基因治疗青少年黄斑变性的实验预计今年开始
Gene Therapy Clinical Trial for Stargardt Disease to Begin This Year
For patients affected with Stargardt disease, there is new reason for hope. Oxford BioMedica, a gene therapy company in the U.K., has received FDA authorization to launch a clinical trial of its StarGen™ gene therapy for the treatment of Stargardt disease.
Up to twenty-eight people will be enrolled in the Phase I/IIa clinical trial to be conducted at Oregon Health and Science University in Portland under the leadership of Peter Francis, M.D., Ph.D. and in France. The United States-based trial is slated to begin this summer.
The third gene therapy treatment for a retinal degenerative disease to move into the human studies, the StarGen™ treatment will follow similar methodology as the ongoing gene therapy treatment trials for Leber congenital amaurosis that have resulted in restored vision for children and young adults who were given just one “dose” of the therapy. By piggybacking a corrected gene onto a virus – the taxicab that will deliver the gene - doctors will replace the disease-causing faulty gene in the retina with the corrected version. On the basis of pre-clinical data, it is anticipated that a single application of StarGen™ to the retina could provide long-term or potentially permanent correction.
“As an initial funder of the project we are pleased to see the StarGen™ clinical trial moving forward and reinforcing the exciting potential for gene therapy to treat patients with Stargardt disease and other vision-robbing retinal degenerations,” says Foundation Fighting Blindness Chief Research Officer Dr. Stephen Rose. “The Foundation is committed to accelerating the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like Stargardt to patients who need them. Our relationship with Oxford BioMedica is an excellent example of this mission in action.”
Stargardt disease is the most common juvenile degenerative retinal disease. The disease is caused by a mutation of the ABCR gene which leads to the degeneration of photoreceptors in the retina and vision loss. StarGen™ uses Oxford's LentiVector® platform technology to deliver a corrected version of the ABCR gene.
In addition to StarGen™, the Foundation Fighting Blindness has been partnering with Oxford BioMedica in its development of UshStat™ gene therapy for the treatment of Usher syndrome 1b. This clinical trial is scheduled to begin later this year. The Foundation also supported Oxford in earlier preclinical studies of RetinoStat®, a gene therapy treatment for wet age-related macular degeneration currently enrolling participants in a Phase I clinical trial at The Wilmer Eye Institute at Johns Hopkins.
The Foundation will provide updates as this trial progresses and recruitment begins. For additional information on Oxford BioMedica and their StarGen™ treatment, their full release is available. |
