Rhenovia Pharma has announced 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. Eighty per cent are caused by genetic defects but they also include rare forms of cancer, auto-immune 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™ STRI) aimed at modeling the complex interplay between biological mechanisms in striatum, the brain region that is most affected in HD.
HD is a fatal, rare neurodegenerative disease that is particularly difficult to treat because of the very broad spectrum of symptoms it causes, involving involuntary movement disorders, cognitive deficits and psychiatric manifestations.
”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,” said Doctor Serge Bischoff, president and CEO of Rhenovia.
Doctor Bischoff continued, “It will allow us to integrate the complexity of the biological systems affected by HD and to address the multifactorial nature of this disease.”
The objective of Rhenovia’s HD program is to provide new tools and solutions to optimize 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 modeling 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.
“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,” added Bischoff.
“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.”
Rhenovia recently presented initial data on how the firm approaches HD syndromes by biosimulation at the seventh Annual Huntington’s Diseases Therapeutics Conference held in Palm Springs (California, USA).
The poster entitled ‘Use of biosimulation to facilitate drug discovery in Huntington’s disease’ can be downloaded from Rhenovia’s website.