Development of new stem cell treatment for retinal diseases

The company ReNeuron has announced that it has signed a patent and know-how license agreement with Schepens Eye Research Institute, Boston, USA, regarding the Company’s ReN003 stem cell therapy programme focused on diseases of the retina.

An affiliate of Harvard Medical School, Schepens recently announced that it is to join forces with the MassachusettsEye and Ear Infirmary in Boston to create the world’s largest pre-clinical and clinical ophthalmology research centre.  ReNeuron has been collaborating with Schepens in the early development of its human retinal precursor cells (hRPCs).  Based on the successful results of this initial collaboration, the Company has, through this license agreement, secured the relevant intellectual property rights to develop and commercialise its hRPCs in the field of human retinal stem cell therapeutics.  ReNeuron will continue to collaborate closely with lead Investigator Dr Michael Young and his team at Schepens to take the Company’s ReN003 programme through late pre-clinical development and into an initial clinical trial in the US in patients suffering from retinitis pigmentosa, a blindness-causing disease caused by degeneration of the photoreceptor cells in the retina. 

Researchers at Schepens have already published data describing the ability of the hRPCs to integrate with host retinal tissue in rodent models of damaged retina and differentiate into the light-sensitive rod cells found in healthy retina.  Subsequently, a novel and highly efficient proprietary cell expansion process has recently been optimised which does not involve genetic modification or other similar manipulation of the hRPCs.  This expansion technology is currently being employed by ReNeuron to grow and bank clinical-grade hRPCs to the quantities required for future clinical studies.  

Subject to regulatory advice and the results of IND-enabling late pre-clinical studies, the ReN003 programme is expected to enter its clinical phase in approximately 18 months.  Importantly, although retinitis pigmentosa is the initial target disease, the hRPCs developed in the programme will almost certainly be applicable as cell therapy candidates for other blindness-causing diseases, such as age-related macular degeneration and diabetic retinopathy. 

Schepens Eye Research Institute fights blindness by developing new technologies, therapies and knowledge to preserve and restore vision. Through a continuum of discovery, the Institute works toward a future in which blindness is prevented, alleviated, and, ultimately, cured.

Founded in 1950 by famed retinal surgeon Charles L. Schepens, M.D., Schepens Eye Research Institute is the largest independent eye research institute in the United States and an affiliate of Harvard Medical School. Since its inception, the Institute has trained more than 600 postdoctoral fellows in various disciplines of eye research; trained more than 500 eye surgeons who now practice around the world; and published more than 4,600 scientific papers and books about health and eye disease.

About ReNeuron:

ReNeuron is a leading, clinical-stage stem cell business.  Its primary objective is the development of novel stem cell therapies targeting areas of significant unmet or poorly met medical need.  

ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant disease conditions where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments.  ReNeuron’s lead candidate is its ReN001 stem cell therapy for the treatment of patients left disabled by the effects of a stroke. This therapy is currently in clinical development.  ReNeuron’s ReN009 stem cell therapy is being developed as a treatment for peripheral arterial disease, a serious and common side-effect of diabetes. The Company is also developing stem cell therapies for other conditions such as blindness-causing diseases of the retina.

ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research.  The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.