SUNDAY, MAY 4
Morning Course | 10:00 am - 1:00 pm
Proteins and peptides represent a significant segment of the therapeutics spectrum with many promising candidates under early development or in late-stage clinical trials. Several of these molecules are poised to make a substantial impact, especially in the under-represented or unrepresented categories such as neurological disorders and neurodegenerative diseases. A key challenge to be overcome with protein and peptide based biologics, however, is their effective delivery to the target site while achieving the optimum balance of stability, safety, bioavailability, and patient compliance. This short course will provide a broad overview of the opportunities and challenges in the development of the next generation of protein and peptide therapeutic delivery systems.
Topics to be covered:
Therapeutic spectrum
- Introduction to biologics based on proteins and peptides
- Different classes of protein and peptide biologics
- Representative examples in clinical use
- New molecules under development – next-generation biologics
- Clinical needs and opportunities
- Clinical advantages of biologics over small molecules
- Current niche served by protein and peptide biologics
- Unrepresented clinical indications and opportunities
- Key challenges in protein and peptide delivery
- Issues related to stability
- Route of administration
- Biological barriers (e.g. blood-brain barrier)
- Bioavailability for therapeutic benefit
- Safety/toxicity balance
New developments in enhancing protein stability
- Use of biostable/synthetic scaffolds
- Chemical modifications
- Packaging in nano and mesoporous carriers
- Molecular chaperones for enhanced serum stability
New developments in overcoming delivery challenges
- Integration of delivery vectors with biologics
- Protein/peptide API-based nanomaterials
- Alternate routes/interfaces for administration
- Transgenic delivery systems
Future directions in protein and peptide delivery
- New clinical indications
- Novel delivery systems
- New routes of administration
Instructor:
Pankaj Karande, Ph.D., Assistant Professor, Department of Chemical & Biological Engineering, Center for Biotechnology and Interdisciplinary Studies, Rensselaer Polytechnic Institute - Biography
Afternoon Course | 2:00 - 5:00 pm
The discussion will focus on non-injectable technologies such as nasal, pulmonary, and oral delivery technologies, and the challenges and opportunities in development for converting these technologies into successful products. In addition, the discussion will address the category of technologies sometimes referred to as minimally-invasive that has the potential to significantly improve the patient experience. Technologies in development in this area will be discussed along with the pros and cons. Finally, a number of commercial examples will be reviewed as case studies where non-invasive or minimally invasive technologies have been successfully commercialized.
Topics to be covered:
When and where to introduce delivery into the product development pipeline
Technology and strategy selection and implementation
Opportunities in non-invasive and minimally invasive technology approaches for delivering biologics
- Pulmonary inhalation
- Nasal spray
- Oral delivery systems
- Implants
Emerging approaches using delivery formulation and device strategies
- Microneedles
- Active transdermal systems
- Oral device delivery
Effect of various technologies on drug product stability and deliverability. What are the strategies and tools for assessment?
Case studies of late stage development programs and commercial product examples such as Zosano PTH microneedle patch in Phase III, Exubera insulin inhalation, Lupron depot yearly implant, FluMist nasal spray vaccine
Instructors:
Christopher A. Rhodes, Ph.D., President, Christopher A. Rhodes & Associates LLC - Biography
Frank Tagliaferri, Ph.D., Vice President, R&D, 4P Therapeutics - Biography
Tuesday, May 6 | 6:00 - 9:00 pm
Topics to be covered:
Why are high concentration dosage forms increasingly becoming important?
What are the challenges of high doses? What factors define them?
Viscosity limitations in production and delivery
Current state of the art with devices for high-volume injections
- Current understanding of what causes high viscosity in mAbs
- Studies probing underlying molecular mechanisms
- Engineering high viscosity causing molecules
- High-throughput (HT) methods for measuring viscosity: Ensuring accuracy and precision
- HT screening tools for predicting viscosity
- Excipients for reducing viscosity
- Aggregation in high-concentration formulations
- Temperature dependence of viscosity
Fill/finish challenges
Alternate methods of producing high concentration MAb solutions
Instructors:
Christopher M. Olsen, Ph.D., Senior Scientist, CMC Analytical Chemistry, Alcon Laboratories, Inc., a Division of Novartis - Biography
Nicholas J. Darton, Ph.D., Platform Manager, Arecor Ltd. - Biography
* Separate Registration Required