ReNeuron Group plc announces that it has signed a patent and know-how license agreement with Schepens Eye Research Institute (Schepens), Boston, US, 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 Massachusetts Eye 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.   

Under the agreement, ReNeuron will take responsibility for the funding of the ReN003 programme and will pay Schepens license maintenance fees, together with milestone and royalty payments based on clinical and commercial success with the therapies developed.  The initial phase of ReNeuron’s collaboration with Schepens has benefited from an industrial grant from a major US specialty healthcare company and ReNeuron intends to build upon this programme-specific funding as further late pre-clinical data emerges over the coming months.

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.  

Dr Michael Young, Associate Scientist at Schepens, Associate Professor of Ophthalmology at Harvard Medical School, and lead Investigator on ReNeuron’s collaboration with Schepens, said: “It is a tremendously exciting time to be working in the field of ocular regeneration.  We are therefore delighted to be continuing our collaboration with ReNeuron under this new agreement, bringing us closer to the day when blindness can be treated with stem cells.”                                                                                        

Michael Hunt, chief executive officer of ReNeuron, said: “We are delighted to be building on our existing collaboration by signing this agreement with Schepens Eye Research Institute, a world leader in the research of regenerative and cell-based treatments for ocular diseases.  This license agreement establishes and formalises the path to clinical translation of our highly novel ReN003 programme for diseases of the retina, a disease area acknowledged in the field as one of the most promising for a cell-based therapeutic approach.”

Retinitis pigmentosa (RP) is the name given to a group of inherited diseases of the retina that all lead to a gradual and progressive reduction in vision.  This decline in vision is caused by the death of photoreceptor cells (rods and cones) found in the retina.  

Night blindness and difficulties with peripheral vision are the earliest and most frequent symptoms of RP, with reading and colour vision affected later.   The age at which symptoms start is variable and the rate of deterioration of vision also varies from person to person.  

RP is typically diagnosed in adolescents and young adults and most sufferers will be legally blind by the age of 40.  The disease affects approximately 1 in 3000 to 4000 people.

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.

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.