Magnolia Room Workshops & Ballroom 1 Presentations

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7:15am – 8:45am | CONCURRENT WORKSHOPS – LIVESTREAM BEGINS

Magnolia Room | 7:15am – 8:45am
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

DOING BUSINESS IN JAPAN WORKSHOP:
The Innovative Regulatory Pathways for Commercialization in Japan – Latest Progress and Outlook

Japan takes pride in its forward-looking regulatory system for regenerative medicine products that include cell and gene therapies, and has further upgraded its scheme by deploying the Sakigake Designation System since 2016. An introduction to the system was presented at last year’s Cell & Gene Meeting on the Mesa. This workshop will dive even further into the system this year, and will be augmented with firsthand experiences from three progressive clinical development companies from overseas.

Workshop Facilitator:
Kunihiko Suzuki, Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); Vice Chairman and Member of the Board, MEDINET Co.

7:15am – 7:20am | Welcome Remarks
Speaker:
Yuzo Toda, Chairman, Forum for Innovative Regenerative Medicine (FIRM)

7:20am – 7:30am | Japan: Best Place for Commercialization of Regenerative Medicine
Speaker:
Masahiro Uemura, Director, Bio-Industry Division, Commerce and Information Policy Bureau, Ministry of Economy, Trade and Industry (METI)

7:30am – 7:40am | Regenerative Medicine Product Regulations in Japan – Enhancing the Development of Advanced Therapy
Speaker:
Kiyohito Nakai, Ph.D., Director, Medical Device Evaluation Division, Pharmaceutical Safety and Environmental Health Bureau, Ministry of Health, Labor and Welfare (MHLW)

7:40am – 7:50am | Experiences from Japan: Sakigake Designation System for Regenerative Medical Products
Speaker:
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

7:50am – 8:05am | Development of an “Off the Shelf” Cell Therapy for Ischemic Stroke and Other Indications Under the Regenerative Medicine Regulatory Framework in Japan
Speaker:
Gil Van Bokkelen, Ph.D., Chairman and CEO, Athersys

8:05am – 8:20am | Autologous CD34 Cell Therapy for Critical Limb Ischemia: A Long-term Japanese-American Partnership
Speaker:
Douglas Losordo, M.D., EVP, Global Head of Research and Development and Chief Medical Officer, Caladrius Biosciences

8:20am – 8:35am | Introduction of Gene Therapy for Rare Genetic Diseases in Japan
Speaker:
David Lennon, Ph.D., President, AveXis

8:35am – 8:40am | Q&A

8:40am – 8:45am | Closing Remarks
Speaker:
Yoshitsugu Shitaka, Ph.D., Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); President, Astellas Institute for Regenerative Medicine (AIRM)

8:45am – 9:00am | SHORT BREAK IN LIVESTREAM

9:00am – 9:15am

WELCOME REMARKS {Ballroom 1}

Speakers:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine (ARM); President and CEO, Hitachi Chemical Advanced Therapeutics Solutions; GM, Hitachi Chemical Regenerative Medicine Business Sector

9:15am – 10:15am

PLENARY SESSION: CHARTING THE PATH – LESSONS FROM THE PIONEERS OF CELL AND GENE THERAPY COMMERCIALIZATION {Ballroom 1}
This session will explore how far we have come in recent years with the approval of the first gene and cell therapy products and a burgeoning industry pipeline expected to produce numerous additional new therapies in the coming years. Executives from three of the companies who have been at the forefront of cell and gene medicine will share their perspectives on the major scientific, regulatory and technical developments of recent years that have contributed most to the sector’s current success.

Chair:

Robert Preti, Ph.D., President and CEO, Hitachi Chemical Advanced Therapeutics Solutions; GM, Hitachi Chemical Regenerative Medicine Business Sector

Speakers:

David Lennon, Ph.D., President, AveXis

Ron Philip, SVP, Head of Global Commercial, Spark Therapeutics

Pascal Touchon, SVP and Global Head, Cell and Gene, Novartis Oncology

10:15am – 10:45am | MORNING BREAK – NO LIVESTREAM AT THIS TIME

10:45am – 12:00pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

SPOTLIGHT SESSION: COMMERCIALIZING GENE THERAPIES FOR HEMOPHILIA {Ballroom 1}
10:45am – 11:15am
Compared to traditional methods for managing hemophilia, gene therapy holds the promise of greatly improving patient lives. This session brings together drug developers and manufacturers working to bring curative, state-of-the-art gene-based therapies to patients. The panel will explore opportunities and challenges of preclinical and clinical AAV programs for hemophilia.

