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In the development of cell and gene therapy products, there is a critical data gap that exists between the discovery and development phases. Every day our understanding of the modalities of advanced therapies grows, and more precise and efficient technologies exist for fully characterizing these drugs and assessing efficacy, safety, and potency while looking ahead to manufacturing readiness.
It is time to move past the idea that in the development and commercialization of cell and gene therapies ‘the process is the product.’ AmplifyBio aims to replace that saying with a new one: ‘the product is the product.‘ The addition of these capabilities to complement our in vivo preclinical work means that cell and gene therapies can now be differentiated based on safety and efficacy profiles and specific product characteristics, not development processes.
The R&D team at A-DOC is made up of scientists with broad backgrounds and expertise in the discovery, characterization, and scale up advanced therapies. Every client challenge is addressed cross-functionally by experts with experience bringing cell and gene therapy candidates from discovery to IND submission.
Bridge the gap between early R&D and CMC without in-house investment in laboratory facilities, expertise, process development, or training. Let Amplify-DOC become an extension of your lab. These are just some of the ways clients are using our services to better characterize their cell therapies:
Understanding the effect of edits to cell therapies meant to increase the potency and durability of response is crucial to successful therapeutic development. A-DOC can help understand which proteins or pathways are ideal to modulate by utilizing non-viral knock-in, knock-down, and knock-out strategies coupled with in vitro and in vivo models (at A-SET) to select the best combination of edits based on functional results.
Characterization of cell therapies from early R&D all the way through scale-up is critical to ensure that potency and mechanism of action remain unchanged. A-DOC can help by analyzing genotype and phenotype using single-cell capabilities and assessing therapy performance in a variety of functional assays. Creating a clear definition of “the product” based on multiple characteristics linked to potency and efficacy early in development ensures that process changes do not impact product function.
Editing cells has the potential to create safety risks to patients due to unintended genomic changes or simply not enough understanding of the impact of edits on the biology of the cells (e.g. potency, persistence, off-target/unintended signals). A-DOC has expertise in non-viral cell editing methods that are more specific and safer than viral editing approaches. We can help clients assess genomic stability and off-target editing using industry standard technologies. Working together with A-SET, we can generate actionable biological safety data both in vitro and in vivo to identify and mitigate safety concerns early in development to help clients create the safest therapy possible.
A-DOC can partner with clients to utilize our full TCR discovery pipeline to identify TCRs against any potential tumor antigen. The identified TCRs go through a full set of characterization assays including functional verification (e.g. T cell activation, cell killing, cytokine release, etc.), specificity, alloreactivity, and mechanism of action analysis. We can support using these TCRs in both a cell therapy format or even as a protein therapeutic (e.g. bispecific) utilizing our in-house protein science development capabilities. AmplifyBio provides a one-stop development experience because both TCR-T and TCR protein therapies can go through preclinical studies at the Amplify-SET site and can be manufactured at the A-DOC facility to ensure seamless scale-up, technical transfer, continuity in characterization approach, and IND support.
Understanding the features of different solid tumors is critical to ensuring that a given therapy has the potential to be efficacious. Tumor heterogeneity and evasion mechanisms differ across tumor types and even within the same patient. A-DOC can help clients by using bioinformatic analyses to assess tumor microenvironment (TME) features, resident and infiltrating cell types, tumor-associated and neoantigens present, and tumor evasion features. These tools can help identify the best in vitro and in vivo models for therapy characterization at both A-DOC and A-SET to ensure each therapy has the best chance to be successful.
Understanding a cell therapy product does not end once a patient is dosed. To fully understand whether the therapeutic hypothesis is valid, it is critical to monitor the pharmacokinetics and pharmacodynamics of the drug in humans. A-DOC and A-SET teams have tools to help clients understand the distribution and function of their therapies including tracking cells in the blood and tumor, assessing changes in the tumor architecture and microenvironment based on therapy pressure, and characterizing changes in the dosed cells and tumor at the protein and genome levels.
With the expertise to design the right study for every model and flexibility for data-driven implementation, AmplifyBio is your partner to trust for Preclinical Contract Research Organization Services. Talk with us and discover how we can support your study and compliance requirements.
