Rare, Orphan And Neglected Neurodegenerative Diseases – New Research Announced

According to the Ministry of Health in France, there are almost 7,000 rare diseases, with 25 million sufferers in Europe alone. A rare disease is an illness that affects fewer than on in 2,000 people, and there is no cure for most, as the only treatments currently available only improves the quality of life. Genetic defects account for 80% of rare diseases, which also include rare types of cancer, congenital malformations, autoimmune disorders, infectious diseases and intoxications. The annual Rare Diseases Day (Feb, 29 2012) aimed to improve diagnosis and treatment of rare diseases to gain further international recognition.

Rhenovia Pharma, a leading biotech company that specializes in biosimulation to discover new drugs for the treatment of neurogenerative, neurological and psychiatric diseases, has decided to expand its expertise to cover rare, orphan and neglected diseases and announced that it is starting drug research programs for Huntington’s disease (HD) and Duchenne muscular dystrophy (DMD).

Rhenovia has decided to start building a new biosimulation platform (RHENOMS(TM) STRI) that focuses on modeling the complex interaction between biological mechanisms in the striatum, a key area in the brain that plays a major role in HD, a fatal, rare neurodegenerative disease, which is extremely difficult to treat due to the wide range of symptoms, like involuntary movement disorders, cognitive deficits and psychiatric manifestations.

Doctor Serge Bischoff, president and CEO of Rhenovia said:

“It is exactly because of this variety and often opposite syndromes that the biosimulation approach is probably the most appropriate strategy in the search for new treatments. It will allow us to integrate the complexity of the biological systems affected by HD and to address the multifactorial nature of this disease.”

Rhenovia wants to consolidate its own pipeline of drug candidates, with the HD program aiming to provide new tools and solutions to optimize the Drug Discovery and Development (DD&D) process. It furthermore aims to speed up the quest in searching for new drugs and treatment methods that relieve HD symptoms and modify the course of the disease. A further objective is to consolidate Rhenovia’s own pipeline of drug candidates.

Rhenovia also wants to expand its rare, orphan and neglected disease program by developing a modeling and simulation platform that provides a better understanding of the basic mechanisms that cause cognitive impairment and mental affects linked to muscular dystrophies. Their first focus will be on DMD.

Bischoff explained:

“This choice is dictated by our privileged partnership with the French Association against Myopathies and also by the urgent medical need of DMD patients for specific treatments of syndromes linked to the deterioration of some brain functions.

Strengthening our efforts in the domain of neglected, and especially rare diseases, will not only contribute to a better understanding of the biological mechanisms and pathological manifestations underlying these diseases, but will also markedly consolidate the biosimulation technology mastered by Rhenovia.

The huge progress made by Rhenovia’s neuroscientists, chemists and informatics, physics and mathematics engineers in the last two years means we can expect significant advances by simulating deficits of rare diseases to help to shed light on the neurodevelopmental (such as autism), neurodegenerative (such as Alzheimer’s) and psychiatric diseases (such as depression and schizophrenia) affecting broader patient populations.”




3 Condivisioni