At iXCells, we believe everyone deserves a cure no matter how rare the disease is.
Our researchers and scientists combined in-depth understanding of cell engineering and RNA therapeutics are dedicated to help you accelerate your drug development, including individualized medicines. Our customized preclinical CRO services include patient iPSC reprogramming, cell line engineering, iPSC-neuron differentiation, and ASO / siRNA screening.
We help patients in the race against time. Our relationship with rare patients advocacies allow us to understand the uniqueness of each ultra-rare case and streamline our business logistics. With a patient-centric research support, we also help our customers and partners to exchange their experiences and resources during their journey in seeking the cure for rare diseases.
Together we can turn the impossible into miracles.
As the cell model expert, we are specialized in a variety of disease-relevant cell systems, including immortalized cell lines and iPSC-derived models. We help generate patient-specific iPSC lines and iPSC-neurons which are valuable tools to shorten your time in developing treatments for epilepsy and other neuronal disorders.
We have intensive experience in assay development, including gymnosis and transfection optimization in different cell systems. Our experience encompasses various clinical-grade ASO technologies including 2’MOE-, 2’O-Me-, LNA- and cEt-modified gapmer and steric blocking ASOs.
We understand each rare case is unique. Our patient-centric services enable foundation leaders and research partners to establish their preclinical studies based on disease progression, mode-of-action information, POC results, financial sources, etc.
We believe that the joined forces of researchers, physicians, patient advocacy and regulatory professionals are critical to tackle rare diseases. We create a platform for our clients and partners share their experiences / resources and establish best practices along their journey in fighting devastating diseases.
Focused on the development of antisense oligonucleotide (ASO) therapeutics for a patient with serious, life-threatening rare disease
Our team of experts helped a patient with a rare genetic neurological disorder for preclinical drug development. This patient carries an ultra-rare mutation in one of the ion channel proteins, and the allele-specific antisense oligonucleotides (ASOs) were designed to target the mutant mRNA. Patient-specific iPSC-derived neurons were generated by iXCells and used for high throughput screening. ~1000 ASOs were tested at two concentrations in the primary screen, and 8 of them were selected for hit confirmation and downstream clinical test.
