LYSOGENE aims to develop and use the extraordinary medical potential of advanced therapies based on gene transfer enabling the treatment, by a single dose protocol, of genetically caused neuro-degenerative illnesses in children.
Dedicated to providing innovative treatments for children suffering from rare conditions of the central nervous system, LYSOGENE is involved in fields of high scientific and ethical value and is building the basis for its future development. This is why the company chose, from its very beginning, to develop its work around four major commitments.
LYSOGENE is a catalyst for the full range of complex expertise required for the design of innovative and high performance solutions, and for successfully carrying out its mission. Its expertise covers a range of complex fields dealing with:
- Rare diseases and in particular pediatric pathologies of the central nervous system.
- Viral gene transfer.
-┬áThe design of therapeutic vectors, suitable for clinical development.
- All the regulatory issues concerning studies on effectiveness, toxicology and bio-distribution, GMP vector production (managing and controlling issues of viral Quality and Security).
- A strategy and methodology for totally unique and high performance regulatory streamlining.
LYSOGENE positions regulatory issues at the heart of its development programme, given the complexity and changing nature of the challenges it faces: small patient numbers, pediatric demographics, adaptational design, validation of clinical criteria, etc.
LYSOGENE has proved its exceptional capacity to facilitate and accelerate the transfer of technology from the research lab to the patient's bed, in a complex pathology of the central nervous system.
The development of advanced therapies, particularly in children, presents numerous challenges. To be efficient and become a leader in such an environment demands in-depth knowledge, the anticipation and management of highly complex challenges and risks that are scientific, operational, financial and regulatory, clinical and human.
LYSOGENE is focussed on excellence, rigour and audacity, as much in the scientific and medical aspects of its work, as in the methodology, optimisation of procedures and the use of valuable resources.
The development of cutting-edge treatments that use the latest advances in genetic knowledge and are aimed at children, requires total ethical commitment. LYSOGENE places ethics, patients and their families at the heart of its issues, because they are are and must remain the direct beneficiaries of safe and effective bio-pharmaceutical products.
In this field in particular, success is very closely dependent on the in-depth understanding of the conditions and their impact on the daily lives of patients and their families and by the assimilation and use of this understanding at each stage of┬á development, especially during the clinical trial phase.
LYSOGENE's primary responsibility is to develop and deliver treatments for extremely vulnerable patients, in extreme suffering, for whom there is no available cure.
By extension, LYSOGENE aims to improve the quality of life of all the families concerned.
But its commitment to social responsibility does not stop there: through the broad scope of scientific and medical activities with high added value, LYSOGENE aims to positively impact all of the communities involved: patients, families, pharmaceutical companies, financial institutions.