Sylentis seeks innovative therapeutic agents based on interference RNA (RNAi), a new technology whose discoverers were awarded the Nobel Prize for Medicine in 2006. Focused primarily on treatments for ophthalmology, it has two compounds in clinical trials for glaucoma and ocular pain associated to dry eye syndrome, and other molecules in pre-clinical development in other areas.
Sylentis’s aim is to exploit RNAi technology to develop innovative pharmaceuticals to treat significant medical needs. Sylentis activities are focused on the development of drugs for glaucoma, ocular pain associated to dry eye syndrome, Chron’s disease, and neurodegenerative disorders. In the short term Sylentis will focus on ocular indications and in particular in glaucoma and dry eye syndrome, indications for which the company has two compounds in clinical trials.
In addition, Sylentis has implemented a quality management system that integrates Good Manufacturing Practice (GMP). For this purpose Standard Operating Procedures (SOPs) have been established to standardize the development process from discovery to manufacturing of drugs. Implementation of this quality system certifies that Sylentis complies with national laws and international GMP guidelines.
In January 2011 Sylentis received the authorization from the Spanish Medicaments Agency (AEMPS) to manufacture investigational drugs. Sylentis received in January 2011 the brand “Madrid Excelente”. This achievement given by The Autonomous Community of Madrid recognizes and certifies a Company for its excellence in management, innovation, social awareness and its contribution to social and economical development of the region.
Sylentis will continue to seek to reinforce this patent portfolio over the time with the filing of patent applications as new inventions are generated in R&D projects.
Stages in the development of therapeutic RNAi products:
- Identify therapeutic application. This involves identifying genes responsible for a disease that must be silenced. For this there are two tracks, the physiopathologic-through knowledge of the mechanism of the disease-and drug-mimicking the action of an existing drug.
- Design of therapeutic molecule named siRNAs (sort Interference RNAs) targeting the gene silencing identified through bioinformatics tools. As explained, each siRNA was designed from a sequence complementary to the mRNA it is intended to degrade.
- In vitro validation and selection of more effective siRNAs. Different siRNAs are tested in cell cultures expressing the gene of interest, in order to determine the most effective compound.
- Development of siRNA administration mode. route of administration and dosage formulation.
- In vivo validation in appropriate animal models that mimic the disease targeted. The experiment aims to test the efficacy of siRNA in animals and is called experimental proof of concept (EPOC, its acronym in English).
- Toxicology studies in animal models. These tests are the regulatory preclinical phase, it will be the basis for requesting permission to begin human trials (IND).
- Clinical efficacy and safety study in humans. It is divided into three phases, each with its own objectives and the cost increases exponentially.