Lineage Launches New Cell Therapy Program in Corneal Endothelial Disease
Lineage Cell Therapeutics, Inc. a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf”, cell therapies for serious medical conditions, today announced the expansion of its cell transplant pipeline with the launch of COR1, a new corneal endothelial cell (CEnC) therapy program, in preclinical development for the treatment of corneal endothelial disease. Corneal endothelial disease is a progressive, often hereditary condition where cells on the inner layer of the cornea die, causing cornea swelling and vision loss.
Applicable indications for COR1 are expected to include Fuchs Endothelial Corneal Dystrophy (FECD) and Bullous Keratopathy. Utilizing Lineage’s proprietary cell manufacturing and expansion platform, AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent cell Engineering), the Company has manufactured “off the shelf” corneal endothelial cells with identity, morphological, and functional characteristics which meet initial internal criteria and support further development. Lineage plans to advance this new product candidate into preclinical testing and will provide additional developmental updates on the COR1 program as they become available.
“Corneal endothelial disease is natural next application of our platform. Millions of people are candidates for corneal transplants for which today there is only one donor for every 70 diseased eyes globally. The current supply of CEnC’s from cadaveric sources is further limited by the low availability of organ donors, as well as by inconsistent yield and quality.
We believe we are well positioned to produce and provide a consistent and “off-the-shelf” allogeneic source of CEnC’s by applying our proprietary AlloSCOPE platform and our development expertise from the OpRegen® program to this new ophthalmology program,” stated Brian M. Culley, Lineage CEO. “Importantly, CEnC therapy from cadaveric sources has already been approved in Japan to treat corneal endothelial disease, providing evidence for the underlying mechanism of action.
Having this clinical and regulatory precedent creates a highly attractive opportunity for us to develop a more consistent and cost-effective product that could address the global shortage of donor cells. We have leveraged the capabilities of our AlloSCOPE platform and a proprietary pluripotent cell line to launch this new, internally-developed and wholly-owned initiative, and have successfully manufactured and characterized corneal endothelial cells that meet our initial attributes for identity and scale.
We have elected to advance the COR1 program into preclinical testing and we also believe the recent accomplishments reported with our AlloSCOPE “5D” manufacturing process could be applied to this program to further reduce production costs through large-scale production.”
About Corneal Endothelial Disease
Fuchs endothelial corneal dystrophy (FECD) is the most common primary corneal endothelial dystrophy and the leading indication for corneal transplantation worldwide. FECD is characterized by the progressive decline of corneal endothelial cells (CEnC’s) and the formation of extracellular matrix (ECM) excrescences in the Descemet’s membrane (DM), called guttae, that lead to corneal edema and loss of vision. FECD represents one of the largest underserved populations in ophthalmology, affecting approximately 7.33% of adults over 30 years of age globally, with the patient population projected to rise from approximately 300 million in 2020 to 415 million by 2050.
The current addressable market spans 2-6 million patients in the United States and approximately 16 million in Europe. Globally, there is only 1 donor cornea available for every 70 diseased eyes that need one. Over 13 million people worldwide are currently waiting for a corneal transplant, while global demand for keratoplasty reaches 12.7 million cases annually.Bullous keratopathy is caused by edema of the cornea, resulting from failure of the corneal endothelium to maintain the normally transparent, dehydrated state of the cornea. Most frequently, it is due to Fuchs corneal endothelial dystrophy or to corneal endothelial trauma which can occur during intraocular surgery or after placement of a poorly designed or mispositioned intraocular lens implant.
Cell therapy treatment for FECD is minimally invasive when compared to corneal transplant surgery and is currently administered via an injection into the anterior chamber of the eye (the fluid-filled space in front of the iris). Following injection, the patient is supine for several hours to enable the cells to settle onto the back surface of the cornea (Descemet’s membrane). If successful, the new endothelial cells form a monolayer and restore the cornea’s fluid-pumping function, clearing the corneal edema.
About the AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering) Platform
The AlloSCOPE (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering) platform highlights the key attributes of Lineage’s in-house technology and describes a differentiation and production modality from which Lineage can manufacture millions of doses of an allogeneic, cell-based product derived from a single initial pluripotent cell line, conferring consistent, cost-effective, and scalable cell-based production and which can be applied across multiple programs.
From our proprietary AlloSCOPE platform, we successfully completed a current Good Manufacturing Practice (“cGMP”) production run from a custom, two-tiered cell banking system, featuring a genetically-stable master cell bank (MCB) created from a single, well-characterized pluripotent cell line, which generated a working cell bank (WCB), which then provided the source material for two final cell-based product candidates. AlloSCOPE “5D” describes an application of AlloSCOPE with the goal of higher scale production with reduced manipulation.
About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf”, cell therapies for serious medical conditions. Lineage’s programs are based on its proprietary cell-based technology platform, AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering), and associated development and manufacturing capabilities. From this proprietary AlloSCOPE platform, Lineage develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or substantially identical to cells found naturally in the human body.
These cells are created by applying directed differentiation protocols to established, well-characterized, and self-renewing pluripotent cell lines. These protocols generate cells with characteristics associated with specific and desired developmental lineages, and in some instances may be designed to have additional beneficial properties. Cells derived from such lineages are transplanted into patients in an effort to replace or support cells that are absent or dysfunctional due to degenerative disease, aging, or traumatic injury, and to restore or augment the patient’s functional activity.
Lineage’s pipeline currently includes: (i) OpRegen® cell therapy, a retinal pigment epithelial cell therapy in Phase 2a development under a worldwide collaboration with Roche and Genentech, a member of the Roche Group, for the treatment of geographic atrophy secondary to age-related macular degeneration; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of spinal cord injuries; (iii) ReSonance™ (ANP1), an auditory neuronal progenitor cell therapy in preclinical development under a collaboration with William Demant Invest A/S for the
potential treatment of auditory neuropathy; (iv) PNC1, a photoreceptor neural cell therapy research initiative being developed for the potential treatment of vision loss due to photoreceptor dysfunction or damage; (v) RND1, a novel hypoimmune induced pluripotent stem cell line being evaluated for development under a gene editing partnership; (vi) ILT1, a cell therapy research initiative focused on the issue of large-scale production of undifferentiated pluripotent cells, which if successful could be evaluated for the production of islet cells to support a potential treatment of Type 1 Diabetes; and (vii) COR1, a corneal endothelial disease cell therapy in preclinical development for the potential treatment of corneal endothelial disease.
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