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Cultivating Our Roots

2019 ISCT ANnual report

ISCT Board of Directors, Strategic Planning 2020-2022 Event. Vancouver, Canada. February 2019

Cultivating Our Roots

2019 ISCT ANnual report

ISCT Board of Directors, Strategic Planning 2020-2022 Event. Vancouver, Canada. February 2019

JohnRasko

John EJ Rasko, AO, MBBS, PhD,
FRCPA, FRACP, FAHMS
ISCT President
(2018-2020)
Australia

These Are Challenges That ISCT Was Made For

The science is advancing, but the workforce needed to make it into an accessible clinical reality is not yet here.

I am genuinely excited to welcome you to ISCT’s second annual report. This cornerstone publication provides an overview of our strategic trajectory, and a moment of appreciation and acknowledgment for the contributions of our membership across the world.

Last year, we saw the start of a golden age for cell and gene therapy, and the accelerated evolution of our society. This year, we are taking steps to further sustain our growth.

Our Society has evolved in the past few years to meet the ambitions of a golden age of cell and gene therapy. Alongside that ambition, it is important to acknowledge where we have come from. The current state of cell and gene therapy is the result of decades of hard work, passionate dedication, and tireless perseverance.

From our pioneering investigators and lab technologists, to our regulatory stewards, to our business and industrial leaders, ISCT maintains a continued and comprehensive trajectory of advancement for our field. This is an opportune time to look back and to reflect upon our roots to cultivate the developments that we will enable and indeed push into the future.  

Within this report, you will see our efforts over the past year to connect membership across our global Society, to communicate to the public responsibly and respectfully, and to translate cutting edge scientific inquiry into concrete clinical procedures and products. In short, we are here after more than a quarter of a century to continue our mission to connect, communicate and translate.

This year, we are introducing a “Strategic Features” section, where you will learn about key ISCT initiatives. Here, you will have a chance to learn about our efforts to engage lab technologists and the dramatic impact of our mentorship program. You will also learn more about the successes of our Cell Therapy Training Course, updates to our global engagement model, and the exciting reshaping of Cytotherapy, our flagship publication.

In helping the public access safe and effective cell and gene therapies through our Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapies; in serving together across fields of academia, regulation, and commercialization to improve lives around the world, we have amassed a wealth of allies working to a common goal that has expanded substantially over the past year.

The past year also marks the growth of a sustained commitment and growing pursuit of mentorship within our Society. With our mentorship program at its third year, we have achieved exciting and unprecedented growth and passionate interest driving engagement at a global level. Our mentorship program focuses on career and professional development, but it is also built for members to connect personally and to develop collaborative dialogue that works to continually advance our field.

My term as President of the Society has been characterized by a commitment to the advancement of our communications. From our Board summit that brought our leadership team together in Vancouver in January 2019, to our annual and regional meetings which connect members from across the globe, we have prioritized personal engagement as part of our communicative strategy. We have reached out to allied organizations to create agreements and strategic partnerships, including significant memoranda of understanding laying out concrete action plans for the future we share with our allies. The Signature Series of our Scientific and Commercialization Committees are just one strong example that successfully showcases what we bring to the table.  

At its core, ISCT is built on the fundamental ideal of connection. This key aspect of our identity leads me to believe that ISCT represents a guiding beacon for the tsunami of innovation that is cell and gene therapy today.

Over the past year, blood stem cell gene therapy for transfusion-dependent thalassemia was approved in Europe. Gene-directed immunotherapy techniques have undergone significant consolidation across the globe. Reimbursement and clinical implementation issues are at the forefront of focus for governments and industry. The science is advancing, but the workforce needed to make it into an accessible clinical reality is not yet here.

Ahead of us lie great challenges if we are to sustain our growth and meet emerging demands in our field. The Good Ship “Cell and Gene Therapy” needs more hands on deck! Laboratories need to be supported as they scale upwards to meet industrial requirements. Training must be expanded so that expertise can grow and spread.

These are challenges that ISCT was made for. The pioneers and leaders we honour in our field have set strong foundations and provided a vision for a world where those with unmet medical needs have hope.

They point to a bright future ahead of us. As we continue to carry the momentum of decades into the work ahead, I invite you to rejoice with me at the flourishing of our field. Let us continue to cultivate our roots, and do the good work now needed to advance onwards.

QueenieJang

Queenie Jang, BSc (Pharmacy), MBA,
Chief Executive Officer
Canada

There Is No Society Like Ours

ISCT is unique. Because of this, we stand ready to advance our mission and to also lead others with this pursuit.

Our Society has grown throughout 2019.

