Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss
The company’s lead cell therapy candidate, OpRegen, has demonstrated efficacy in dry AMD.
Lineage Cell Therapeutics is expanding its pipeline to develop a cell therapy for the potential treatment of hearing loss.1
With the expansion, Lineage is adding a third neuronal cell type to its platform. The company has filed for intellectual property covering the composition and methods of generating this type of cell therapy, including the generation of auditory neuronal progenitors, as well as methods of treatment involving these potential cell therapies. The company will initially focus on auditory neuropathy spectrum disorders.
“Expanding into the treatment of hearing loss is an example of the power of our proprietary technology and builds on our recent success in cell therapy. Traditional product development has failed to deliver pharmacological agents for patients with auditory deficiencies and the field is in need of new approaches, like ours," Brian Culley, chief executive officer, Lineage, told CGTLive.
Lineage’s pipeline currently consists of cell therapies for the potential treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA), spinal cord injury, and non-small cell lung cancer. Its lead candidate is OpRegen, a human retinal pigment epithelium (RPE) cell therapy being developed in collaboration with Roche and Genentech. It is currently being assessed in a phase 1/2 clinical trial (NCT02286089) for the treatment of GA in dry AMD.
"We are building on the same principles which helped us generate clinical outcomes in certain types of blindness which were beyond the reach of traditional approaches," Culley added. "By utilizing the same 'replace and restore' principles, we are optimistic that we can safely provide durable and functional cells and improve the quality of life for affected individuals. We have been able to launch this new program rapidly, with a modest investment, and because our cell lines are capable of becoming any cell in the body, we have opportunities to add more programs like this, if we elect to do so.”
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Interim results from OpRegen’s trial were presented in September 2021.2 The data showed the investigational therapy’s efficacy, including an average difference in Best Corrected Visual Acuity (BCVA) of over 2 ETDRS lines (10.8 letters read) between treated and untreated eyes in a cohort of patients 9 to 12 months after treatment.
As of September, the trial had enrolled 24 patients in 4 cohorts, the first 3 of which enrolled participants with BCVA of 20/200 or worse. The fourth cohort enrolled 12 participants with BCVA from 20/65 to 20/250 with smaller mean areas of GA and also investigated a new “thaw-and-inject” formulation of OpRegen.
In November 2021, the company reported that all 4 patients from the fourth cohort who had been treated with OpRegen experienced retinal tissue restoration as seen on optical coherence tomography, as well as experiencing improved visual acuity--improvements which remained statistically significant between treated and untreated eyes at 9, 12, and 15 months post-treatment.3
“There is nothing approved in dry AMD. Notably, the leading approaches have not shown any improvement to visual acuity. OpRegen may be able to address not only the underlying anatomical problem, but also provide improved visual acuity, all with one-time dosing,” Culley previously told CGTLive.
