”
StarGen is currently in a Phase I/II clinical trial at Oregon Health & Science University (OHSU) and the Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts Institut in Paris. UshStat is also in a Phase I/II human study at OHSU. The Foundation Fighting Blindness funded critical laboratory studies that made these human trials possible.
The treatments work by delivering copies of normal genes to the retina, replacing the defective, disease-causing genes. Researchers believe that a single treatment can halt disease for several years, perhaps a lifetime.
“This licensing agreement validates the Foundation’s mission in an unprecedented fashion,” says Dr. Rose. “Our passionate founders established the Foundation more than 40 years ago, because essentially nothing was known about inherited retinal degenerative diseases, and no one in the commercial sector was investigating potential treatments. But now, thanks to the Foundation’s sustained commitment to driving groundbreaking research, we’re on industry’s radar screen in a big way. It is an exciting milestone for the Foundation and the millions who need sight-saving treatments.”
6月29日，2012 - -两个前途的视网膜疾病基因疗法的临床开发StarGen™ Stargardt病UshStat的® Usher综合征1B型-已收到了重大的推动。一个大型国际制药公司，赛诺菲，已授权的治疗，从牛津大学BioMedica，领先的基因疗法的公司。牛津BioMedica根据该协议的条款，将收到来自赛诺菲最初的3万元的款项，将是未来的特许权使用费和里程碑发展支付的资格。该公司将继续共同努力，将通过人类研究和市场的处理。“我们非常高兴地看到，一家大型制药公司这样一个强有力的承诺，为遗传性视网膜疾病基因疗法斯蒂芬博士说，”玫瑰首席研究人员，基金会战斗失明。“赛诺菲的投资表明该公司的这些治疗潜力的信心，挽救视力，并在同一时间，它增强了获得通过临床开发过程的治疗和谁需要它们的人的承诺。” StarGen是目前在阶段I / II期临床试验在俄勒冈卫生科学大学（OHSU的）和中心医院NATIONALE D'Ophthalmologie DES Quinze Vingts研究所在巴黎。UshStat也是在第一阶段I / II型人类在OHSU研究。资助该基金会战斗失明关键的实验室研究，使这些人体试验成为可能。提供正常基因的副本到视网膜，更换有缺陷的，致病基因的治疗工作。罗斯博士说：“ 研究人员认为，一个单一的治疗可以阻止疾病好几年了，也许是一辈子。“此许可协议，以前所未有的方式证实了该基金会的使命。“我们的热情创始人基金会成立超过40年前，因为基本上没有已知的遗传性视网膜变性疾病，没有一个在商界被调查的潜在治疗。但现在，由于基金会的持续承诺，推动开创性的研究，我们对行业的一个大的雷达屏幕上。这是一个激动人心的里程碑为基础和以百万计的需要视力节省治疗。“ |
