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The Art of Gene Therapy digital summit is a live two-day event drawing on the collective experience of industry leading gene therapy companies and regulatory agencies such as the FDA, Orchard Therapeutics, bluebird bio, REGENXBIO and many more.

The digital summit will shine a light on gene therapy future technologies, technical challenges, lessons learned from scale up to commercialization and what is on the horizon for regulatory guidelines.


Day 1 | Tuesday, 28 July 2020
11am-3pm ET


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Introduction/Opening Remarks

Tuesday, 28 July 2020

11am-11:30am ET

Janet Lambert – CEO at Alliance for Regenerative Medicine

Join Janet Lambert – CEO at Alliance for Regenerative Medicine

  • Overview of the gene therapy market
  • Current challenges
  • Where we might be headed

Speakers

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Janet Lambert

CEO
Alliance for Regenerative Medicine


Janet Lynch Lambert joined ARM in 2017 as the organization’s first CEO. With more than 25 years in public and private sector management, Janet is an experienced government relations and business professional with an extensive record of accomplishment. Janet most recently served as the Acting Head of Engagement for the All of Us Research Program at the National Institutes of Health and as head of the Outreach Office in the Office of the NIH Director. Prior to joining NIH, she was Vice President of Government Relations and head of the Washington office of Life Technologies, aiding the company in its growth from $300 million in annual sales to more than $3 billion.

Prior to Life Technologies, Janet held leadership positions in government relations, marketing and business development at large and small life science organizations, including GE and InforMax. Her experience also includes legislative and staff leadership positions in the U.S. Senate and House of Representatives.

Janet received her MBA in International Business from Georgetown University and her B.A. in Political Science from Stanford University. She lives in the Washington, D.C. area with her husband and two daughters.

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Yesterday’s Learnings: Trial and errors from
scale up to commercialization

Tuesday, 28 July 2020

11:30am-12:15pm ET

Yesterday's learnings with Susan Abu-Absi – SVP, Pharmaceutical Development & Technology at bluebird bio

Joins us as Susan Abu-Absi from bluebird bio takes us through the trial and errors from scale up to commercialization


Speakers

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Susan Abu-Absi

SVP, Pharmaceutical Development & Technology
bluebird bio


Susan Abu-Absi, Ph.D. is Senior Vice President of Pharmaceutical Development & Technology at bluebird bio, a gene therapy company located in Cambridge, MA. The integrated product platforms at bluebird bio encompass gene therapy, cancer immunotherapy and gene editing – providing the potential to treat a broad range of serious diseases. She leads the teams that develop the CMC strategy and deliver and support the manufacturing processes and analytical methods required to advance therapies through clinical stages to commercial products and throughout the lifecycle. Bluebird bio’s first product, ZYNTEGLO™, received conditional MAA approval in June of 2019.

Susan has over 20 years of experience in biologics process development and manufacturing. Prior to joining bluebird bio, she spent more than ten years in the Manufacturing Sciences & Technology organization at Bristol-Myers Squibb. During her tenure at BMS, she supported clinical and commercial biologics drug substance manufacturing at internal sites and at contract manufacturers. She led teams of scientists, engineers and statisticians tasked with improving process robustness and yields and introducing new technologies into manufacturing. As a leader at BMS, Susan contributed to the successful development and commercial supply of several products, including the launches of Opdivo®, Empliciti®, Yervoy®, and Nulojix®. Before her tenure at BMS, Susan was a member of the Process Sciences team at Bayer Healthcare in Berkeley, CA.

Susan earned a Ph.D. in Chemical Engineering from the University of Minnesota and a B.S. in Chemical Engineering from the University of Toledo (Toledo, OH)..

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Tomorrow's View: What’s next for
analytical development?

Tuesday, 28 July 2020

12:30pm – 1:30pm ET

We take a look ahead with Audrey Chang – Head of Process Solution Services R&D at MilliporeSigma

Discussion Points:

  • What assays are being performed today?
  • How to develop assays to cope with evolutions in manufacturing
  • Regulatory guidance
  • Innovations for tomorrow
  • Increasing throughput
  • Rationale for CQA assessment

Speakers

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Audrey Chang

Head of Process Solution Services R&D
MilliporeSigma


Audrey Chang Ph.D. earned her PhD in Biology at The Johns Hopkins University; Baltimore Maryland and has over 25 years of government and industry experience in conducting biological products testing and in managing laboratories and her current role as Head of Process Solution Services R&D, MilliporeSigma she is responsible for managing and developing a team of PhD level scientific experts that provides greater focus to the development of new services and technologies while providing technical solutions and support to clients and operating laboratories. She and her department work to identify emerging products, technology trends, and global regulatory requirements and champion activities to implement new business plans.

