ReNeuron inked a patent and know-how license agreement with the Schepens Eye Research Institute in Boston, which will enable the U.K. firm to develop and commercialize therapeutics based on human retinal precursor cells (hRPCs). Lead program in this field is the preclinical candidate ReN003, for the treatment of retinitis pigmentosa, which is projected to start in clinical trials in about 18 months.
Under terms of the deal ReNeuron will have responsibility for funding the ReN003 program and will pay Schepens license maintenance fees, plus development/sales milestone and royalties. The firm in addition maintains that the hPRCs could have potential in cell therapies for other blindness-causing disorders, including age-related macular degeneration and diabetic retinopathy.
ReNeuron and Schepens have carried out an initial collaboration based on the hRPCs, and as a result of the license agreement ReNeuron will continue to work with the institute to progress ReN003 through late preclinical development and into early clinical U.S. trials. ReNeuron says work at Schepens has previously demonstrated the ability of hRPCs to integrate with host retinal tissue in rodent models. A recently optimized non-genetic cell expansion process is currently being used by ReNeuron to grow and bank clinical-grade hRPCs at quantities that will be needed for clinical trials.
The firm’s cell therapy programs are founded on a stem cell expansion technology enabling the growth of selected human stem cells, from a single tissue sample, into banks of quality-assured stem cell lines sufficient to treat perhaps thousands of patients, it claims. Importantly, the cell expansion technology can be readily scaled up for clinical and commercial use without the need to re-derive cell lines from an earlier, non-quality assured prototype. This capability has enabled ReNeuron to focus on developing allogeneic stem cell treatments addressing diseases with large patient populations.
The firm’s lead clinical-stage stem cell therapy program, ReN001, is being developed to help reverse functional deficit in patients who have been left disabled by an ischaemic stroke. The treatment is based on a neural stem cell line, designated CTX, and is undergoing a first-in-man clinical trial in the U.K. ReNeuron claims the Pisces study (Pilot Investigation of Stem Cells in Stroke) is the world's first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients, while the company is in addition the first to have received regulatory approval for any stem cell-based clinical trial in the U.K.