Products in the pipeline – Cell Cure’s OpRegen

by Alexey Bersenev on December 10, 2014 · 1 comment

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This is third post of the series Products in the Pipeline. In this series we (community!) will provide publicly available information about therapeutic products – candidates for commercialization by cell therapy industry companies.
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Prepared by m.cea for StemCellAssays.com, narrowed by Alexey Bersenev
(updated: 12/15/2014)

Product candidate name: OpRegen®
Developer: Cell Cure Neurosciences Ltd. of Israel (a subsidiary of BioTime, Inc. with Teva Pharmaceutical Industries, Ltd. and HBL-Hadasit Bio-Holdings, Ltd. as minority shareholder partners)
History of development: Since 2005 by Cell Cure Neurosciences Ltd. (a ES Cell International Pte Ltd) acquired by BioTime, Inc. in 2010
Type of cells: Human allogeneic embryonic stem cell-derived retinal pigment epithelium (RPE), expanded ex vivo.
Tissue source: Inner cell mass of blastocyst, derived from fertilized oocytes. Pre-embryo donated as supernumary tissue to science rather than discarding during standard IVF treatment. hESC cell line established – HAD-C 102.
Processing steps: Laser blastocyst ZP breaching, mechanical dissection, isolated ICM plated, ES culture to embryoid body development on human foreskin fibroblasts feeder layers in KO medium, ES cells isloation, hESC master cell bank, further culturing steps w/ nicotinamide and TGF-β Growth Factors (Activin A) until RPE cells, plating, expansion, working cell bank, expansion, harvest, cryopreservation.
Phenotypic composition: positive for MiTF-A, RPE65, Bestrophin, Mertk, Otx2, ZO-1, CD81 and CRALBP
Stability/Safety: Genetically stable, normal karyotype, normal senescence & terminally differentiated to its RPE fate
Proposed mechanisms of action: Cell replacement to restore cell cycle action to support photoreceptor.
Scientific background: RPE cell replacement therapy is a potential regenerative treatment which looks to restore lost retina cells at the base of the macula to establish a new and healthy support layer to the photoreceptors which are loosing function as a result of the old RPE layer dying off. It is envisioned that by replacing the lost RPE layer the remaining photoreceptors will once again function properly thereby either arresting the decline of the age related condition or actually improve lost visual acuity.
Key publications:
PLoS One. 2012;7(6):e35325. doi: 10.1371/journal.pone.0035325
Cell Stem Cell. 2009 Oct 2;5(4):396-408. doi: 10.1016/j.stem.2009.07.002
Ongoing trials: Phase 1 – Dry AMD – Geographic Atrophy – NCT02286089
Administration: Vitrectomy with sub-retinal injection of cell suspension with dosing from 50,000 to 500,000 cells
Key patents:
hESC Culture System – WO2008120218 & US Patent 8,597,947 & US Patent App 13/357948 (Notice of Allowance issued)
Stem Cell Derived RPE Cells – WO2008129554 & US Patent App 12/450943 & Espacenet Family
Method of Selecting RPE cells – WO2013114360
IP Positioning summary: Cell Cure’s underlying US Patent on Pluripotent Culture System methods (hESCs from ICM Blastocyst stage) employs a propriatary xeno-free feeder system and is therefore a valuable addition to the state-of-the-art science to maintain Pluripotent embryonic cells for further derivation into somatic cell lineages. Biotime has a leading position in hESC Blastocyst IP as a result of Geron’s IP assets in hESC science which Biotime acquired. In addition it should be noted that the process of extracting hESCs from Blastocyst ICM stage embryos results in their termination – a point that restricts the IP due to European objection to destructive methods of embryonic cell extraction.
The RPE derivation methods defined by Cell Cure have been issued Patents in various International Territories but are not, as of writing, protected in the US due to various USPTO Examination issues, including prior disclosure of the use of Nicotinamide in the above cell culture patent literature and Ocata’s lead in issued patents & prior art in RPE IP.
In relation to the sector competition please see the following link for an overview: BTX Hadasit.

{ 1 comment… read it below or add one }

Ross Macdonald December 11, 2014 at 8:40 pm

Great information from Stem Cell Assays: keep it up!

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