Oligonucleotide therapeutic development is complex. Certara pioneered support for the first FDA approval of an oligonucleotide therapy. Our team continues to bring value to the ever-expanding list of Oligonucleotide therapy programs by leveraging our expertise and experience within Clinical Pharmacology, Pharmacometrics, Regulatory Support, and Health Technology Assessments.
In the last 3 years, Certara supported customer success of 23 Oligonucleotides programs by:
- Leveraging in-silico clinical trial modeling using applicable data to predict clinical outcomes.
- Optimizing the clinical pharmacology drug label claims.
- Incorporating Health Economic Outcomes research and strategies to ensure proper market access alignment in a competitive landscape
“Whether you are developing RNAi, siRNA, miRNA or antisense oligos, Certara has experience/success developing these molecules”
- Example 1: The client needed to understand and determine the fate and delivery of their siRNA therapy, which was challenging due to its rapid degradation. We determined the organ of specific delivery and uptake, which was also the site of efficacy for the indication. We also developed models to assess the biomarker accumulation following repeated dosing. CTA: Learn More
- Example 2: The client sought to develop an oligonucleotide therapy that would knock down a key protein implicated in the pathogenesis of a neurological disorder. They needed to link plasma drug dosing with the resulting exposure and efficacy. Certara scientists developed PK & PK/PD Models to perform simulations to determine the dosages associated with optimal clinical outcomes. CTA: Contact Us
- Example 3: The client was struggling to correlate RNAi plasma exposure with tissue exposure and target engagement in their pre-clinical animal models. Certara’s team designed a series of animal studies with a fusion protein conjugated to a mAb to bolster the data sets used to build a PK/PD model for predicting target engagement from limited plasma data.
Complex Biologics include therapeutic modalities such as oligonucleotide therapies, monoclonal antibodies, engineered proteins and antibody fragments, bi-specific platforms, T-cell directed therapies, chimeric antigen receptor T-cell (CAR-T) therapies, antibody-drug conjugates, vaccines, gene therapies, and more. Because of their complex pharmacology, complex biologics tend to face additional challenges in clinical development and navigating regulatory pathways. At Certara, we have the expertise and experience to help our clients navigate both pre-clinical and clinical development for various complex biologic treatment modalities.