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A French pre-clinical biotech company offers its expertise in drug discovery and repurposing to identify personalised medicine for patients with rare monogenetic diseases, under the scope of a technical or a research cooperation agreement with patient associations, academic organisations and private companies.
The French company is a pre-clinical biotech company with a strong expertise in the fild of monogenetic disorders designing and developing miniaturised and automated in vitro cellular and phenotypic assays based on the use of patient cells. It is developing its first drug-candidate in the field of inborn skin diseases.
The company is already working closely with several patient organisations in the following areas of expertise: screening molecules, drug repurposing, inherited metabolic disorders, genodermatoses and inborn neurodevelopmental disorders.
It has research laboratories with facilities for cell culture, cellular biology, molecular biology, biochemistry and chemistry. It is based into a very fruitful environment with excellent healthcare science.
The process implemented by the company is the following:
- it collects biopsies from patients with a well-established monogenetic disorder in order to isolate fibroblasts and keratinocytes that are amplified and biobanked through its own database.
- assays are then developed using these primary or immortalized attaching cells in a standard format 384-well plates.
- the company uses imaging, spectrometry and mass spectrometry to follow the most clinically-relevant phenotypes of the diseased cells for screening molecules.
The technology can be easily settled for using other types of attaching cells. The development of a miniaturized 2D reconstituted skin suitable for high throughput screening is on-going.
The company has assembled its own and proprietary collection of molecules approved for human use that is suitable for drug repurposing. The collection contains Active Pharmaceutical Ingredients (APIs), excipients, Generally Recognized As Safe (GRAS), food supplements and phase one metabolites of APIs.
It is looking to enhance collaborations with patient associations, as well as academic organisations and private companies on drug discovery projects for molecule screening on patient primary cells and drug repurposing, offering research or technical cooperation agreements.
The company has developed technology and know-how that provides the most relevant screening assays for monogenetic disorders.
The company's main innovations can be described as follow:
1/ Fibroblasts and keratinocytes isolation and bio-banking from skin biopsies coming from patient with a diagnosed monogenetic disorder.
2/ Miniaturized and automated cellular screening assays.
3/ Microscopy facilities for HT screening on living cells.
4/ Mass spectrometry coupled with liquid chromatography adapted for HT screening based on metabolomic and fluxomic experiments.
5/ High throughput screening know-how and facilities.
6/ Original and proprietary collection of molecules approved for human use that allows drug candidates identification for repurposing and new therapeutic targets and pathways.
7/ Privileged access to 100k original molecules library
8/ Privileged access to Absorption, Distribution, Metabolism, and Excretion (ADME) in vitro %26 in vivo facilities.
9/ Strong network of medical doctors in the field of inborn error of metabolism and genodermatoses.
The technology and process are fully operational. The company has already generated several proofs of concept that have led to drug developments and compassionate uses of drugs.
The granted patents are related to the company's drug candidate which is under development.
The French company is looking to collaborate with patient organisations, academic stakeholders or private companies that would like to benefit from its expertise, know-how, and technology.
It is offering to cooperate under a technology or a research cooperation agreement, providing its services to help patients with rare skin diseases and no currently idetified treatment.