Lineage Launches New Cell Therapy Program in Corneal Endothelial Disease

Key Terms

allogeneic medical

Allogeneic describes a process or material involving different individuals of the same species, such as cells, tissues, or organs donated from one person to another. It is important to investors because products or treatments based on allogeneic sources can enable scalable, off-the-shelf solutions, potentially reducing costs and increasing accessibility in healthcare and biotech industries.

corneal endothelial disease medical

Corneal endothelial disease is a condition in which the innermost layer of cells on the clear front surface of the eye stops removing excess fluid, causing the cornea to swell and vision to blur. For investors, it matters because it drives demand for medical treatments, surgical procedures, implantable devices and drugs, and can influence regulatory approvals, clinical trial activity and revenue potential in eye-care companies.

fuchs endothelial corneal dystrophy medical

Fuchs endothelial corneal dystrophy is a progressive eye disease in which cells on the inner surface of the cornea fail, causing the clear front of the eye to swell and vision to blur, often like a once-smooth window becoming foggy. It matters to investors because it creates ongoing demand for treatments, surgical procedures and medical devices, making it a driver of revenue and clinical development in ophthalmology markets.

bullous keratopathy medical

A painful eye condition where the cornea (the clear front window of the eye) swells and develops fluid-filled blisters because its inner cell layer can’t pump fluid away, leading to blurred vision and light sensitivity. It matters to investors because it defines a clear patient need and potential market for treatments, devices, or surgical procedures—think of it like a damaged windshield that urgently needs repair, creating demand for effective solutions.

corneal endothelial cells medical

Corneal endothelial cells form a single layer on the inner surface of the cornea and act like tiny pumps that keep the cornea clear by controlling its water balance. They matter to investors because loss or dysfunction of these cells causes vision-threatening swelling and scarring, making them a key target for medical treatments, surgical devices, and drug approvals that can drive commercial value and regulatory risk in ophthalmology-related investments.

pluripotent medical

A pluripotent cell is a type of stem cell that can develop into nearly any specialized cell in the body, such as heart, nerve, or liver cells. Think of it as a versatile blank building block that researchers can coax into different parts for repairs, drug testing, or cell therapies; its broad potential makes it important to investors because advances or setbacks in pluripotent cell technologies can significantly affect the value of regenerative medicine and biotech ventures.

cagr financial

Compound Annual Growth Rate (CAGR) measures the average yearly growth of an investment, revenue, or other metric over a multi-year period as if it had grown at a steady rate each year. Think of it like the constant speed that would take you from the starting value to the ending value over the same time—useful because it smooths out ups and downs and lets investors compare different assets or performance periods on an even footing.

• New Internally-Developed and Wholly-Owned Asset Benefits From Existing Ophthalmology and Manufacturing Expertise
• Fuchs Endothelial Corneal Dystrophy (FECD) Afflicts More Than 7.3% of the Population with a Predicted 10-Year CAGR of 7.7%

CARLSBAD, Calif.--(BUSINESS WIRE)-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), 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. For more information, please visit www.lineagecell.com or follow the company on X/Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. In some cases, forward-looking statements, can be identified by terms such as “believe,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “suggest,” or the negative version of these words and similar expressions. Such forward-looking statements include, but are not limited to, statements relating to: the potential of the COR1 preclinical program and our continuing development and planned pre-clinical activities, including our potential to produce and provide a more consistent, cost-effective and “off the shelf” product that could address the global shortage of donor cells; the application of recent advancements with our AlloSCOPE “5D” platform to the COR1 preclinical program to potentially reduce production costs through large-scale production; the potential of the AlloSCOPE platform and its ability to manufacture millions of doses of an allogeneic, cell-based product, including islets cells, derived from a single initial cell line, conferring consistent, cost-effective, and scalable cell-based production, across multiple programs, and the extent to which such ability will remain beyond the reach of many competitors. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the following risks: that development activities, preclinical activities, and clinical trials of our product candidates may not commence, progress or be completed as expected due to many factors within and outside of our control; that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; that the ongoing Israeli regional conflict may materially and adversely impact our manufacturing processes, including cell banking and product manufacturing for our cell therapy product candidates, all of which are conducted by our subsidiary in Jerusalem, Israel; that Lineage may not be able to manufacture sufficient clinical quantities of its product candidates; and those risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the SEC, including Lineage’s most recent Annual Report on Form 10-K filed with the SEC and its other subsequent reports, which are available on the SEC’s website at www.sec.gov. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Lineage undertakes no obligation to update any forward-looking statement to reflect events that occur or circumstances that exist after the date on which they were made except as required by law.

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Lineage Cell Therapeutics, Inc. IR

Ioana C. Hone

(ir@lineagecell.com)

(442) 287-8963

Russo Partners – Media Relations

Nic Johnson or David Schull

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(David.schull@russopartnersllc.com)

(212) 845-4242

Source: Lineage Cell Therapeutics, Inc.