The Vector

Volume 7, Issue 4: 21st Annual Meeting

Editorial Team

Phillip Doerfler, PhD - Editor, The Vector
Melvin Rincon, MD, PhD - Associate Editor, The Vector

Inside This Issue

President's Message
Annual Meeting News
Government & Regulatory Events
Industry Events

President’s Message

In this, my final message as President of our Society, I wish to thank you for your membership and contributions to both ASGCT and the field of gene and cell therapy. This has been a very exciting twelve months for this field with FDA approvals of three gene therapies in the United States. ASGCT too has reached new heights, expanding our Society to a record 2,800 members. Later this week, we will meet in Chicago for our 21st Annual Meeting. As of today, registration has already surpassed 3,000 people and will become the best-attended meeting in our history. Moreover, we are welcoming more exhibitors and sponsors than ever before.

Three years ago the society developed an ambitious strategic plan and over the last year, the Board of Directors and I have worked to complete all the goals that were listed. To this end we have build a secure financial footing for the Society, modernized our web site and digital footprint, and deepened our regulatory and legislative impact. On Friday, the Society published its most recent white paper, “Addressing the Value of Gene and Cell Therapy: Enhancing Patient Access to Transformative Therapies.” This paper poses critical questions and offers insightful viewpoints on valuing gene and cell therapies. Doug Olson, one of the early CAR-T patients, succinctly summarized at the recent ISCT meeting why this discussion is so essential for potential patients, ASGCT, and all stakeholders in stating, “A cure that is not accessible to all patients is not a cure at all.” The fact that our society is now active in not just in the research setting, but through public policy and popular discourse illustrates how the field has come of age in ASGCT’s 21^st year. Later this year the Society will be launching two other new program initiatives including a broad patient education program and significant career development grants. As ASGCT builds upon the successes in the field, we recognize it is crucial to support new scientists in the critical early stages of their careers.

On Saturday, May 19, I will hand the gavel to Michele Calos who will become the 22nd president of the American Society of Gene and Cell Therapy. Michele and the entire Board of Directors are well-poised to lead the Society as even more cell and gene therapy strategies move from investigational agents to standard of care in the next year.

Thank you for letting me serve as your President and I look forward to seeing you all in Chicago.

Annual Meeting News

Keynote Presentations

Dr. Kathy High will deliver the George Stamatoyannopoulos lecture on Friday May 18th. Dr. High began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders including gene therapy strategies. Her work on gene therapy for hemophilia led to a series of studies that characterized the human immune response to AAV vectors in a variety of target tissues and has now progressed to clinical translation of potential genetic therapies for multiple inherited disorders.

Dr. Nick Restifo will be delivering the Presidential Symposium on Thursday May 17th. Dr. Restifo’s research focuses on enhancing T cell-based immunotherapies and he has made broad contributions to the field of cancer immunity including identification of myeloid derived cell subsets that suppress anti-tumor T-cell responses and discovery of a novel subset of long lived human T memory stem cells in humans.

Download the Mobile App

Now is the time to finalize your schedules with the full information on scientific presentations now available. To make that even easier, you can use the newly-release ASGCT app. Read the research, build your schedule, find other attendees, and schedule meetings through the app, available on both the Apple App Store and Google Play.

Call for Applications for the Junior Newsletter Editor

ASGCT is looking for a new Junior Editor to join The Vector's editorial team. The Junior Editor serves a one year term, appointed every year by the ASGCT Publications Committee. Candidates are asked to submit a CV, statement of interest, and 500 word summary of this article. At the conclusion of the Junior Editor's term, the successful volunteer is expected to ascend to the position of Associate Editor and ultimately Editor.

The roles and responsibilities are as follows:

• Coordinate with ASGCT staff and the Editors to solicit scientific content and summaries for Breaking Through section of The Vector
• Review and edit scientific summaries before they are added to The Vector
• Participate on planning conference calls with the Editors and ASGCT staff

If you are interested in serving as the Junior Editor or have any questions, please send your CV, a brief statement of interest, and a 500 word summary of this article to Samantha Kay, ASGCT Project Manager, skay@asgct.org.

Government & Regulatory Events

ASGCT Releases White Paper Addressing the Value of Gene Therapy

The value of gene therapy has become an increasingly prominent topic following the 2017 FDA approval of three gene therapies (including gene-modified cell therapies). The discussion of value related to gene therapy must include their vast potential to treat a variety of diseases, many of them rare disorders with great unmet need. ASGCT has therefore created a white paper—Addressing the Value of Gene Therapy: Enhancing Patient Access to Transformative Treatments. The paper highlights the substantial value of gene therapy and the resulting significance of addressing barriers to patient access to approved therapies...Read More

Commercialization Workshop

Value Frameworks for Gene and Cell Therapy: 9:30 – 10:30 a.m.

The panel will review recent value framework efforts and discuss ways to define value for therapies with uncertain durability and how recent assessments of gene and cell therapies have impacted payer decision making.

Regulatory Advances: 11 a.m. – 12 p.m.

Speakers:

• Peter Marks, MD, PhD, FDA Center for Biologics Evaluation and Research
• Snehal Naik, PhD, Janssen Research & Development, LLC
• Robert Pietrusko, Pharm.D. Voyager Therapeutics Inc.
• Lynne McGrath, MPH, PhD, REGENXBIO

CAR T Cell Therapies Workshop

Accreditation: 2:20 – 3 p.m.

In this session Drs. Nikiforow and Warkentin will describe the development of standards to promote quality practice in immune effector cell administration. They will also discuss the voluntary, peer-led FACT accreditation program.

Implementing Commercial Products: 3:30 – 5 p.m.

In this session we will discuss the challenges of implementing commercial adoptive immunotherapy including practical considerations and our current understanding of the financial systems and potential barriers faced by patients and centers. 

Annual Meeting Symposia

Do Current Pricing Strategies Assure Fair Access to the Values of Gene-based Therapies?
Organized by the Ethics and Government Relations Committees
Thursday, May 17, 2018 at 8:00 am – 10:00 am in Salon A-3, 4

Tipping Point: Gene Therapy Entering the Market
Organized by the Clinical Trials and Regulatory Affairs Committee
Friday, May 18 8:00 AM – 10:00 am in International Ballroom South

Industry Events

Commercialization Workshop

Tuesday, May 15: 8:30 a.m. – 5 p.m.

The Commercialization Workshop, will focus on the pathways to the commercialization of gene and cell therapy products and the unique challenges emerging technologies pose in creating acceptable business models. You may register onsite at the Chicago Hilton on May 15.

Industry Symposia

Tuesday, May 16 – Friday, May 18

There are seven industry-sponsored symposia throughout the annual meeting and they cover everything from AAV innovation to clinical experience to market experience. Take the opportunity to learn from specialty leaders in every corner of the field during one of these far-reaching discussions.

Tools & Technology Forum

Thursday, May 17: 5:15 – 7:30 p.m.
Friday, May 18: 5:45 – 7:45 p.m.

After finding your favorite posters during the poster presentation session, head over to the Tools & Technology Forum—also in the exhibit hall—for 15 minute product, service, tool, and technology presentations from the most innovative companies in gene and cell therapy. 

Exhibit Hall

Make time to visit the more than 100 exhibitors in the Exhibit Hall, open at various times throughout the entirety of the 21st Annual Meeting. All of our exhibitors are presenting innovative products and services for the cutting-edge developments leading that continue to promote growth in the field. Booths will be staffed during coffee breaks, networking opportunities, poster sessions, and the Tools & Technology Forum—see the 2018 Program Guide for exact hours.

Thank You Supporters