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Regulatory Horizons: 2020 vision on FDA
guidelines

Tuesday, 28 July 2020

1:45pm – 2:45pm ET

Regulatory horizons with Peter Marks – Director of CBER at FDA

Joins us as Peter Marks from the FDA looks at the impact of the 2020 guidelines issues by the FDA and what that means for the future of gene therapy.

  • Today’s and tomorrow’s gene therapy regulatory landscape
  • The regulatory importance of defined manufacturing and analytic methodologies to the success of gene therapy
  • Efforts to facilitate the development of bespoke (individualized) gene therapy products

Speakers

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Peter Marks

Director of CBER
FDA


Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.


Day 2 | Wednesday, 29 July 2020
11am-4pm ET


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The Next Decade of Gene Therapy:
Q&A panel discussion

Wednesday, 29 July 2020

11am-12pm ET

Moderated by Anthony Davies from Dark Horse Consulting

Join us in our Q&A panel discussion as Anthony Davies from Dark Horse Consulting speaks to:

  • REGENXBIO
  • AskBio
  • Orchard Therapeutics
  • In Vivo
  • MilliporeSigma

Speakers

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Anthony Davies

Founder and CEO
Dark Horse Consulting


Anthony founded Dark Horse Consulting in 2014, bringing 20+ years of leadership experience in product, process and manufacturing development to cell and gene therapy companies in need. Anthony has a proven track record in managing pharmaceutical pipelines, is a skilled liaison with international regulatory agencies, and has an intense familiarity with a wide range of biologics, and cell and gene therapies. He is a highly sought-after keynote speaker and chair of national and international conferences and seminars, noted for his provocative, thoughtful and sometimes contrarian presentations.

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Sarah Thomas

VP, Quality
REGENXBIO


Sarah Thomas is the Vice President of Quality at REGENXBIO, responsible for the Quality Assurance and Analytical Development/Quality Control organizations. She has over 30 years’ experience in pharmaceutical and biotech Quality. She has experience working with products at all phases of development, from early clinical phases to commercial distribution. She has led Quality teams through the transition from clinical to commercial operations, and multiple product approvals. Ms. Thomas received an MBA from George Washington University and a BS in chemistry from Butler University.

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Lucie Ellis

Managing Editor
In Vivo


Lucie Ellis has been covering the ups and downs of the pharmaceutical industry since joining the Informa Pharma Intelligence team in 2012. Prior to being Managing Editor of In Vivo, Lucie was a Senior Editor at Scrip and Pink Sheet. She is responsible for the production of In Vivo’s print and online editions and also contributes feature-length articles to the publication on the topics of leadership, R&D, innovation and more.

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Andy Holt

VP, Business Development
AskBio


Andy Holt, VP of Business Development at AskBio, has spent more than 10 years in commercial development and management roles working in contract testing, contract manufacturing, and therapeutic development companies working in the cell and gene therapy space. Andy also serves as the VP of business development for Viralgen, Askbio’s affiliate focused on AAV contract manufacturing.

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Ran Zheng

Chief Technical Officer
Orchard Therapeutics


Ran Zheng is Chief Technical Officer at Orchard Therapeutics, overseeing multiple functions within technical operations including process development, manufacturing, quality, facilities and engineering, supply chain, and CMC lifecycle management. Ran has over 25 years of experience in biopharmaceutical industry. Prior to Orchard, Ran held various leadership positions in process development, manufacturing, quality and supply chain at Amgen, Genzyme and Diosynth.

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Ranjeet Patil

Segment Head, Cell and Gene Therapy
MilliporeSigma


Ranjeet Patil heads a global team of bioprocessing experts for cell and gene therapy segment. In current role, he and his team provide consultative expertise to address process challenges and guide development of innovative solutions by MilliporeSigma. For past 10 years, he has been working in MilliporeSigma’s technical functions in North America and Asia. His previous roles include early stage bioprocess development, virus clearance validation and process optimization & troubleshooting. Ranjeet is a bioprocess engineer and holds a post-graduate degree from Northeastern University, Boston, US.

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Better Tomorrow: Industrializing AAV Production

Wednesday, 29 July 2020

12:15pm – 1:15pm ET

Enabling The Reliable Supply of AAV Gene Therapy Vectors for Clinical and Commercial Applications with Luis Maranga – Chief Technical Operations Officer at Voyager Therapeutics

Hear from Luis Maranga of Voyager Therapeutics on:


  • Strategies for AAV development and manufacturing
  • Voyager’s Sf9/baculovirus platform
  • Process optimization
  • Achieving reliable AAV vector supply for low and high demand diseases

Speakers

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Luis Maranga

Chief Technical Operations Officer
Voyager Therapeutics


Dr. Maranga has over 20 years of experience leading the development and manufacturing for biologics, vaccines, and gene therapy products. Dr. Maranga was the technical lead for three BLAs (Optaflu/FluCellVax, Opdivo, and Empliciti), led the development of dozens of investigational products, and was plant manager for small (500‐2,000L) and large‐scale (150,000L) biologics manufacturing facilities.

