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:
ReSonance™ (ANP1) - Auditory Neurons
ReSonance™ (ANP1), is an auditory neuronal cell transplant for the treatment of hearing loss. Hearing loss is a significant unmet medical need and by 2050, nearly 2.5 billion people are estimated to be impacted by listening impairments across the globe. The loss of auditory nerve cells can lead to auditory neuropathy, even when hair cells and the cochlear nucleus remain intact. A cell-based therapy designed to replace lost or dysfunctional auditory neurons may restore hearing and enhance the degree of success of a cochlear implant procedure by repopulating the cochlea with transplanted, functional auditory neurons.
In September 2024, we reported on the successful continued preclinical development of ReSonance at the 59th Annual Inner Ear Biology Workshop. ReSonance was successfully manufactured by a proprietary process, developed in-house at Lineage’s good manufacturing practice (GMP) production facility, at clinical scale and demonstrated relevant in-vitro functional activity. In addition, the generation of a cryopreserved, ready to administer thaw-and-inject formulation of ReSonance at a clinically testable dose, demonstrated successful engraftment and survival in multiple preclinical hearing loss models.
Preclinical testing of ANP1 continues through 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.
Auditory neuropathy is a challenging hearing disorder in which the inner ear successfully detects sound but has a problem with sending signals from the ear to the brain, currently accounting for approximately 10% of cases of sensorineural hearing loss (SNHL) in children. Current state of the art medical knowledge suggests that auditory neuropathies play a substantial role in hearing impairments and deafness. Hearing depends on a series of complex steps that change sound waves in the air into electrical signals. The auditory nerve then carries these signals to the brain. Auditory neuropathy can be caused by a number of factors including damage to the auditory neurons or loss of these neurons. Researchers are still seeking effective treatments for those affected with auditory neuropathy.
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.