Mayo Clinic, Oxford BioMedica collaborate to develop gene therapy for glaucoma

Oct 31 2011

Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, today announces that it has entered into a research and development collaboration with Mayo Clinic, Rochester (USA) to develop a novel gene therapy for the treatment of chronic glaucoma.  Under the terms of the agreement, Mayo Clinic and Oxford BioMedica will undertake pre-clinical studies to establish the feasibility of treating glaucoma using Oxford BioMedica's proprietary LentiVector^® gene delivery technology expressing a COX-2 gene and a PGF-2α receptor gene in order to reduce intraocular pressure.  The collaboration includes an option for exclusive US rights to license Mayo Clinic's glaucoma technology, which Oxford BioMedica can exercise upon completion of pre-clinical studies under confidential terms agreed by Mayo Clinic and the Company.

The collaboration builds on earlier pre-clinical research, conducted by Eric Poeschla M.D., Mayo Clinic and his research team, which has established initial proof-of-concept for this approach to treating chronic glaucoma.  This work will be extended using the LentiVector^® technology and may support the rapid transition of another novel ocular gene therapy into clinical development.

Glaucoma is a group of eye diseases characterised by vision loss due to damage of the optic nerve affecting over 60 million people worldwide (source: Mayo Clinic).  Over 90% of glaucoma is classed as primary open-angle glaucoma (also known as chronic glaucoma or chronic open-angle glaucoma) which results from a partial blockage within the trabecular meshwork of the eye, the tissue mainly responsible for draining the internal fluid of the eye (aqueous humour).  As the aqueous humour builds up, it causes increased intraocular pressure which can damage the optic nerve.  Damaged parts of the nerve and retina lead to permanent patches of vision loss and, in some cases, can lead to blindness.

Current treatment options for glaucoma aim to reduce intraocular pressure either through topical methods (e.g. eye drops) or eye surgery, however these approaches are not effective in all cases.  Patient compliance with topical treatments can be very poor, especially those requiring daily application, and many therapies have side effects which can restrict chronic use.  Consequently, there remains a large unmet medical need in a significant proportion of the patient population.  Oxford BioMedica has conducted pre-clinical and clinical studies that suggest a single application of its LentiVector^® platform products can provide sustained or permanent therapeutic activity.  By using a novel gene therapy to provide long-term control of intraocular pressure, this approach could minimise the risk of disease progression.

Stuart Naylor, Chief Scientific Officer of Oxford BioMedica, said: "We believe that our LentiVector^® gene delivery system is perfectly suited to tackle chronic diseases of the eye.  In addition to our four Phase I/II ocular programmes partnered with Sanofi which are progressing well, we are keen to explore new opportunities to which we can apply our unique technology.  We are pleased to be working with Mayo Clinic, a global leader in medical research, on a potential treatment for glaucoma that will further leverage our LentiVector^® platform technology and broaden our ocular development pipeline."

Dr Eric Poeschla at Mayo Clinic, USA, commented: "Numerous aspects of glaucoma are favorable for this approach.  The disease's lifelong persistence and the incomplete efficacy and adherence seen with current treatment methods are two of the main problems that make achieving a sustained therapeutic effect via gene therapy an appealing prospect.  In addition, the target tissues involved in regulating intraocular pressure are relatively small and confined, which enhances gene delivery feasibility.  Finally, we have shown that the approach causes sustained reduction in intraocular pressure in pre-clinical models.  We are pleased to collaborate with Oxford BioMedica on research and clinical translation for the treatment of glaucoma."