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 This GRC brings together expertise and unpublished data on small, large and cell-based drugs to highlight the form and formulation challenges and opportunities that span these modalities and integrate cross-cutting techniques and 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 a whole 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 cell and gene therapies and microbial cancer therapies, including oncolytic viruses for treatment of solid poorly vascularized tumors and personalized medicines to precisely tailor the treatment to the patient, all having the prospective to revolutionize the field. The conference will also touch on the use of synthetic biology 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
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.
The conference will consist of nine sessions, on the topics listed below. The conference chair is currently developing their preliminary program, which will include the names of the invited speakers and discussion leaders for each of these sessions. Please check back regularly for updates to this information.
- Keynote Session: Gaps in Disease Treatment Impacting Large and Small Patient Populations
- Innovative Formulation Strategies for Complex Molecules
- Alternate Delivery of Gene Based Therapies
- Characterization Technologies Bridging Small and Large Molecules
- Artificial Intelligence and Other In Silico Methodologies
- Design and Characterization of Microbial Therapies
- Solubility and Permeability Challenges for Beyond the Rule of 5 Molecules
- Novel Manufacturing and Formulations for Therapies of One to Billions
- Drug Delivery and Manufacturing of Global Health Medicines