Dr. Maranga was previously with Bristol‐Myers Squibb Company (BMS), where he was Vice‐President and Global Head Biologics Development; General Manager and Site Head for Devens Biologics Commercial Operations; and Executive Director, Manufacturing Sciences and Technology. Prior to BMS, Luis served as Head, Technical Development at Novartis, and held R&D roles for biologics and vaccines at AstraZeneca/MedImmune and Merck & Co.

Dr. Maranga holds PhD and BS degrees in Chemical Engineering from Universidade Nova de Lisboa (Portugal) and from the Instituto Superior Tecnico (Portugal), respectively.

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Promise of the Future: CRISPR and Gene Therapy

Wednesday, 29 July 2020

1:30pm – 2:30pm ET

Hear from Patrick Sullivan – Head of GENM R&D at MilliporeSigma

Recent small scale clinical trials have excited the Scientific and Medical communities about the potential promise of CRISPR based therapeutics. These trials have also shown CRISPR to be safe within the limits of the clinical study designs. To fully realize the promise of CRISPR based therapeutics, new technology platforms will need to be developed to improve both efficacy and safety. Manufacturing capabilities and new delivery methods will also need to be developed. Recent advances in new CRISPR based technologies to improve activity and specificity will be discussed in this presentation. Our vision for broader application of these technologies will also be discussed.


Speakers

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Patrick Sullivan

Head of GENM R&D
MilliporeSigma


Pat Sullivan is the Head of R&D for Gene Editing and Novel Modalities at MilliporeSigma, part of the Process Solutions division of Merck KGaA, Darmstadt, Germany. He is responsible for the development of new gene editing tools and new viral vector manufacturing processes.

Previously, he was the Head of Applied Solution’s Emerging Biology Research & Development. Pat was responsible for developing innovative products and new technology platforms targeting Applied Market customers with a focus on Clinical Testing and Pharmaceutical Drug Development.

Prior to joining Merck KGaA, Darmstadt, Germany, Pat served as the VP R&D for Sigma-Aldrich Life Science R&D, responsible for all new product development in the areas of genomics, proteomics, functional genomics, and cell-based assays. From 1988 to 2007, Pat worked for several Pharmaceutical companies including Searle, Pharmacia and Pfizer supporting and leading new target/biomarker discovery programs in inflammation and cancer. From 2000 to 2002, Pat worked for Incyte Genomics as the VP of Global Operations responsible for all products and services.

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A-Gene: Applying Quality By Design Principles
To The Development And Manufacture
Of Gene Therapies

Wednesday, 29 July 2020

2:45pm-3:45pm ET

Michael Lehmicke – Director of Science and Industry Affairs at Alliance for Regenerative Medicine talks A-Gene

This is a first-of-its-kind publication that compiles information from more than 50 gene therapy experts to create a reference guide on applying Quality by Design (QbD) principles to the development and manufacture of gene therapies.

  • The cell & gene therapy sector is in need of clear, evidence-based industry standards and best practices for manufacturing. To address this need, ARM has brought together 50+ experts in the gene therapy field to create A-Gene, a reference guide for applying Quality by Design principles to gene therapy manufacturing. The document will be released this summer.
  • A-Gene will focus on topics including quality target product profiles, comparability and lifecycle management, regulatory considerations, development & use of standards, critical quality attributes & risk assessment, upstream/downstream processing, and process control strategy.
  • By creating consensus on industry best practices in these areas, A-Gene will help existing developers scale up their production capabilities and avoid costly and time consuming regulatory delays. It will also provide a valuable tool for the next generation of gene therapy developers, allowing them to more effectively plan for manufacturing at scale earlier in the development timeline.

Speakers

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Michael Lehmicke

Director of Science and Industry Affairs
Alliance for Regenerative Medicine


Michael joined ARM in 2018 as its first Director of Science and Industry Affairs. Michael has over 20 years of R&D experience in biomaterials, medical devices and regenerative medicine. He has led product development teams for class II devices, human cell and tissue-based products, and drug/device combination products. He is a creator and an inventor with multiple U.S. patents to his name.

Michael has a MSc in /Biomedical Engineering, with a focus on tissue engineering, from Drexel University. Michaels areas of expertise include cell-based tissue engineering, bioceramics, biodegradable polymers, project management, strategic pipeline development, and business development.

He is passionate about regenerative medicine and believes that it represents our best hope for meeting many unmet clinical needs, thereby changing patient’s lives for the better.