Crossing the Chasm: Clinical to Commercial Scale-up of Gene Therapies

Mar 08, 2019

Q&A with Kevin Slatkavitz, President/Founder, ThinkQuality, LLC

Patients are waiting – raising the stakes for breakthrough gene therapies and heightening the spotlight on the need for timely, innovative and reliable manufacturing scale-up solutions. We recently spoke with Kevin Slatkavitz, MassBio’s co-chair for the Drug Formulation, Manufacturing & Supply working group and President & Founder of ThinkQuality, LLC, about the upcoming member forum, “Crossing the Chasm: Clinical to Commercial Scale-Up of Gene Therapies,” taking place April 25th  at 8 am at MassBio.

What was the thinking behind bringing this forum to the MassBio community?

The MassBio community is a hot bed for advancing novel gene therapy drugs – many of these receiving fast track and/or breakthrough therapy status from the FDA. And, right now there’s a tremendous potential opportunity for sponsor companies to move quickly through development to approval and launch. Everyone wants to find the HOV lane to do this, as patients are waiting at the end, but there’s no substitute to building an understanding of your biopharmaceutical product and processes early and throughout development milestones.

We all know that while there are many facets of the drug development process that can stymie those great intentions, manufacturing scale-up from the clinical to commercial stage continues to pose the greatest challenge. This only becomes more of a barrier as gene therapies are expected to take on diseases with much larger patient populations than those currently on the market for rare diseases. There is no better time than now to bring together an esteemed panel and the MassBio community to discuss solutions to these bottlenecks.

Register for the forum here.

Tell us about the invited speakers who will be on the panel?

First of all, let me just say that our goal was to aim high – to identify and draw upon insights and expertise from leaders representing industry, government agencies and academia. We are delighted and honored to have confirmed such an outstanding and diverse group of speakers to share their perspectives on these challenges and opportunities.

Speakers include: Dr. Peter Marks (FDA, Director – CBER), Dr. Kelley Rogers (NIIMBL Technical Program Manager for NIST Office of Advanced Manufacturing), Dr. Susan Abu-Absi (BlueBird Bio, Sr. VP – Pharm Dev & Technology), Dr. Rod Rietze (Novartis, former Director – Strategic Development & Innovation for Cell & Gene Therapy Dev & Manufacturing), and Dr. Scott McMillan (Chief Operating Officer – uniQure). All have expressed great enthusiasm to be on the panel and are very much looking forward to the event.

You can find the full bio of each speaker, here.

Anyone who has heard Dr. Marks speak understands what I mean when I say that having him on the panel is priceless – it is always enlightening to hear his perspective on so many topics, including gene therapy manufacturing and the regulatory process. We are also very fortunate to have Dr. Rogers who will provide a breadth of knowledge on advancements from the latest innovative collaborations across industry and academia at NIIMBL (National Institute for Innovation in Manufacturing Biopharmaceuticals) and NIST (the National Institute of Standards and Technology) as they pertain to scale-up challenges.

Additionally, we are very pleased to have three experienced gene therapy development and manufacturing leaders from our local MassBio community to share their perspectives and draw on their experiences – Dr. Susan Abu-Absi who is immersed in BlueBird Bio’s current early and late stage developments, Dr. Scott McMillan of uniQure, with over 25 years of biotechnology experience from bench to commercial scale as well as operations, and Dr. Rod Rietze whose “been there and done that” for the approved gene therapy drug, Kymriah.

Who’s the target audience?  Who should be planning to attend the forum?

Given the significance, timeliness, and broad interest in this topic, I’m really looking forward to seeing a diverse group of professionals from many disciplines across the MassBio ecosystem in attendance. We are looking to have a highly interactive session with conversation between our esteemed speakers and the audience during the panel discussion, audience Q&A, and networking.

This is intended for anyone who is currently in the midst of, or soon to be wrestling to anticipate, identify or resolve manufacturing scale-up challenges. This forum will provide an opportunity for those interested to benchmark with and hear from other experts in the field – we encourage your participation. Overall, it’s for anyone who has lessons to share or anyone who has innovative solutions that may benefit others.

We envision attendance from leading scientists and professionals with expertise in many disciplines including; analytical, tech ops, manufacturing, and quality/regulatory affairs. We also hope to see business leaders from sponsor companies, innovators and facilitators from CDMOs, CMOs and service providers, and thought leaders from academia. In addition, this forum will be pertinent for those on the periphery, but who are heavily invested in the action and need to better understand scale-up risks and uncertainties to better assess impact on what they do such as investors and patient advocates. At the end of the day, patients are waiting.

Is there anything you need from the MassBio community?

Yes – absolutely! We really want to know what concerns are keeping everyone up at night. What are your questions regarding gene therapy clinical to commercial manufacturing scale-up? We’d like to know. 

In fact, our esteemed speakers have been asking, “What do people want to hear? What questions and concerns would they like us to discuss?” So, yes, MassBio community, we’d like to hear from you, so our speakers can make this a highly successful and engaging forum. Please send any questions and concerns that you would like to have addressed during this forum as soon as possible, but no later than Friday, March 22 to

We thank you in advance and look forward to hearing from you! And, while you’re thinking of questions, don’t forget to register for the forum, here.

Dr. Kevin J. Slatkavitz is President and Founder of ThinkQuality, LLC – an Associate Member of MassBio and innovative consulting firm providing clients across the life science ecosystem with practical strategies, solutions and coaching to address a wide array of quality and regulatory compliance-related challenges and opportunities from early stage discovery through critical CMC development milestones to product approval/launch and beyond. 

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