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How to save $1 billion on drug development

Executing clinical studies during the pandemic has been challenging. However, innovations such as telehealth and decentralized clinical studies with access to liquid biopsies that can be collected at home offer a new paradigm for the future.

Genomic Expression has developed OneRNA®; a novel RNA Platform for Next-Generation Cures, that we can leverage to inspire, Insilco model, then design, stratify, and ultimately de-risk clinical studies.

Implementing biomarkers can save drug developers + $1 billion

Clinical drug development is risky and costly; however, using biomarkers that inform response-and-resistance mechanism, patient selection, and minimal residual disease can greatly reduce risk, size, and length of study and, therefore, cost.

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Biomarkers are going digital

Historically, biomarkers have been a protein or nucleic acid that was identified by staining tissue or analyzing blood using old analog technologies that could only analyze one biomarker at a time.

The analog and binary relationship between a single drug with a single biomarker have changed dramatically with the implementation of Next Generation Sequencing (NGS), which is able to detect many biomarkers at the same cost but provides much more information.

Thus the paradigm of ONE disease and ONE drug has changed as drugs in oncology are now approved for multiple cancers, and NGS assays that detect multiple biomarkers and thereby guide multiple treatment options are FDA-approved.

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Both developments are moving drug discovery and development into the digital era, where the biomarker ultimately becomes an algorithm.

RNAai signatures using our OneRNA® platform as a CDx option

Prognostic biomarker algorithms based on RNA are already implemented into standard of care, e.g., in breast cancer in the form of a multiplex PCR assay.

OncoTypeDx incorporates the quantitative analysis of 16 RNA transcripts and clinical data to produce a treatment recommendation in early-stage breast cancer patients, thereby guiding treatment decisions for patients who don’t need additional toxic chemotherapy treatment.

Thus, the use of algorithms based on RNA expression is not new; in fact, a long list of these prognostic algorithms is used today in standard of care.

OneRNA® offer drug developers the opportunity to develop an RNA algorithm as a companion diagnostic option, further de-risking studies by eliminating the need for reengineering assays and sub-optimal and risky reliance on a small subset of biomarkers.

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Decentralized clinical studies and self-collected liquid biopsy sample specimens

80% of clinical studies fail due to a lack of enrollment. Delaying enrollment ultimately delays launch and can cost the sponsor its first-mover position, revenue, and market share.

Decentralization broadens trial access to reach a larger, and potentially more diverse, pool of patients—a new requirement of the FDA.

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Both saliva and urine are underutilized sample specimen types, and RNA is an underutilized biomarker species that can provide much more information on the etiology of the disease.

Unfortunately, RNA is not a stable biomarker and, therefore, precautions need to be taken to stabilize the biomarker at the time of collection.

About Genomic Expression 

Genomic Expression is operating as a CLIA lab located in MA. We developed OneRNA®, a quantitative clinically validated RNA platform for Next Generation Cures because, by analyzing RNA, we can detect disease, monitor health, and design next-generation cures.

We clinically validated RNA biomarkers in self-collected samples, such as saliva and urine, which can be collected in patient homes.

Finally, we validated collection devices, instructions, and reagents that stabilize nucleic acids and proteins in the sample at the time of collection, as well as a backend HIPAA-compliant platform that tracks everything from order, through shipping and lab processing, to delivery of results directly to patients (optional) or to the ordering physician/sponsor.

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We originally focused on oncology first because of access to tissue samples; however, the platform itself is tissue and disease agnostic.

It is our opinion that RNA is an underutilized biomarker species for some well-understood reasons; RNA is not stable and its analysis is complicated and quantitative, adding a significant level of complexity.

It is also our opinion that saliva and urine are underutilized sample specimen types that can be leveraged together with Telemedicine to provide evidence-based care in the comfort of the patient's home.

We have overcome the key hurdles in bringing RNAseq into clinical practice, and we have thereby enabled clinical-grade RNAai signatures using our OneRNA platform as a CDx option in tissue as well as in self-collected liquid biopsies.

We also believe that such a platform can further dramatically de-risk and accelerate drug discovery and development, as well as improve the basic understanding of the disease etiology.

For more information go to http://www.genomicexpression.com/