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Integrated Drug Discovery Services with Patient-Specific Disease Models for N-of-1 Solution

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. 



Rare Base Logo
Boston Childrens Hospital
Pitt Hopkins
Client 1
nof1 Labs
Lydian Accelerator

Over the past few years, iXCells has screened hundreds of antisense oligonucleotides (ASOs) for our lab and we are extremely satisfied with their service. The team at iXCells has immense expertise in the handling of iPSCs and iPS-derived neurons and delivers high-quality data in a timely fashion. The company has dedicated staff for screening who are always responsive and a pleasure to work with. We would highly recommend iXCells as a reliable partner.


Cell Model Development

Cell Model Development

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.  

RNA Therapeutics

RNA Therapeutics

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.

Patient Centricity

Patient Centricity

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.

Information Exchange

Information Exchange

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. 

Case Study 
Individualized ASO Drug Development

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.

Focused on the development of  antisense oligonucleotide (ASO) therapeutics for a patient with serious, life-threatening rare disease 
  •  Patient specific iPSC-derived neurons (cultured for 30 days) for ASO gymnosis 
  • ASO screening in 96-well cell culture plates
  • Automated sample handling using pipetting robot

  • Multiplex qPCR assay

  •  TaqMan-probe-based readout of normal and disease-specific SNPs in 384-well format

  • Screened ~1000 ASOs, identified 8 ASO candidates for hit confirmation and downstream clinical test
Figure 2a. Robust Performance and Large Screening Window using Positive and Negative Control ASO Reagents
Figure 2b. High Reproducibility Across Biologic Replicates
  1. Two test ASOs showed dose-dependent knock-down of GOI
  2. Minimal effect of Negative control ASO reagents on Target Gene across screening plates
  • Each dot represents one biological sample (average of technical triplicates)
  • All samples are normalized to the average of non-treatment samples
  • Plate 1 and Plate 2 are biological duplicate plates.
  • PC = Positive Control ASO
  • NC = Negative Control ASO

ASO Screening Using Patient-Derived Cell Model

Our comprehensive Antisense Oligonucleotides (ASOs) screening services employ human (patient-derived) cell models. The iXCells scientific team have extensive experience with ASO in vitro screening using various disease relevant cellular models, including human iPSC-derived neurons and primary cells.


Antisense Oligonucleotides (ASOs) are single-stranded deoxynucleotide analogs, usually 15–20 bp in length. Their sequence is complementary to the sense sequence of target mRNA. ASOs can be used in traditional drug discovery workflows for target identification and validation and are now being explored as therapeutics themselves.  ASO-based therapy is an active area of drug development designed to treat a variety of gene specific diseases, especially in the setting of personalized medicine and orphan diseases.



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