Drug Discovery Programs for Rare Neurodegenerative Diseases

Rhenovia Pharma, a leading biotech company in biosimulation applied to the discovery of new medications to treat neurodegenerative, neurological and psychiatric diseases, announces today that it has initiated drug research programs in rare, orphan and neglected diseases. Rare diseases are defined as those affecting fewer than one in 2,000. 80% are caused by genetic defects but they also include rare forms of cancer, autoimmune disorders, congenital malformations, infectious diseases and intoxications. France’s Ministry of Health data suggest that there are nearly 7,000 rare diseases. Europe alone accounts for some 25 million sufferers.

No cures exist for most rare diseases. The only currently available treatments are those that improve the quality of life. The annual Rare Diseases Day, which took place this year on February 29, extends the international recognition of these diseases with the aim of improving diagnosis and treatment. Rhenovia’s decision to expand its expertise to cover rare, orphan and neglected diseases is based on  the need to respond to this pressing challenge. Its initial focus will be on Huntington’s disease (HD) and Duchenne muscular dystrophy (DMD). As a first step, Rhenovia is building a new biosimulation platform (RHENOMS(TM) STRI) aimed at modelling the complex interplay between biological mechanisms in striatum, the brain region that is most affected in HD.

The objective of Rhenovia’s HD program is to provide new tools and solutions to optimise the Drug Discovery and Development (DD&D) process and accelerate the search for new treatment strategies  and medications, not only for relieving HD patients from their symptoms, but also for modifying the course of their illness. A further objective is to consolidate Rhenovia’s own pipeline of drug candidates. A second direction in Rhenovia’s rare, orphan and neglected disease program is the development of a  modelling and simulation platform designed to better understand the basic mechanisms underlying cognitive impairment and mental affects associated with muscular dystrophies with a first focus on DMD.