Therapies that regenerate organs hold the potential to stop and even reverse disease and the damage associated with aging, an exciting new paradigm in medicine. Thus, we are using multiple modalities to affect differentiation and self-renewal pathways of endogenous stem cells in situ, including expansion of terminally differentiated stem cells such as cardiomyocytes. We have advanced two programs into clinical trials: a small molecule that regenerates cartilage in osteoarthritis and a long-acting biologic that regenerates the intestinal barrier in inflammatory bowel disease (IBD). Another regenerative therapy for lung diseases is initiating clinical trials for idiopathic pulmonary fibrosis in 2024. Our earlier-stage regenerative medicine programs seek to address diseases of aging such as vision loss, heart failure and pulmonary diseases.

We are pursuing disease-modifying approaches to neurological diseases that impact hundreds of millions of people worldwide by targeting distinct pathologic mechanisms that underly these conditions. For example, we are targeting neuroinflammation to treat neurodegenerative diseases such as Parkinson’s and Alzheimer’s disease. We are also leveraging novel biological insights and our long-acting drug design platform to target new pathways implicated in neurodegeneration and psychotic disorders.

Chronic inflammation is now recognized as a destructive pathogenic mechanism central to many diverse indications and is intrinsically linked with metabolic diseases. By directly targeting inflammatory and metabolic pathways, we seek to overcome inflammation-related challenges. One of our IND-enabling programs targets the inflammatory pathways that are influential in lung diseases such as chronic pulmonary obstructive disorder and adult respiratory distress syndrome. Other programs for inflammatory diseases include a novel biologic to treat non-alcoholic steatohepatitis and a gut-retained small molecule that protects the gut epithelium from damage in inflammatory bowel disease. We also have multiple programs to treat obesity and related diseases that are built around a long-acting incretin therapy. These oral, bi-agonist, and tri-agonist therapies were enabled by our long-acting, oral peptide and antibody platforms—one of which is now undergoing IND-enabling studies.

Drug repurposing

Our ReFRAME library is the largest open-source collection of drugs that have all been approved or undergone human safety testing (~12,000 and growing). This resource enables researchers around the world to identify and repurpose known drugs for new indications, vastly accelerating the development process. Calibr-Skaggs has identified and repurposed several drugs for other indications, four of which have resulted in INDs.

​Novel DNA-Encoded Library (DEL) and small-molecule libraries

To expand our screening capabilities, we designed custom DEL libraries of billions of compounds to enable one-pot screening against high-priority targets that are difficult to find small molecule drugs for using conventional screening approaches. Combined with our expertise in developing drug screening platforms, these libraries greatly enhance our drug discovery capabilities.

Long-acting and oral platform

We have developed separate, complementary technologies that enhance half-lives of small-molecule, peptide and biologic therapies to enhance drug efficacy and reduce burden on patients. Fewer administrations also will help reduce toxicity of new medicines and costs of global health treatments. These technologies include generating depots with injectables, prodrug formulations, peptide stapling and antibody fusions, which have led to four clinical-stage programs in inflammatory bowel disease, neurodegenerative disease, HIV and malaria.

Cell-based screens/target ID

Cell-based screens and identifying druggable targets are instrumental in the drug discovery process. At Calibr-Skaggs, we have optimized cell-based screening processes, such as imaging-based and phenotypic screens, for multiple indications. We use multiple technologies to identify targets in these cells, including target deconvolution, genomics and proteomics. Leveraging our extensive ReFRAME, DEL and small molecule libraries in our cell-based screening platforms and our expertise in identifying drug targets, we can quickly screen for promising hits and launch novel drug development programs.