Form and Formulation Development for Novel and Unmet Medical Needs

Global health, rare diseases, and other unmet medical needs have unique challenges that can be met by an exciting range of new modalities. This GRC brings together expertise and unpublished data on small & large molecular drug candidates and gene therapies to highlight the form and formulation challenges and opportunities that span these modalities and integrate cross-cutting techniques and scientific approaches.

Novel small molecules with features previously only possible in large molecules will be covered. These include bispecific small molecules, such as Proteolysis-Targeting Chimeras (PROTACs) designed to target intracellular disease-causing proteins for degradation, which are an entirely new approach to small molecule drug discovery with regards to target amplification and inhibition. In addition, the production and development of novel biologics systems will be covered. Topics including gene and microbial therapies for cancer and other applications. Specifically the meeting will cover bacteria engineered for treatment of solid poorly vascularized tumors, using the microbiome for therapeutic purposes, new approaches to and delivery routes for preclinical and clinical gene therapy, and personalized medicines to precisely tailor the treatment to the patient, all having the potential to revolutionize the field. The conference will also touch on the use of synthetic biology and directed evolution to produce innovative therapeutics inspired by both nature and technology, with recent advances in producing protein and nucleic acid biologics.

These medicines do not look anything like traditional, immediate release small molecules intended for extracellular targets and delivered through solid-oral dosage forms and, therefore, have unique challenges with respect to form, formulation, and characterization. This GRC will aim to illuminate technical solutions that cover formulation development and characterization of novel molecules, including cutting edge artificial-intelligence/modeling approaches, the latest characterization technologies that are applicable to a range of modalities, and drug delivery systems that can be used to get the active reagent to its site of action in the body. The COVID pandemic has illustrated the importance of drug product manufacturing and the agenda will therefore also include a manufacturing track covering diverse, scalable and flexible manufacturing approaches across several drug modalities.

Form and formulation can also enable solutions to critical global health challenges. This GRC will sound the call for action to address these very different form and formulation needs, including WHO zone 4 stability requirements, low cost-of-goods-sold, easy to administer formulation and delivery systems, and distribution in remote areas of the globe.

This conference aims to share successes, challenges, and lessons learned from academic, industrial, government, and non-profit scientists to meet the needs of patients across the world.