LYSOGENE

LYSOGENE is a leading gene therapy biotechnology company

LYSOGENE is a leading gene therapy biotechnology company developing breakthrough treatments targeting severe genetic pathologies with Central Nervous System involvement and high unmet medical needs. Founded in 2009 and having grown rapidly, LYSOGENE is the only biotechnology company in the world that is specialised in the development of intracerebral gene therapy aimed at treating pathologies affecting the central nervous system, which is the main cause of neuro-degenerative disease mortality in children. Certain advanced therapies, such as gene therapy offer natural advantages compared with the administration of recombinant proteins, which is almost the only therapy available on the market for the treatment of some peripheral symptoms in a limited group of lysosomal storage disorders. As well as the high cost of these treatments and the difficulty in producing the proteins, they do not reach the central nervous system when injected intravenously and their effectiveness remains limited due to their rapid elimination. This means that repeated injections of proteins are required, sometimes in large doses. In a single dose treatment, intracerebral gene therapy enables the central nervous system to be targeted, providing constant levels of therapeutic protein. This unique feature (single dose v. repeated treatments) represents not only a considerable benefit for future patients and their families, single dose treatments should also present the considerable advantage of offering an economical form of therapy that is much more in line with the considerable budgetary constraints currently weighing on health care systems around the world. In 2013, LYSOGENE successfully completed its Phase I/II gene therapy study using its lead product (SAF-301) in patients with Sanfilippo syndrome. The SANFILIPPO syndrome is a CNS illness or storage disorder that is extremely severe and debilitating, fatal in childhood and that currently has no known treatment. The clinical stage trials of an intracerebral gene therapy for a pediatric condition represent a world first for a private company. This comes only three years after validation of the proof of principle in animal trials. Rare or orphan diseases affect less than 5 in 10,000 people according to European criteria or less than 300,000 people according to criteria in the USA. In total, rare diseases affect 3 million people in France and more than 30 million in Europe. Out of 8,000 rare diseases identified to date, more than 80% are of genetic origin and 50% affect children. They are the primary cause of neurodegeneration in children. For the last few years, they have been unanimously considered as model diseases, indisputably driving medical advances in providing universal assistance to all patients, including those suffering from common illnesses. LYSOGENE aims to expeditiously advance the clinical development and worldwide commercialization of SAF-301. LYSOGENE is actively expanding its pipeline to additional genetic diseases with high unmet medical needs. Currently, the Company is lauching its new gene therapy translational program LYS-XXX. LYS-XXX targets a severe monogenic indication with strong CNS involvement. The company aims to translate LYS-XXX to phase I/II clinical stage in EU and US no later than 2016.