Chair:
Jerry Keybl, Ph.D., Head of Cell and Gene Therapy Manufacturing Franchise, MilliporeSigma
Speakers:
Jonathan Garen, Chief Business Officer, uniQure
Dan Levin, Global Commercial Development and Hemophilia Marketing Lead, Pfizer
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics

COMPANY PRESENTATIONS {Ballroom 1}

11:15am – 11:30pm
11:30am – 11:45pm
11:45am – 12:00pm

12:00pm – 1:15pm | LUNCH – NO LIVESTREAM AT THIS TIME

1:15pm – 3:30pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

PANEL: NAVIGATING ACCEPTANCE, UPTAKE AND AFFORDABILITY ACROSS THE LIFECYCLE {Ballroom 1}
1:15pm – 2:15pm
Sponsored by Evidera
Regenerative and advanced therapies are now entering a changing and increasingly restrictive global environment. There is movement towards value assessment across the lifecycle by both regulators, HTA agencies and payers in the U.S. and EU. There is also growing focus on the affordability of costly therapies. Success in the emerging environment requires focus on value demonstration across the entire lifecycle from the earliest stages of development thru post-market differentiation as new therapies become available. This session will bring together regulators, manufacturers and payers to discuss what “good” looks like in the new global healthcare environment.

Chair:
Eric Faulkner, VP, Precision and Transformative Medicine, Evidera; Executive Director, Genomics Biotech and Emerging Medical Technology Institute, National Association of Managed Care Physicians
Speakers:
John Doyle, Dr.P.H., SVP and General Manager, Enterprise Solutions, Real-World and Analytics Solutions, IQVIA
Louis Jacques, M.D., Chief Clinical Officer, ADVI
Pamela Keith, Director, Oncology Reimbursement, Access and Value Marketing, Juno Therapeutics, a Celgene company
Ron Philip, SVP, Head of Global Commercial, Spark Therapeutics
Pilar Pinilla-Dominguez, Senior Scientific Adviser, National Institute for Health and Care Excellence (NICE)
Richard Powell, M.D., Chief Medical Officer, MedPOINT Management

COMPANY PRESENTATIONS {Ballroom 1}

2:15pm – 2:30pm
2:30pm – 2:45pm
2:45pm – 3:00pm
3:00pm – 3:15pm
3:15pm – 3:30pm
3:30pm – 3:45pm

3:45pm – 4:00pm | AFTERNOON BREAK – NO LIVESTREAM AT THIS TIME

4:00pm – 6:00pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

PANEL: OPPORTUNITIES AND CHALLENGES IN RARE DISEASE {Ballroom 1}
4:00pm – 5:00pm
Sponsored by Cell and Gene Therapy Catapult
Cell and gene therapies have moved from promise to reality in 2018, with rare diseases often in the vanguard of these remarkable new living medicines. Many firms have built substantial franchises in rare diseases, but there is still great unmet need and the potential to displace and disrupt existing therapies. This panel will explore the opportunities and challenges in rare diseases across cell and gene therapy modalities as these therapies come to market; exploring technical challenges, licensing, adoption and reimbursement in healthcare systems and public or patient attitudes to this medical revolution.

Chair:
Keith Thompson, CEO, Cell and Gene Therapy Catapult
Speakers:
Max Colao, Chief Commercial Officer, Abeona Therapeutics
Geoff MacKay, President and CEO, AVROBIO
Matthew Patterson, President and CEO, Audentes Therapeutics
Alvin Shih, M.D., CEO, Enzyvant

COMPANY PRESENTATIONS {Ballroom 1}

5:00pm – 5:15pm
5:15pm – 5:30pm
5:30pm – 5:45pm
5:45pm – 6:00pm

6:00pm | PARTNERING CLOSES – LIVESTREAM ENDS FOR THE DAY

7:15am – 8:45am | CONCURRENT WORKSHOPS – LIVESTREAM BEGINS

PATIENT AND PUBLIC ATTITUDES TOWARD GENE THERAPY WORKSHOP

Magnolia Room | 7:15am – 8:45am

PATIENT AND PUBLIC ATTITUDES TOWARD GENE THERAPY WORKSHOP

7:15am – 7:20am | Welcome Remarks
Speaker:
Dena Ladd, Executive Director, Missouri Cures

7:20am – 7:40am | Perceptions and Misconceptions: What is the current landscape?
This discussion will address existing patient and public attitudes toward gene therapy, and examine gaps in awareness and understanding as well as ideas on tools to fill them going forward.
Speakers:
Michelle Berg, VP, Patient Affairs and Community Engagement, Abeona Therapeutics
Susan Sikora, Program Director, ARM Foundation for Cell and Gene Medicine

7:40am – 8:20am | Successful Partnerships Between Industry and Patient Organizations
This presentation will explore case studies on how industry can successfully partner with the patient communities they serve, and how the patient perspectives can be integrated into clinical research and educational programs.
Speakers:
Amy Fisher, Patient Advocacy Lead, Spark Therapeutics
Kristin Smedley, President and Co-Founder, Curing Retinal Blindness Foundation
Kimberly Trant, Director, Head of Patient Advocacy and Engagement, Audentes Therapeutics

8:20am – 8:40am | Externally-led Patient-Focused Drug Development Meetings: What are they and what are the benefits?
This portion of the workshop will discuss how these meetings are organized and the systematic approach they provide to ensure that patient’s experiences, perspectives, needs and priorities are meaningfully incorporated into drug development and evaluation.
Speaker:
Jen Farmer, Executive Director, Friedreich’s Ataxia Research Alliance

8:40am – 8:45am | Closing Remarks
Speaker:
Dena Ladd, Executive Director, Missouri Cures

8:45am – 9:00am | SHORT BREAK IN LIVESTREAM

9:00am – 9:15am

OVERVIEW OF THE ALLIANCE FOR REGENERATIVE MEDICINE’S INITIATIVES {Ballroom 1}

Speaker:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

PANEL: LARGE PHARMA/BIOTECH’S LEADERSHIP ROLE – SUPPORTING THE COMMERCIAL SUCCESS OF CELL AND GENE THERAPIES {Ballroom 1}
9:15am – 10:15am
The era of cell and gene therapy has arrived. This panel will discuss large biopharma’s role and likely impact in developing and commercializing new and disruptive technologies globally.

