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Enhancing Cell & Gene Therapy Discovery With In Vivo Imaging Biomarkers

This blog post was written in partnership with Invicro, a subsidiary of REALM IDx and an industry leading imaging CRO and analytics solution provider that supports biopharma companies across all stages of the drug development pipeline from preclinical through Phase 0-IV.

The field of cell and gene therapy has witnessed remarkable advancements, promising revolutionary treatments for various diseases such as genetic disorders and cancer. However, realizing their full potential necessitates overcoming challenges, including optimizing delivery platform biodistribution and transduction profiles, as well as establishing robust pharmacodynamic (PD) biomarkers that can be used to stratify and/or assess efficacy in early clinical studies. To these ends, in vivo imaging provides powerful tools that enable valuable, quantitative insights into cell and gene therapy programs.

Empowering Real-time Insights into Biodistribution

Biodistribution analysis involves investigating the ability of cell and gene therapies to reach and transduce their intended target tissues, at the right time, and is crucial for predicting efficacy and potential off-target effects. Imaging techniques, such as in vivo fluorescence imaging (FLI), cryo-fluorescence tomography (CFT), positron emission tomography (PET) and single-photon emission computed tomography (SPECT), offer invaluable solutions for assessing cell and gene therapy platform biodistribution.

For assessment of delivery vehicle distribution, radioisotopes or fluorescent reporters can be used. Imaging labels that are readily attached to common gene therapy agents enable researchers to precisely track their journey within the body as it happens. This allows monitoring of the distribution of these agents, ensuring they reach the intended target tissues efficiently and are excreted in a manner that aligns with target safety profiles. Such insights are crucial for optimizing delivery, efficacy and improving treatment outcomes.

Enhancing Transduction Efficiency Insights

Gene delivery relies on the efficient and optimized delivery of the intended genetic material into target cells. This transduction, downstream of agent biodistribution, can be readily assessed using imaging reporter genes such as luciferase (BLI), GFP/RFP (FLI or CFT) or PET imaging reporter genes, which are seeing successful clinical translation and can be used with relatively simple 11C or 18F small molecule substrates.

There can be various barriers or challenges to successful transduction, such as inefficient delivery to target cells, limitations in the uptake of genetic material or issues with the stability of the therapeutic vector. In vivo imaging accelerates the development of gene therapies by allowing scientists to fine-tune vectors and delivery methods for maximum effectiveness. The power of such imaging can be even stronger through the use of radiochemistry and translational imaging via PET or SPECT.

Biomarkers for Assessment of PD and Efficacy

As for other therapies, effective cell and gene therapy screening, candidate selection and first-in-human translation relies on the identification and validation of robust PD biomarkers that provide indications of treatment efficacy and safety, as early as possible after treatment administration. In vivo imaging, including both nuclear medicine and molecular approaches, equips researchers to longitudinally evaluate treatment responses over time and more effectively select and stratify patient populations in clinical translation.

These approaches provide essential data for refining therapy protocols and demonstrating therapeutic benefits in preclinical and clinical studies. Examples of gene therapy-based imaging biomarkers that have seen outstanding results at Invicro include noninvasive quantitative imaging of cardiac function, brain metabolism and bone morphology across a number of rare genetic diseases such as Canavan’s, Hunter’s, Friedrich’s ataxia and Pompe disease.

Invicro: Your Partner in Cell & Gene Therapy Imaging

Execution of cell and gene therapy imaging studies requires the skills of many scientific disciplines and the expertise to enable the coordination of all facets of complex experiments, including animal models, radiolabeled test agents, longitudinal assays and multi-modal imaging. As a leading imaging CRO, Invicro works collaboratively with our partners preclinically and clinically across a diverse range of technologies including molecular and functional imaging, and offers solutions across a full spectrum of animal species, routes of administration and vector types. With industry-leading expertise and operational excellence, we answer complex research questions and enable our partners to make better go/no-go decisions – faster.

About Invicro

Invicro is an integral part of the REALM IDx, Inc. group, which aims to accelerate personalized medicine, discover novel therapeutic targets and develop innovative therapeutic technologies for unmet medical needs.

As a global research partner to the pharmaceutical, biotech and contract research organizations, Invicro helps to enhance the discovery and development of life-changing drugs, using a novel platform that brings together best-in-class expertise, imaging biomarkers, core lab services, analytics and software. We answer questions that support more timely and accurate decision-making throughout the development process, from pre-clinical research to late-stage trials.

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