Usher Syndrome Gene Therapy Safe Thus Far In Three Patients
Nov. 20, 2012 – Oxford BioMedica, a leading gene therapy development company in the United Kingdom, has received a positive interim safety review of its ongoing Phase I/IIa clinical trial for the UshStat® gene therapy for people with Usher syndrome type 1B. The first three patients experienced no adverse events for up to six months after treatment. The positive review by the study’s Data Safety Monitoring Board, an independent panel of ophthalmology and gene therapy experts, enables the company to treat additional patients at a higher dose level.
Oxford BioMedica also reported that its StarGen™ gene therapy for Stargardt disease and RetinoStat® gene therapy for wet age-related macular degeneration continue to perform well in human studies.
The Foundation Fighting Blindness funded critical laboratory research that made all three gene therapy clinical trials possible.
Eight patients — four with severe levels of disease and four with less severe levels — have experienced no serious adverse events for up to 16 months after treatment in the Phase I/IIa StarGen clinical trial. An additional four patients are now being treated with a higher dose.
In the Phase I RetinoStat clinical trial, nine patients have been treated at three dose levels, and an additional nine are now being treated at the target dose level. The therapy has been safe for up to 19 months. The company also reports that higher doses of RetinoStat are resulting in higher levels of the therapeutic protein in the patients’ eyes.
A major benefit of gene therapy is that a single treatment, administered through an ocular injection, may halt vision loss for several years or perhaps a lifetime.
Sanofi, an international pharmaceutical company, is partnering with Oxford BioMedica in the development of the StarGen and UshStat gene therapy products.
11月20日，2012 - 牛津BIOMEDICA ，公司在英国领先的基因治疗的发展，已获得肯定的临时安全审查其正在进行的第一阶段I / IIa期临床试验的UshStat ®基因治疗的人与Usher综合征1B型。前三个患者经历到治疗6个月后，没有不良反应事件。的积极检讨这项研究的数据安全监测委员会，一个独立的面板眼科和基因治疗专家，使公司，以治疗更多的患者在一个较高的剂量水平。
牛津BIOMEDICA也报道，其StarGen ™基因治疗Stargardt病病和RetinoStat ®基因疗法治疗湿性年龄相关性黄斑变性的继续表现良好，在人类研究中。基金会资助的战斗失明八的病人- 4个用更少的严重程度与疾病的严重程度和至关重要的实验室研究，基因治疗的临床试验可能。-已经历了长达16个月无严重不良事件，在第一阶段的治疗后我/第IIa StarGen的临床试验。4位病人正在被用一个较高的剂量，在I期RetinoStat临床试验，9的患者已在三个剂量水平处理，和一个额外的9个正被处理的目标剂量水平。长达19个月的治疗已安全。该公司还报告称，高剂量的RetinoStat导致更高水平的治疗性蛋白在病人的眼睛。基因治疗的一大好处是，一个单一的治疗，给药通过眼部注射液中，可数年或阻止视力减退，也许是一辈子。，一家国际制药公司赛诺菲，与牛津大学BIOMEDICA合作的StarGen和UshStat基因治疗产品的发展。 |
