LYSOGENE is a clinical stage pioneering biotechnology company in the field of gene therapy targeting severe genetic pathologies of the central nervous system in children.

 

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.

LYSOGENE is currently conducting the clinical trials of its first product, SAF-301, an experimental treatment aimed at treating type III A Mucopolysaccharidosis, also known as SANFILIPPO type A 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.

It is in this framework that LYSOGENE operates, and with the vision of the potential that the first single dose treatment of lysosomal conditions affecting the central nervous system represents.