2011年2月8日爱尔兰电，欧洲药监局已经批准爱尔兰著名的都柏林三一学院附属的Genable科技有限公司的一种基因药物治疗RP的孤儿药资格。
具体药物的治疗原理和治疗范围我不是看的很懂，有可能是所有显性遗传的RP患者，大家看看吧，还有获得了孤儿药资格以后人体实验会很快开始的，还是很令人期待的。
Genable Technologies – a gene based medicines platform
Genable Technologies Ltd. is a privately held, venture backed, Dublin (Ireland) based bio-pharmaceutical company developing new gene medicines to treat "dominant" genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries.
Genable utilizes well-established, clinically safe & effective AAV vectors to obtain expression of RNA interference (RNAi) molecules which suppress the expression of both the faulty and normal gene copies and replaces this with a gene subtly altered to become refractory to suppression but still encoding a normal wild type protein.
The combination of suppression and replacement (S&R) overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations in a wide range of disorders. Genable's technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.
Genable's first gene medicine - GT038 - is for treatment of patients with rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP) - a debilitating form of inherited blindness resulting from a diverse array of mutations in the RHO gene.
This sub-type of adRP affects approximately 1 in 30,000 people and represents an already identified and potentially treatable population of around 30,000 patients in the US and Europe. Whilst we estimate the market opportunity for GT038 in this sub-type of adRP to be over $500m, adRP collectively affects approximately 1 in 12,000 people.
Genable will employ the same approach to develop other gene medicines for a number of other sub-types of adRP. The company has applied for Orphan Drug Status for GT038.
Most importantly, Genable has established robust proof of principle for its approach in a mouse model of RHO-adRP where GT038 is delivered to the eye by local (subretinal) injection. |
