Lovelace, Exemplar to work on Sickle Cell Disease model under NIH contract

22-Jul-2019 - Last updated on 22-Jul-2019 at 13:54 GMT

(Image: Getty/Ca-ssis)

Lovelace’s support and the inclusion in a $20m NIH contract enable Exemplar to continue the development of a porcine animal model for Sickle Cell Disease research.

The contract research organization (CRO) Lovelace Biomedical will support four institutions, including Exemplar Genetics, a research center in Iowa, US, under a contract that the former company was awarded by the National Institute of Health’s (NIH) National Center for the Advancement of Translational Sciences (NCATS).

The $20m (€17.7) contract will see Lovelace lead the institutions’ research to create and characterize disease models for unmet medical needs, initially, Exemplar’s MiniSwine model for Sickle Cell Disease (SCD).

Exemplar, which develops animal models to mimic rare human diseases for the discovery of new treatments, aims to create a genetically modified swine that will represent the genetic and phenotypic characteristics of SCD in humans.

Following the development, the partners will characterize the disease, using the human disease as a benchmark in terms of which biological characteristics to study, and then conduct a natural history study of the swine across approximately two years.

The study will potentially result in a well understood pharmacologic model of SCD that can be used for future studies with drug intervention. Additionally, the researchers will develop an assay to measure pain in the model.

“The potential utility of an animal model with improved recapitulation of human symptoms is expected to be widely embraced by the research community,” said Christopher Rogers, Exemplar’s CSO, noting the ‘desperate need’ of the rare disease research community for better models to advance potential treatments.

ExeGen animal models

The current market for murine models is exceeding $1bn, however, small animal models often fail to fully reproduce the human disease condition.

According to Exemplar, the MiniSwine research models are more anatomically, physiologically, and genetically similar to humans than mice with regard to lung anatomy and physiology and respiratory immune system responses.

These characteristics enable their use as potent models of bacterial and viral pneumonias, while their human-like size also makes them a more suitable model for imaging studies.

Exemplar has already developed ‘ExeGen’ models for heart disease, cancer, and various rare genetic diseases that are available to researchers to work on, aiming to assist in small molecule and gene therapy research.