Lineage Takes Delivery of Gene-edited Hypoimmune Cell Line Under Partnership With Factor Bioscience

Key Terms

induced pluripotent stem cell medical

Cells taken from an adult (such as skin or blood) that scientists ‘reprogram’ so they behave like versatile early-stage cells capable of becoming many different cell types in the body. For investors, these cells matter because they enable development of personalized therapies, safer and faster drug testing, and potential regenerative treatments—like resetting a gadget to factory mode so it can run many different apps—creating new commercial opportunities and affecting biotech valuation and risk.

iPSC medical

Induced pluripotent stem cells (iPSCs) are ordinary adult cells that scientists have reprogrammed back into a versatile, embryonic-like state so they can become many different cell types. Think of them as factory-reset cells that can be turned into heart, nerve, or blood cells for testing drugs, modeling diseases, or developing cell therapies. For investors, iPSCs signal potential high-reward opportunities in new treatments and research tools but also carry technical, manufacturing and regulatory risks that affect commercial prospects.

hypoimmunity medical

Hypoimmunity means an unusually weak or underactive immune system that cannot respond to infections or other threats as effectively as a healthy immune system. For investors, hypoimmunity matters because it changes demand and risk for medical products and services—like increased need for antibiotics, immune-boosting drugs, or protective therapies—and can affect clinical trial design, regulatory review and the expected costs and market size for companies developing treatments, similar to how a weakened alarm system raises the need for repairs and security upgrades.

human leukocyte antigen medical

Human leukocyte antigens (HLA) are proteins on cell surfaces that act like cellular ID cards, helping the immune system distinguish self from foreign. Investors should care because HLA types influence transplant compatibility, immune-related side effects, patient selection for therapies and clinical trial outcomes; differences can affect drug safety, efficacy, regulatory approval and the need for companion tests, which in turn shapes market size and commercial risk.

CD8+ T cells medical

CD8+ T cells are a type of white blood cell that act as the immune system’s search-and-destroy team, seeking out and killing virus-infected or abnormal cells such as cancerous cells. They matter to investors because many drugs, vaccines and diagnostics aim to boost, measure or redirect these cells; their presence and activity can be a key biomarker for whether an immunotherapy works and therefore affect trial outcomes, regulatory decisions and commercial prospects.

NK cell medical

A natural killer (NK) cell is a type of white blood cell that seeks out and destroys infected or abnormal cells, including some cancer cells, without prior exposure. For investors, NK cells matter because many therapies and diagnostics aim to harness or measure their activity; changes in NK cell behavior can drive the success of drug candidates, influence clinical trial outcomes, and affect the commercial value of companies developing immune-based treatments.

gene-editing technical

Gene-editing is a set of laboratory techniques that change an organism’s DNA by adding, removing or altering specific genetic instructions—think of it as editing words in a document to fix a sentence. For investors, it matters because these tools can create new treatments, improve agricultural crops or reduce manufacturing costs, potentially driving product value, regulatory risk, research milestones and long-term revenue prospects for companies involved.

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 receipt of a novel, induced pluripotent stem cell (iPSC) line containing hypoimmunity edits, from Factor Bioscience Inc. (“Factor”). Generation of the line marks a successful milestone in the strategic collaboration between the two companies, under which Factor developed a proprietary, genetically engineered iPSC line that Lineage can utilize for differentiation into certain cell transplant product candidates. The novel cell line contains edits which are expected to support non-immune privileged and/or non-human leukocyte antigen (HLA) matched indications and includes an additional disease-specific edit with the potential to further differentiate this cell line from other cell therapies. Acceptance of the line triggered a success payment from Lineage to Factor as reimbursement for Factor’s development efforts to date. Factor remains eligible for an additional payment from Lineage subject to Lineage’s entry into an exclusive license to utilize the line. Lineage’s decision to proceed with the program will be based on further performance criteria and the outcome of additional testing, including the evaluation of the line for its ability to adapt to Lineage’s proprietary cell expansion manufacturing platform, the AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering) platform.

