This presentation is co-presented with Angela Bezigos, CEO, Touchstone Technologies, Inc.

Gene therapy is an experimental technique to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of relying on drugs or surgery. Several approaches to gene therapy include:
• Replacing a mutated gene that causes disease with a healthy copy of the gene
• Inactivating, or “knocking out,” a mutated gene that is functioning improperly
• Introducing a new gene into the body to help fight a disease

Although gene therapy is a promising treatment option for diseases including selected inherited disorders, cancers, and viral infections, safety and effectiveness remain a concern. Gene therapy is currently being tested for diseases that have no other cures.

This session will describe the regulatory requirements for gene therapy, how genetic information is transferred into target cells, the significance of vectors and how the latest technologies such as genetic engineering, recombinant DNA technique and mRNA technology can be utilized to improve performance of gene therapy in the treatment of disease.

Objectives:
• Describe major pharmacy compounding adverse events which led to the birth of Drug Quality and Security Act (DQSA)
• Discuss 503A and 503B regulations as they relate to human drug compounding
• Compare and contrast 503A and 503B regulations as they relate to expanded pharmacist training and practice

Virtual FDA Town Hall, (all speakers invited)
Chrissy Cochran, Ph.D., Director, FDA-Office of Bioresearch Monitoring Operations,
Eric Pittman, FDA-Program Division Director, FDA and Industry Speakers, all attendees

Wearables and handheld devices (watches, glasses, smartphones, etc.) can track everything about you. Software is becoming a medical device more-and-more with Artificial Intelligence and Machine Learning. The Internet of Things (IoT) is connecting everything. Is this safe? What if your personal and private information gets into the wrong hands? What if there’s a breach? Although technologies show promise, they also raise risk; ;in fact, technology outpaces ethics. How do we leverage technology without sacrificing ourselves? This presentation will discuss the exciting new technologies, the risks, how we can protect ourselves, our data, and move forward cautiously with safety at the forefront.

In 2005, then FDA Commissioner Dr. Janet Woodcock articulated USFDA’s vision for 21st century pharmaceutical manufacturing as “A maximally efficient, agile, flexible pharmaceutical manufacturing sector that reliably produces high-quality drugs without extensive regulatory oversight.” Since then, giant strides in manufacturing technology and control system architectures to include Pharma 4.0, S88, S95, ERP systems have reliably served as enablers for advanced manufacturing.
In order to sustain the therapies for the personalized medicine era, evolving automated process control system technologies and architectures serve as enablers of adaptive manufacturing. It requires smaller batch sizes with frequent switchovers that should be timely and cost-effective.
Manufacturing of life science products being the lifeblood of life sciences industry, the presentation will enumerate manufacturing control software design leveraging evolving software technologies and computing architectures in life sciences manufacturing. Such a design also facilitates the integration of manufacturing and business functions across several control IT platforms. Highlighted will be the use of Pharma 4.0 architecture, S88 and S95 software modular design standards and CSA validation methodology to realize “Validation by Design”.

2020 helped change the perception of pediatric health and technology and resulted in innumerable breakthroughs and innovations across various fields. This is the right time for a big push in pediatric data science, innovation, medical devices, and technology. Not only could pediatric medical devices, data sets, and systems fill gaps in-hospital care, but they also help monitor children’s health and keep them out of the hospital in the first place. Dr. Terence Sanger, MD, Ph.D. will discuss why pediatric innovation has lagged that of adults, how to identify and implement technologies that significantly improve pediatric care and are most viable for clinical use and commercialization and why data science and Real-World Evidence (RWE) is important for regulatory decision-making and cutting-edge insights

Virtual FDA Town Hall (all speakers invited)
Chrissy Cochran, Ph.D., Director, FDA-Office of Bioresearch Monitoring Operations,
Eric Pittman, FDA-Program Division Director, OBIMO W, FDA Office of Enforcement and Import Operations