mRNA cell engineering strategically targets genes to create novel and potentially superior product profiles for cell transplant therapy applications

Novel engineered iPS cell line to be evaluated by Lineage for potential development of cell transplant therapies for central nervous system diseases under collaboration initiated in early 2023

Lineage to make milestone payments to Eterna in connection with Eterna’s successful development and delivery to Lineage of certain materials

CAMBRIDGE, MA, USA I September 06, 2023 IEterna Therapeutics Inc. (Nasdaq: ERNA) (“Eterna”), a life science company committed to realizing the potential of mRNA cell engineering to provide patients with transformational new medicines, today announced the initiation of certain development activities to generate a novel hypoimmune induced pluripotent stem cell (iPSC) line under its option and license agreement (the “Agreement”) with Lineage Cell Therapeutics, Inc. (“Lineage”). This marks the next step in the strategic collaboration announced in February 2023, under which Eterna is developing innovative engineered hypoimmune iPSC lines that Lineage will evaluate for development into differentiated cell transplant therapies for central nervous system (CNS) diseases and other neurology indications.

“We are excited to move forward with the next phase of our partnership with Lineage,” said Matt Angel, Ph.D., Chief Executive Officer and President of Eterna. “We believe that pluripotent cell therapies have the potential to significantly outperform traditional approaches in certain settings, and this milestone highlights Eterna’s capabilities for generating novel gene-edited iPSC lines using our mRNA cell engineering platform.”

“Our partnership with Eterna reflects an important step in a corporate strategy intended to capitalize on our existing process development capabilities by combining them with cutting-edge cell engineering and editing technologies, to create novel and potentially superior product profiles,” stated Brian M. Culley, Chief Executive Officer of Lineage. “This collaboration reflects our effort to broaden the application of our cell therapy platform and our plans for future success in this growing field. We look forward to leveraging our expertise to develop innovative cell transplant therapies that have the potential to transform the treatment of a wide range of diseases by capitalizing on the convergence of directed cell differentiation and manufacturing with modern gene-editing technology.”

Eterna’s next-generation mRNA gene-editing approach is designed to efficiently inactivate target genes and to replace viral methods for insertion of genes of interest into target cells for long-lasting expression of transgenes. Since announcing the deal earlier this year, Lineage has evaluated its development strategy with a group of leading neurology experts in the U.S. and abroad. As a result of these and other discussions, and an assessment of the competitive landscape, Lineage finalized its selection of specific gene edits for the initial cell lines to be developed by Eterna and made an initial payment to Eterna under the Agreement. It is anticipated that these edits would expand the edited cell lines’ overall utility, including for non-immune privileged or non-human leukocyte antigen (HLA) matched indications and will further differentiate the cell line from others currently in use by competitors. The novel hypoimmune iPSC line to be developed under the Agreement will include the following three edits:

  • 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
  • Targeted insertion of the HLA-E gene, designed to overexpress HLA-E and prevent the allogeneic NK cell response
  • A third undisclosed edit intended to confer clinical differentiation and a competitive advantage in the applicable indications

Under the Agreement, Eterna plans to conduct certain gene-editing activities and provide materials to Lineage for evaluation. Lineage will make milestone payments to Eterna and in connection with Eterna’s successful delivery to Lineage of certain materials. Lineage also has an option to obtain an exclusive license to utilize and sublicense the novel gene-edited cell lines developed by Eterna for preclinical, clinical, and commercial purposes in developing potential treatments for CNS diseases.

About Eterna Therapeutics Inc.
Eterna Therapeutics is a life science company committed to realizing the potential of mRNA cell engineering to provide patients with transformational new medicines. Eterna has in-licensed a portfolio of over 130 patents covering key mRNA cell engineering technologies, including technologies for mRNA cell reprogramming, mRNA gene editing, the NoveSlice™ and UltraSlice™ gene-editing proteins, and the ToRNAdo™ mRNA delivery system from Factor Bioscience. NoveSlice™, UltraSlice™, and ToRNAdo™ are trademarks of Factor Bioscience. For more information, please visit

About Lineage Cell Therapeutics Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical and preclinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen®, a retinal pigment epithelial cell therapy in Phase 2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, is being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer; (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; and (v) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit or follow the company on Twitter @LineageCell.

SOURCE: Eterna Therapeutics