BioTime reported several developments for its ophthalmology portfolio, including:
- Signing a new and expanded licensing agreement with Hadassah Medical Organization of Jerusalem, Israel
- Presentation of data from its Phase 1/2a Dose Escalation Study of OpRegen at ISSCR
- Presentation of the similarities between human retinal tissue and 3D retinal tissue derived using BioTime’s proprietary methodology and pluripotent cells at ISSCR
- Publication of preclinical data forming the foundation of the OpRegen IND submission with the U.S. Food & Drug Administration in TVST
- Complete control of OpRegen development program through consolidation of ownership in Cell Cure NeuroSciences
“OpRegen is making great progress in the clinic,” Adi Mohanty, co-CEO of BioTime, said in a company news release. “Data from the phase 1/2a trial was presented this past weekend at the International Society of Stem Cell Research (ISSCR) conference. As OpRegen continues to show encouraging results, we are leveraging our expertise in ophthalmology to build a leading pipeline of cell therapy products in ophthalmology. The technology for OpRegen for dry AMD was originally licensed from Hadassah. The new license increases our field of use for RPE cells to all eye disorders, and adds photoreceptor cells, for all eye disorders. As part of this process, we have consolidated our ownership of Cell Cure Neurosciences providing BioTime with complete control of the OpRegen development program while at the same time building momentum in developing innovative cell therapies to address ophthalmic conditions with unmet needs."
A poster demonstrating similarities between normal human retinal tissue and laboratory grown 3D retinal tissue derived using BioTime’s proprietary methodology with pluripotent cells (hPSC) was presented during ISSCR. The study compared normal human retinal cells with the BioTime’s proprietary hPSC-derived 3D retinal tissue for the potential use in helping to restore vision for blind people with advanced stages of retinal degeneration. The results presented demonstrated high correlation in gene expression profiles, between the various cellular components of normal human retinal tissue and the hPSC-derived tissue, including retinal pigment epithelium, retinal progenitor, photoreceptor, amacrine and ganglion cells.
In addition, the most recent data from the ongoing phase 1/2 clinical trial of the lead product, OpRegen, for the treatment of dry age-related macular degeneration was presented during the conference. The phase 1/2a dose-escalation study evaluating the safety and efficacy of three different dose regimens of the company’s investigational product, OpRegen. OpRegen has received Fast Track designation from the FDA for treatment of the advanced form of dry-AMD. Details of the trial and about a patient’s eligibility are available at https://clinicaltrials.gov/ with the following Identifier: NCT02286089 (dry-AMD).
TVST Journal Publication
The TVST Journal article is titled “Long-Term Efficacy of GMP Grade Xeno-Free hESC-derived RPE Cells Following Transplantation” and Trevor J. McGill was the lead author. The article concluded OpRegen RPE cells survived, rescued vision function, preserved rod and cone photoreceptors long term in the Royal College of Surgeons rat. These data were key elements in support of the potential clinical utility for the use of OpRegen RPE cells for the treatment of human RPE cell disorders including AMD.
“These studies continue to leverage and expand our proprietary approaches utilizing hPSCs for a broad range of ocular diseases associated with cellular and tissue degeneration that other therapeutic approaches can’t address effectively,” Oscar Cuzzani, MD, PhD, BioTime’s Vice President of Clinical Development, said in the news release. “As ophthalmology remains among BioTime’s core areas of focus, we are uniquely positioned with our broad expertise, capabilities and IP portfolio in regenerative ophthalmology, creating a stronger pipeline to address problems of degenerative diseases of the eye.”