5.1 million people worldwide suffer from recessive retinal dystrophies, rare genetic diseases which cause a total loss of eyesight. Retinal dystrophies are divided into two broad categories: recessive retinitis pigmentosa (+ 3.5 million) and macular dystrophies (+ 1.6 million). For over 20 years, basic research has been funded by the universities and the University Hospitals of Nantes and Montpellier, the French Association against Myopathy and Inserm, which today allows consideration of clinical trials in humans. Horama aims to take over the academic research, to build on it and develop the therapies of tomorrow.
Founded in early 2014, Horama aims to place its first gene therapy medicinal product on the market by 2019. Today, 200 genes are identified as being responsible for retinal dystrophies. Horama is working on a portfolio of products including HORA-PDE6B which will enter in clinical phase in 2016. Denis Cayet, President and founding shareholder of Horama emphasizes the company’s management choices: “We decided from the outset to minimize the company’s structure costs and to choose a joint investment model likely to best support our ambition to heal. That is why our strategy consists of combining conventional fundraising and industrial partnership contracts.»
Within just six months of its creation, Horama already benefits from the confidence of private investors and has managed to raise €450,000 from its shareholders and Vendée entrepreneurs (founders of SODEBO and Ernest SOULARD companies). The high economic potential linked to the rare diseases specific market (€76 billion in 2014), a strong scientific basis and a promising technology, combined with atypical governance and management mode are all characteristics that give confidence in the project.
“Horama’s distinctive characteristic” said Olivier Pichon, BPI Pays de la Loire, “mainly comes from the commitment of its leaders, in association with a virtuous approach and outstanding ethics. In addition, having a real and experienced entrepreneur, such as Denis Cayet, at the helm alongside a leading scientific committee has enabled the transformation of the project into a company at the time when the project was ready. Finally, with such a pool of investors from the very creation of the company, BPIfrance’s participation almost seemed to follow naturally.»
Horama brings together the skills of scientists, who are internationally recognized in the field of gene therapy in order to exploit their academic research within a private structure and treat orphan inherited retinal dystrophies. Thus we find in Nantes, Dr. Philippe Moullier (Director of Research at INSERM [UMR 1089], and Director of the Atlantic Gene Therapies Institute), Dr. Fabienne Rolling (INSERM Research Director, and Director of the “Translational Gene Therapy of retinal diseases” team within UMR1089), Prof. M. Weber (Head of Department at the eye clinic of the Nantes University Hospital, and University Professor- Hospital Practitioner) and Dr. Guylène Le Meur (Hospital Practitioner at Nantes University Hospital specializing in paediatrics ophthalmology and genetic eye diseases, and Lecturer at Nantes University); in Montpellier, Prof. Christian Hamel (formerly Research Director at Inserm, founding director of Montpellier Neurosciences Institute, and current head of the “Genetics and therapy of retinal blindness and optic nerve” team within the Institute [UMR 1051]) and finally, Dr. Vasiliki Kalatzis (Manager of Research at the Montpellier Neurosciences Institute, and Director of the “Gene therapy of retinal dystrophies” group). Thus Horama covers the value chain from discovery of the gene to treatment of patients.
Horama’s gene therapy technique is to inject under the retina, a vector derived from a recombinant adeno-associated virus (AAV) which carries the (healthy) therapeutic gene of interest. This vector will allow the healthy gene to access the cells in which it is mutated or missing and to release the healthy protein. The retina is a particularly suitable area for gene therapy. This small enclosed space actually permits the injection of small doses of vectors that cannot migrate to other parts of the body. Retinal dystrophies are excellent candidates for gene therapy because of their monogenic nature and their slow evolution, which open a broad therapeutic window before the irrecoverable loss of sight.
The concern for patients, is the ability to diagnose and treat the disease at the earliest to have a chance to preserve their visual independence and enable them to lead a normal life. For the community, the challenge is to reduce the cost of the necessary assistance implemented to facilitate the daily life of these patients. Horama aims to treat tens of thousands of people worldwide through the production of gene therapy medicines applied to ophthalmology. Like all health biotechnology companies, investments are critical to developing treatments and turning them into products that can be placed on the market, once all regulatory aspects have been validated. Another 4 million euros fundraising is planned for late 2015, and is needed by Horama to carry on until 2018, with the first projected medicines.
Dominique Constantini, CEO of OsePharma and member of Horama Strategic Committee “it is very moving to see that in the field of gene therapy, we go from dream to clinical reality. Therapeutic innovations in this field represent a powerful engine to create products that break with conventional therapies. Horama is now working on the development of a first very promising product that will certainly be followed by others, thanks to the high quality, multidisciplinary team associated with a product based strategy on which it intends to develop the company.”