Lineage Cell Therapeutics Receives Gene-Edited Stem Cell Line for Immune System Compatibility Studies

Lineage will evaluate the new gene-edited iPSC line for immune compatibility and manufacturing potential before pursuing exclusive rights with Factor Bioscience.

Key findings

  • Lineage Cell Therapeutics has received a gene-edited induced pluripotent stem cell (iPSC) line from Factor Bioscience, which includes “hypoimmune” edits aimed at reducing immune rejection.
  • Lineage is currently testing whether the line fits within its manufacturing platform, AlloSCOPE, before deciding on an exclusive license. 

Lineage Cell Therapeutics, a biotechnology company developing cell therapies for neurological and ophthalmic conditions, has received a new induced pluripotent stem cell (iPSC) line from Factor Bioscience under a collaboration announced in 2023.

Factor Bioscience is a Cambridge-based biotech company that develops new methods for controlling gene expression and cell fate. They currently have over 150 patents.

The iPSC line

The line Factor delivered is genetically engineered with “hypoimmunity” edits to reduce the risk of rejection in derived cell therapy products, for indications where immune matching is unavailable or impractical:

  • B2M gene deletion: intended to reduce immunogenicity (how strongly the immune system recognizes the cells as foreign) by inhibiting rejection by CD8+ T cells.
  • HLA-E gene insertion: intended to increase HLA-E expression and prevent adverse natural killer (NK) cell responses.
  • Third edit (undisclosed): described as a disease-specific change intended to provide “clinical differentiation and a competitive advantage” in relevant indications.

What’s next?

Lineage says, based on the testing and performance criteria, including whether the line can adapt to its proprietary cell expansion manufacturing platform, AlloSCOPE (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering), they’ll decide if they continue with an exclusive license or not. 

Brian M. Culley, Chief Executive Officer of Lineage, said: “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.”

Company snapshot

Lineage is developing allogeneic (off-the-shelf) cell therapies derived from pluripotent cell lines using directed differentiation (protocols that push stem cells toward specific cell types). Its pipeline includes:

  • OpRegen (retinal pigment epithelial cells), in Phase 2a for geographic atrophy secondary to age-related macular degeneration, under a collaboration with Roche and Genentech
  • OPC1 (oligodendrocyte progenitor cells), in Phase 1/2a for spinal cord injuries
  • ReSonance (ANP1) (auditory neuronal progenitor cells), in development under a collaboration with William Demant Invest A/S for auditory neuropathy
  • PNC1 (photoreceptor neural cells), in development for vision loss due to photoreceptor dysfunction or damage
  • RND1, the hypoimmune iPSC line being developed under the gene editing partnership
  • ILT1, a program focused on islet cell transplants for Type 1 Diabetes

More information: lineagecell.com and factorbio.com.

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