|View printer-friendly version|
“We are excited to be evaluating the
The primary objective of the Phase I/IIa study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events (AEs). Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additionally, for the patients in Cohort 4 that receive subretinal delivery of OpRegen utilizing the Orbit SDS, objectives will include the evaluation of the safety of delivery of OpRegen using the Orbit SDS.
About the Phase I/IIa Clinical Study
This is a Phase I/IIa open-label, dose escalation safety and efficacy study of human embryonic stem cell-derived retinal pigment epithelium cells transplanted subretinally in patients with advanced dry age-related macular degeneration with geographic atrophy. The study will enrollapproximately 24 subjects, divided into 4 cohorts. The first 2 cohorts, each consisting of 3 legally blind subjects with best corrected visual acuity (BCVA) of 20/200 or less, received a single subretinal implantation of OpRegen. The third cohort included 6 subjects with BCVA of 20/200 or less, who received a single subretinal implantation of OpRegen. Staggered intervals within and between cohorts are applied to ensure subject safety and welfare. The fourth cohort will include approximately 12 subjects with BCVA between 20/64 and 20/250, who will receive a single subretinal implantation of OpRegen. Cohort 4 includes two formulations of OpRegen; the first 3 subjects were treated with an ophthalmic Balanced Salt Solution Plus (BSS Plus) version of OpRegen. Remaining subjects will be treated with an “off-the-shelf” or “thaw and inject” (TAI) formulation. OpRegen TAI can be shipped directly to sites and used upon thawing, which removes the complications and logistics of having to use a dose preparation facility. Staggered intervals will be applied between at least the first two subjects of each delivery modality to ensure subject safety and welfare.
OpRegen is a retinal pigment epithelium (RPE) transplant therapy in Phase I/IIa development for the treatment of dry-AMD, a leading cause of adult blindness in the developed world. OpRegen consists of a suspension of RPE cells delivered subretinally as an intraocular injection. RPE cells are essential components of the back lining of the retina and function to help nourish the retina including photoreceptors. OpRegen has been granted Fast Track designation from the
About the Orbit Biomedical Subretinal Delivery System
The Orbit Biomedical Surgical Delivery System (Orbit SDS) was designed to help surgeons deliver therapies to the retina with precision and accuracy. To deliver a therapy to the retina a surgeon would traditionally perform a vitrectomy and remove the gel-like substance (the vitreous) that fills the eye. Then the therapy is injected into the eye through the retina, a process called a retinotomy. The Orbit SDS, which received 510K clearance from the
BioTime Inc. IR
Ioana C. Hone
Solebury Trout IR
Gitanjali Jain Ogawa