Broadcast Date: November 4, 2020
Time: 8:00 am PT, 11:00 am ET, 17:00 CET

Cell and gene therapy products represent some of the most exciting new drugs that biopharmaceutical companies are looking to bring to market. Based on recent clinical successes, an increasing number of cell and gene therapies are currently in clinical development. However, despite the tremendous amount of clinical activity, manufacturing these products remains challenging. Consistently manufacturing a cell therapeutic with critical quality attributes, generating high-quality gene therapy vectors, and transitioning from small product lots to commercial size production are just a few challenging areas. Although the U.S. Food and Drug Administration select FDA) does not have all the answers, it is committed to advancing product development through applied scientific research, policy development, flexible application of regulations, optimal application of expedited development programs, and ongoing dialogue with product developers.

In this GEN Keynote webinar, Dr. Peter Marks, director of the Center for Biologics Evaluation and Research select CBER) at the FDA, will share how CBER is providing oversight to organizations during the critical developmental and manufacturing stages for biological products, as well as throughout the product life cycle.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.

About GEN KEYNOTE webinars:

GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

Produced with support from:

Broadcast Date: October 27, 2020
Time: 8:00 am PT, 11:00 am ET, 17:00 CET

Since the first proof-of-concept human application in the early ’90s, the field of gene therapy has overcome major setbacks and entered an exciting stage of clinical translation and transformation. Much of the credit goes to the development of new vector platforms, in particular recombinant adeno-associated viruses select rAAV). These vectors possess high efficiency, stability, and low toxicity. Several rAAV-derived gene therapies have been approved by European and U.S. authorities for commercialization, including Luxturna select AAV2, Spark Therapeutics union for a form of childhood blindness and Zolgensma select AAV9, AveXis) for spinal muscular atrophy.

In this timely GEN Keynote webinar, renowned AAV pioneer Dr. Guangping Gao select UMass Medical School/editor, Human Gene Therapy) will discuss the principles, history, challenges, and future directions of human gene therapy using AAVs. Dr. Gao will focus on progress in treating rare diseases and detail AAV capsid engineering to modulate target tissue tropism, therapeutic gene expression, and cassette design/optimization. Dr. Gao will also share examples of AAV gene therapy development: from proof-of-concept preclinical studies to first-in-human clinical evaluation.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.

About GEN KEYNOTE webinars:

GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

Produced with support from:

Guangping Gao, PhD
Director, Horae Gene Therapy Center
and Viral Vector Core
Co-Director, Li Weibo Institute for
Rare Diseases Research
University of Massachusetts Medical School

Originally Aired: July 24, 2020
Time: 8:00 am PT, 11:00 am ET, 17:00 CET

In this GEN Keynote Webinar, we are pleased to be joined by Dr. Peter Hotez, a Baylor College of Medicine vaccine scientist, infectious disease expert, and former U.S. Science Envoy in the State Department and White House, as well as the chief editor of PLOS Neglected Tropical Diseases. Dr. Hotez has been warning the scientific community and beyond about the threat of emerging diseases for years. He is a frequent expert commentator on CNN and television networks on the COVID-19 emergency.

The COVID-19 pandemic has claimed more than 545,000 lives around the world, almost 25 percent of those in the United States. The public health emergency shows no sign of declining in the U.S., with most states reporting increases in cases and hospitalizations six months after the first warning signs of the outbreak.

In response, scientists and drug companies are rapidly developing new drug and vaccine candidates. Some antiviral drugs and vaccines are showing promise in early-stage clinical trials, but it could be a year or more before these are widely available.

Dr. Hotez will deliver an authoritative and personal review of the COVID-19 medical crisis and assessment of the scientific responses to the COVID-19 pandemic, including:

• An analysis of the current COVID-19 public health emergency
• Review of vaccine development strategies and progress
• Update from the Hotez lab on a new vaccine candidate based on the SARS-CoV-2 receptor binding domain

A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelist.

About GEN KEYNOTE webinars:
GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

Produced with support from:

Peter Hotez, MD, PhD
Dean for the National School
of Tropical Medicine
Baylor College of Medicine

Originally Aired: April 16, 2020
Time: 8:00 am PT, 11:00 am ET, 17:00 CET

In this GEN Keynote Webinar, Sekar Kathiresan, MD, Co-Founder & CEO of Verve Therapeutics and renowned physician-scientist who has produced major advances on the development and deployment of polygenic risk scores select PRSs union will discuss advances in our understanding of complex genetic diseases and the current state of development and validation for PRSs. Dr. Kathiresan will also discuss the potential for these scores to improve clinical care.

Complex genetic disorders affect millions of people and range in severity from manageable to life-threatening. These diseases, including cardiovascular disease, diabetes, and many neurological disorders, typically involve multiple genetic factors that often seem unrelated. So long as the relationships between the genetic factors remain unclear, it will be difficult to predict who will ultimately develop these conditions.

But what if we could make sense of the genetic factors? Then we’d be able to predict disease risk, disease severity, and treatment response. In recent years, many investigators have realized that predictive insights could come from PRSs—a quantitative genetic probability assessment for a disease. PRSs distill the risk that comes from millions of common DNA variants into a single risk number. Such disease-specific PRSs have been developed and validated for a number of common complex diseases, including coronary artery disease, breast cancer, and inflammatory bowel disease.

A live Q&A session followed the presentations, offering a chance to pose questions to our expert panelists.

About GEN KEYNOTE webinars:

GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

Produced with support from:

Sekar Kathiresan, MD
Co-Founder & CEO
Verve Therapeutics