Other Development Programs
Select a program below to learn more
Lineage is developing several additional product candidates which cover a range of therapeutic areas. These product candidates are based on the same pluripotent platform technology and employ a similar guided cell differentiation and transplant approach as our current clinical-stage products. Read more about these programs below:
ANP1 - Auditory Neurons
ANP1 is intended to treat auditory neuropathy spectrum disorders (hearing loss), which affects more than 400 million people worldwide. Positive initial proof of concept results demonstrated delivery, engraftment, and survival of ANP1 cells into specific target areas. Based on these encouraging results, the ANP1 program has been advanced from product concept to functional preclinical testing.
In February 2023, we entered into a collaboration with the University of Michigan and Yehoash Raphael, Ph.D., The R. Jamison and Betty Williams Professor of Otolaryngology, Department of Otolaryngology-Head and Neck Surgery and Lab Director at the University of Michigan Kresge Hearing Research Institute, where preclinical testing of ANP1 has been ongoing. Initial preclinical results have been positive, with a demonstrated ability to deliver ANP cells into specific target areas utilizing standard surgical tools as well as to establish initial engraftment into certain anatomical destinations and survival after transplantation. ANP cells were confirmed to retain the expression of neuronal-specific markers post-transplantation and additionally demonstrated the ability to migrate. These results support the advancement of ANP1 into its new phase of preclinical development, the evaluation of the long-term engraftment of ANP cells and their functional assessment in hearing loss.
We have two pending patent applications for our ANP1 program, a pending U.S. provisional patent application and a pending PCT patent application. It is anticipated that the pending provisional patent application will be converted to a PCT patent application in 2024. It is anticipated that the pending PCT patent application will be converted to a U.S. utility patent application and one or more international patent applications in 2025 and, if issued, would have estimated patent expiration dates in 2043.
PNC1 - Photoreceptors
PNC1 is intended to treat conditions of photoreceptor (rods and cones) loss or dysfunction (vision loss, Retinitis Pigmentosa, etc.). Leveraging our know-how and capabilities in ophthalmology along with our intellectual property covering compositions and methods of generating photoreceptors.
We have rights to two patent families for our PNC1 program. These patent families include issued U.S. and international patents and pending patent applications.
RND1 - Neurology
RND1 is a novel hypoimmune pluripotent stem cell (“iPSC”) cell line being developed in collaboration with Eterna Therapeutics Inc. (“Eterna”), which we intend to evaluate for differentiation into cell transplant product candidates for central nervous system (“CNS”) diseases and other neurology indications.
In February 2023, we entered into an option and license agreement with Eterna to develop engineered hypoimmune iPSC lines that we will evaluate for differentiation into cell transplant product candidates for CNS diseases and other neurology indications. We believe this collaboration allows us to leverage our expertise to develop innovative cell transplant therapies by capitalizing on the convergence of directed cell differentiation and manufacturing with modern gene editing technology. This is reflective of a portion of our corporate strategy which aims to capitalize on our process development capabilities by combining them with cell engineering and editing technologies to create novel cell therapies with potentially superior product profiles compared to currently marketed therapies, if any.
In September 2023, we announced the initiation of certain development activities to generate a novel iPSC line under our agreement with Eterna and our selection of specific gene edits for the initial cell line to be developed by Eterna. 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 the allogeneic NK cell response; and a third undisclosed edit intended to confer clinical differentiation and a competitive advantage in the applicable indications. We expect that these edits may expand the edited cell lines’ overall utility, including for non-immune privileged or non-human leukocyte antigen (“HLA”) matched indications and may further differentiate the cell line from others currently in use by competitors.
VAC Platform
VAC is our immuno-oncology platform using dendritic cells loaded with antigens for the treatment of cancer. As the most potent type of antigen-presenting cell in the body, dendritic cells instruct the human body’s immune system to attack and eliminate harmful pathogens and unwanted cells, including cancer cells. VAC2 is an allogeneic, or non-patient specific, cancer vaccine candidate designed to stimulate patient immune responses to an antigen, human telomerase reverse transcriptase (“hTERT”), which is commonly expressed in cancerous cells but is not usually found in normal adult cells.
VAC2 was the subject of a Phase 1 clinical trial in advanced non-small cell lung cancer conducted by our partner, Cancer Research UK with encouraging primary and secondary endpoint results reported in 2023.
Because many different antigens could be employed as part of this allogeneic dendritic cell system, we believe that strategic collaborations offer the best alternatives to advance the VAC platform moving forward and we continue to be engaged in exploratory discussions for the development of various VAC assets. We intend to continue monitoring the neoantigen vaccine landscape to help inform our corporate strategy and determine the best development path for VAC2 or any other VAC platform programs.