Technology
Silencing the gene
without rewriting the DNA:
how siRNA technology works
PHYSIOLOGICAL CONDITIONS
Under physiological conditions, healthy genes are correctly expressed, leading to the production of proteins necessary for normal cellular function
PHYSIOLOGICAL CONDITIONS
Under physiological conditions, healthy genes are correctly expressed, leading to the production of proteins necessary for normal cellular function
GENETIC DISEASES
In genetic diseases, a DNA mutation can disrupt this balance, leading to the production of defective or harmful proteins that drive the pathology.
GENETIC DISEASES
In genetic diseases, a DNA mutation can disrupt this balance, leading to the production of defective or harmful proteins that drive the pathology.
HOW siRNA SILENCES MUTATED GENES
siRNA (small interfering RNA) acts after gene transcription, at the messenger RNA level, without altering the DNA sequence. It selectively silences the message from the mutated gene, preventing the production of the pathological protein
The result is a targeted and highly specific silencing of the defective gene, while the healthy gene continues to perform its normal function. For this reason, siRNA represents one of the most promising strategies for treating various rare genetic diseases
HOW siRNA SILENCES MUTATED GENES
siRNA (small interfering RNA) acts after gene transcription, at the messenger RNA level, without altering the DNA sequence. It selectively silences the message from the mutated gene, preventing the production of the pathological protein
The result is a targeted and highly specific silencing of the defective gene, while the healthy gene continues to perform its normal function. For this reason, siRNA represents one of the most promising strategies for treating various rare genetic diseases
Silthera Platform
and the data-to-drug approach
The Silthera Platform is Silthera’s proprietary system, developed to accelerate the discovery of siRNA-based therapies for rare genetic diseases.
It integrates bioinformatics, artificial intelligence and biological experimentation into a single system, with the aim of transforming genetic and molecular data into designed candidates more rapidly and with greater precision.
Using a data-to-drug approach, the platform interprets complex biological data through advanced computational models and translates them into therapeutic programmes, reducing time, complexity and risk compared to traditional research and development processes.
01
Data Input
Relevant genetic and molecular data are collected and analysed
02
Data Processing
The data is cleaned, standardised and filtered to ensure the quality, consistency and reliability of the model
03
siRNA Design
The platform designs and optimises targeted siRNA sequences using machine learning modules
04
Molecule
selection
The molecules with the best profile are shortlisted for the next stages of evaluation
05
Preclinical Test Simulation
Candidates are tested virtually (in silico) to estimate efficacy, safety, and the risk of adverse effects (off-target)
06
Laboratory
validation
Only the most promising molecules proceed to experimental validation, where the computational results are verified in the laboratory
A scientific mission with a tangible impact on people
Behind every genetic mutation are individuals, families, and complex care journeys. Silthera was born from listening to patients, caregivers, and advocacy groups who live every day with diseases for which therapeutic options remain limited.