CARLSBAD, Calif. – 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, 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) ReSonanceTM (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.
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.