Chair:
Gbola Amusa, M.D., Partner, Director of Research and Head of Healthcare Research, Chardan
Speakers:
Brian Bronk, Ph.D., Head of External Innovation, Rare Diseases, Sanofi
Martin Golden, SVP, Head of Global Marketing Strategy, Astellas Pharma
Gabriele Proetzel, Ph.D., Director, Regenerative Medicine, Takeda Pharmaceuticals
Bob Smith, SVP, Global Gene Therapy Business, Pfizer

10:15am – 10:45am | MORNING BREAK – NO LIVESTREAM AT THIS TIME

10:45am – 12:00pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

FEATURED TALK: FDA’S EFFORTS TO ADVANCE THE DEVELOPMENT AND APPROVAL OF CELLULAR AND GENE THERAPIES {Ballroom 1}
10:45am – 11:00am

Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

PANEL: RMAT REGULATORY CONVERGENCE: STARTING THE CONVERSATION {Ballroom 1}
11:00am – 12:00pm
Sponsored by Janssen R&D
This session will engage regulators and other industry leaders in an interactive conversation focused on expediting regulatory convergence as well as identifying actions that can be taken to further facilitate global convergence. Topics to be covered include ongoing efforts and priorities for regulatory convergence by focus area including CMC, nonclinical and clinical.

Chair:
Melody Eble, Pharm.D., Director, Global Regulatory Affairs, Scientific Innovation Projects – Regenerative Medicine and Digital Technology, Janssen R&D
Speakers:
Antony Appleyard, Ph.D., Technical Director Regulatory, Diamond Biopharm
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)
Jiwen Zhang, Ph.D., President, Standards Coordinating Body (SCB); Executive Director, Regulatory Affairs, Tmunity Therapeutics

12:00pm – 1:15pm | LUNCH – NO LIVESTREAM AT THIS TIME

1:15pm – 2:45pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

SPOTLIGHT SESSION: TISSUE ENGINEERING AND ORGAN TRANSPLANTATION {Ballroom 1}
1:15pm – 1:45pm
Tissue engineering holds the promise to eliminating the organ transplant waiting list, where over 120,000 patients currently wait, but how close are real solutions? Commercially, tissue engineering products have been limited to acellular or relatively thin constructs that lack vasculature and the complexity of functional tissue. This panel will discuss how recent advancements in decellularization technology and biologic scaffolds are addressing the vascular challenge. The speakers will describe the current pipeline of functional tissue engineered products including whole organs, how advancements in cell therapy have accelerated the path to market, the manufacturing, cell sourcing and distribution considerations when commercializing tissue engineering products and how recent regulatory programs – including RMAT – have fast-tracked the process.

Chair:
Jeff Ross, Ph.D., CEO, Miromatrix Medical
Speakers:
Jim McGorry, CEO, Biostage
Jason Wertheim, M.D., Ph.D., Edward G. Elcock Professor of Surgical Research and Associate Professor, Division of Transplantation, Northwestern University

COMPANY PRESENTATIONS {Ballroom 1}

1:45pm – 2:00pm
2:00pm – 2:15pm
2:15pm – 2:30pm
2:30pm – 2:45pm

2:45pm – 3:15pm | AFTERNOON BREAK – NO LIVESTREAM AT THIS TIME

3:15pm – 5:30pm | CONCURRENT TRACKS – LIVESTREAM RESUMES

PANEL: NEXT GENERATION CELL AND GENE THERAPY IN ONCOLOGY {Ballroom 1}
3:15pm – 4:15pm
With the recent approvals of the first cell and gene therapies in oncology, this panel will discuss what comes next. Focus will include improvements in manufacturing processes and supply chain, lessons learned in regulatory pathways including program prioritization decision making and clinical development approaches to highlight disease population choices, study design including endpoints, utilization of biomarkers and surrogates, comparator groups, study duration and safety assessments.

Chair:
Timothy Schroeder, Founder and CEO, CTI Clinical Trial and Consulting
Speakers:
Maria Fardis, Ph.D., President and CEO, Iovance Biotherapeutics
Rachel Haurwitz, Ph.D., President and CEO, Caribou Biosciences
Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen R&D, Janssen Pharmaceutical Companies of Johnson & Johnson
Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio

COMPANY PRESENTATIONS {Ballroom 1}

4:15pm – 4:30pm
4:30pm – 4:45pm
4:45pm – 5:00pm
5:00pm – 5:15pm
5:15pm – 5:30pm

5:30pm | LIVESTREAM ENDS FOR THE DAY