• Discover the best approach for developing a cryopreservation strategy
• Discuss the principles of cryopreservation as learned from immune cells, and stem
• Learn more about developing cryopreservation reagents and protocols for cell & gene therapy manufacturability
• Understand the criticality of source materials on the quality of the final product

Speaker:

Robert Newman, PhD
Chief Scientific Officer
FUJIFILM Irvine Scientific (CA, USA)

Commercializing a new cell therapy drug is complex. Do it right and you are well positioned for commercialization and scaling to meet demand. Do it poorly and you risk delays, confusion and budget overruns. Join Point B to learn about some of the common pitfalls of commercialization and the lessons learned about critical operational readiness indicators from their work on autologous cell therapy launches.

Speakers:

Susan Michl
Principal, Cell & Gene Therapy, Life Science Practice
Point B, LLC

Cheryl Wolf
Principal, Cell & Gene Therapy, Life Science Practice
Point B, LLC

In vivo gene therapies, using viral vectors based on AAV as vehicles to directly deliver genetic material to the patient´s body, are increasingly successful. Initially, novel gene therapies were directed towards rare orphan disease, but now they are targeting a growing number of common diseases including Parkinson´s, Alzheimer´s and Rheumatoid Arthritis.

This causes major challenges in manufacturing sufficient amounts of AAV vectors, since the classical production methods, originally developed in academic settings, are far from robust and scalable. The presentation will present the latest developments in using stable cell lines as producer cells and explain how the ELEVECTA® system enables fully scalable AAV vector production without plasmid transfection or helper viruses.

Speaker:

Dr. Nicole Faust
CEO 
CEVEC Pharmaceuticals

Adeno-associated virus (AAV) is one of the three major viral vectors used in clinical trials for gene therapies and is required at high titres and at high quality. At OXGENE and WuXi Advanced Therapies, we aim to revolutionize AAV manufacture in two ways. First, by developing and optimizing transient expression systems and manufacturing platforms. Second, by implementing innovative plasmid-free manufacturing systems. Tetracycline-Enabled Self-Silencing Adenovirus (TESSA™) vectors contain all necessary components for AAV manufacture but can be used at scales of up to 2000L.

In this webinar, you will learn:

• Challenges involved with scalable AAV plasmid production
• How engineering of the rep and cap genes improves transient AAV production
• The development of a robust and scalable AAV suspension transient expression system
• How TESSA™ technology can yield more than 10-fold more AAV vectors and produce particles which are 5- to 60-fold more infectious compared with traditional approaches

Speaker:

Yiyu Dong, PhD
Associate Director of Viral Vector Process Technology Development (VVPTD)
WuXi AppTec

Viral safety and risks for cell and gene therapy, how to improve pathogen safety of cell and gene therapy.

Speaker:

Daniel Strauss, Ph.D.
R&D Director
Asahi Kasei Bioprocess America, Inc.

Adeno associated viral vector (AAV) is the delivery vehicle of choice for potentially curative in vivo gene therapy. Following a couple of landmark approvals, this field has experienced accelerated pipeline growth and investor interest in the past five years. Currently AAV therapies are predominantly targeting rare genetic disorders for which the patient population is often limited. As a result, drug developers feel immense pressure to be first to market to commercialize their therapies.

In this Webcast, Lonza CGT will focus on a robust, streamlined platform process for fast production of AAV using Lonza’s clonal suspension HEK293 cell line, proprietary plasmids to ensure high productivity and in process analytics enabling enhanced full to empty capsid ratio. We will further discuss Lonza’s approach for building reliable, de-risked path to clinic to avoid unforeseen costs and compliance-related delays.

Educational value:

Speed to clinic with a de-risked, robust, scalable platform process for AAV production High AAV productivity and yield with Lonza’s proprietary plasmids (Rep/cap + pHelper) and clonal HEK293 cell line Full/empty analytics enabling USP and DSP optimization at early stages/small scale Pre-qualified platform assays

Speaker:

Suparna Sanyal
Head of Viral Vectors Commercial Development
Lonza

Biolayer Interferometry has established itself as an easy, fast, accurate and cost effective method for quantitation and kinetics measurement of proteins, antibodies, peptides and small molecules. Recently, a BLI solution for quantitation of multiple AAV serotypes using CapSelect AAVX nanobody probes was announced and getting rapid acceptance by the gene therapy researchers and AAV manufacturers.

However, easy and accurate measurement of empty/full capsid ratio remains challenging. Here we present a novel method that utilizes the affinity capture followed by a total ssDNA measurement using GatorPrime BLI system. The method is capable of quantitation of different serotype capsids and determination of the empty/full ratios on a single easy-to-use automated BLI platform

Speaker:

Robert Zuk
CTO
Gator Bio

Robust analytics are critical for process monitoring and assurance of product quality. This talk with review strategies for the identification and monitoring of Critical Quality Attributes (CQA) in the context of gene therapies and will provide a general overview of common methods used for CQA monitoring for gene therapy products.

Speaker:

Lyndi Rice
Head of Gene Therapy Analytical
BioMarin Pharmaceutical Inc.

As the demand for cost-effective cell therapies increases, managing the complexity of cell culture is critical to success. For process developers, one aspect that’s critical is to establish key starting materials from reliable suppliers early on. Selecting the right materials can help reduce costs and support seamless process transfer into clinical development. Join our session to learn how to establish a reliable supply chain of quality materials that can help you build a robust and reproducible workflow.

Speaker:

Andres Castillo
Process Manager
Sartorius

Ancillary raw materials used in cell and gene therapy (CGT) manufacturing are those materials that come into contact with the drug product during the manufacturing process but are not intended to be part of the final drug product. This talk discusses some of the pros and cons of using off the shelf raw materials versus custom raw materials. Off the shelf ancillary materials are materials that are bought directly from a Supplier’s catalog for use in manufacturing. However, customization is often needed to fit the unique processes involved in CGT manufacturing. For example, vialed ancillary materials may need to be changed into a bagged configuration to allow for a closed manufacturing process. Drug product manufacturers may also develop their own novel ancillary materials. This talk will discuss some keys to customization success, available risk management tools, and current pain points across the industry for consideration.

Speaker:

Lili Belcastro
Principal Scientist, Development Material Sciences & Engineering, Cell Therapy Development
Bristol Myers Squibb