“Our partnership with Factor supports our plan to create novel and superior product candidates by combining our manufacturing and process development capabilities with cutting-edge cell engineering and editing technologies,” stated Brian M. Culley, Chief Executive Officer of Lineage. “This achievement under our collaboration with Factor supports our plan to broaden our cell therapy platform through the addition of new technologies and indications, as we await further updates from our lead cell therapy program, OpRegen^®, for dry age-related macular degeneration with geographic atrophy. We look forward to leveraging the knowledge and expertise we have developed in retinal cell transplantation to additional cell therapy product candidates which may have the potential to transform the treatment of a wide range of diseases. We view the convergence of directed cell differentiation with modern gene editing technology as an exciting new branch of medicine, and we plan to continue to advance this emerging space.”

“The cells we have delivered to Lineage embody the incredible potential of Factor’s gene-editing technology and represent a major milestone in the partnership between our companies,” said Matt Angel, Ph.D., Chief Executive Officer and President of Factor. “We believe Factor’s state-of-the-art cell-engineering technologies have the potential to enable treatment of diseases in ways not previously possible. Factor and Lineage together are driving progress at the leading edge of engineered cell therapy development to benefit patients.”

Lineage’s exclusive option and license agreement (the “Agreement”) with Factor was announced in 2023. The novel hypoimmune iPSC line received by Lineage contains a set of specific edits selected in a development strategy vetted with subject matter experts in the U.S. and abroad, as well as under a broader competitive landscape analysis. The edits include: the targeted deletion of the B2M gene, designed to reduce the immunogenicity of product candidates derived from the lines by inhibiting rejection by CD8+ T cells; the targeted insertion of the HLA-E gene, designed to overexpress HLA-E and prevent adverse NK cell responses; and a third undisclosed edit intended to confer clinical differentiation and a competitive advantage in the applicable indications. The Agreement provides Lineage an option to obtain an exclusive license to utilize and sublicense the novel gene-edited cell line developed by Factor in a specific field for preclinical, clinical, and commercial purposes.

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 and associated development and manufacturing capabilities. From this platform, Lineage designs, develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or 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. 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^TM (ANP1), an auditory neuronal progenitor cell therapy in development under a collaboration with William Demant Invest A/S for the potential treatment of auditory neuropathy; (iv) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage; (v) RND1, a novel hypoimmune induced pluripotent stem cell line being developed under a gene editing partnership; and (vi) ILT1, a cell therapy initiative focused on islet cell transplants for the treatment of Type 1 Diabetes. For more information, please visit www.lineagecell.com or follow the company on X/Twitter @LineageCell.

About Factor Bioscience Inc.

Founded in 2011, Factor Bioscience is a biotechnology company focused on using its patented gene-editing platform to develop life-saving cell and gene therapies. Factor is privately held and headquartered in Cambridge, MA. For more information, visit www.factorbio.com.

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,” “goal,” “suggest,” or the negative version of these words and similar expressions. Such forward-looking statements include, but are not limited to, statements relating to: our plans to develop new cell lines into potential differentiated cell transplant product candidates, including those that possess hypoimmune qualities, and the potential indications thereof; our expectations regarding the utility of edited cell lines, and our ability to broaden our cell therapy platform, or create novel and superior product candidates, by combining edited cell lines with our previously developed expertise and/or manufacturing and process development capabilities, to affect the treatment of a wide range of diseases or benefit patients; our ability to differentiate a cell line from those of competitors, including through a disease-specific edit, and to develop treatments that are differentiated from our competitors, including through a disease-specific edit; the potential of Factor’s gene-editing technology, including the potential to enable treatment of diseases in ways not previously possible. 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 Lineage, in its sole discretion, including based on further performance criteria and the outcome of additional testing, may elect not to exercise its license under the Agreement; that the potential development of new cell lines into new product candidates, or the success of those product candidates, may not be realized; 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

Media Relations for Lineage: Russo Partners

Nic Johnson or David Schull

(Nic.johnson@russopartnersllc.com)

(David.schull@russopartnersllc.com)

(212) 845-4242

Media Relations for Factor Bioscience: RF|Binder

Ted Deutsch or Armel Leslie

FactorBioscience@rfbinder.com

(609) 544-5454

Source: Lineage Cell Therapeutics, Inc.