This is a fact that characterizes the second annual report of the ISCT. Throughout the year, we have worked to rekindle our roots. Not only did we renew our focus towards CGT labs across the globe, but we also worked to foster an environment of mentorship within the Society. Along the way, we expanded our global footprint by making deeper connections across our regional partnerships.

Following up on a year of evolution through these actions, we are pushing the growth of the society towards a sustainable direction.

The passion across our leadership and membership has been palpable this year. The guidance of our ESP committee has resulted in an inspiring mentorship engagement. The new vision of Cytotherapy engages technologists and investigators alike. Meanwhile, the increasing success of our annual and regional meetings points to one realization: 

ISCT is ready to take on the challenges that lie ahead for the cell and gene therapy field.

Throughout 2019, the legacy of the ISCT shone bright.

Key contributors like co-founder Dr. Nancy Collins returned to help push the Society forwards. A renewed partnership with our daughter organization FACT helped us engage technologists globally. All the while, the insights of our founders have paid massive dividends towards our task today.

The founding vision of ISHAGE, which would become the ISCT, identified key needs that have now grown urgent. The field needs to develop a robust workforce. It needs programs that kickstart sustainable cycles of knowledge growth. It also needs collaborative communication platforms that emphasize a global membership. The efforts of our founders now give us momentum to push these key areas.

We have taken this momentum and pushed to engage labs on a global scale. Across academia and industry, ISCT provides useful resources and communications infrastructure. We support technologists developing manufacturing and quality control procedures necessary for clinical advancement.

We have pushed to cultivate scientific expertise around cell and gene therapy across the globe. Our ISCT Mentorship and Cell Therapy Training Course programs provide a platform for scientific leaders across the globe to cultivate the next generation of investigators.

We have pushed, finally, to engage the scientific community through Telegraft and Cytotherapy. Both publications have adopted methods to more deeply engage with collaborators. The editorial boards across both publications have also recruited to expand their expertise in CGT. In 2019, both publications took on vibrant visual redesigns and refining the scope of their visions.

This is an exciting moment where the potential of our field demands decisive action.

As the science races forward, cell and gene therapy requires people that can implement it. We must continue to cultivate technologists in laboratories, effective public-facing communicators, conscientious accreditors, and ongoing clear-sighted leadership. After all, there is no other Society like us.

We exist at a global scale. We engage the entire translational chain of science behind cell and gene therapy. We share a common vision, carried forward by over 41 standing committees.

ISCT is unique. Because of this, we stand ready to advance our mission and to also lead others with this pursuit.

With us are an effective and global network of collaborators, ranging from state regulatory bodies to industrial powerhouses to key academic opinion leaders. Last year, we drew on connections across this network to hold joint meetings across the world. These include SCSS-ISCT in the Asia region, ISCT ANZ-ISSCR-ASGCT in the Australia and New Zealand region, and our ISCT-SCA regional meeting under the umbrella of SOMICET’s Scientific Congress in the South and Central America region. We have developed joint initiatives that will sustain the advancement of the field so that we can address the gap between what we know and what we can implement.

The Society continues to advance. As we enter 2020, we will continue to optimize our engagement platforms for members. We will continue to grow our membership globally. We will work together to draw the future of the field into present realities, and by doing so, move from rekindling our roots to defining our legacy.

Our Board of Directors (2019-2020)

The ISCT Board of Directors consists of:

  • President (Chair of the Board of Directors)
  • President-Elect
  • Immediate Past President (Chair of the Strategic Advisory Council)
  • Global Secretary
  • Global Treasurer
  • Regional Vice Presidents

  • Chief Scientific Officer
  • Chief Regulatory Officer
  • Chief Commercialization Officer
  • 4 Elected ISCT Members
  • Senior Editor of the Society’s Journal
  • Chief Executive Officer. 

The Board of Directors is the main administrative body which manages the governance and strategic oversight of the Society.

Chair

John Rasko, AO, MBBS, PhD, FRCPA, FRACP, FAHMS
President

June 2018 – June 2020
Royal Prince Alfred Hospital
Sydney, Australia 

Directors

Bruce Levine, PhD
President-Elect

June 2018 – June 2020
Barbara and Edward Netter Professor in Cancer Gene Therapy
University of Pennsylvania
Philadelphia, PA, United States

Catherine Bollard, MBChB, MD
Chair, Strategic Advisory Council (Immediate Past President)
June 2018 – June 2020
Children’s National Health System
George Washington University
Washington, DC, United States

Lizette Caballero, BS, MT(ASCP)
Global Secretary
June 2019 – June 2022
UCSF Blood and Marrow Transplant Lab
San Francisco, CA, United States

Emily Hopewell, PhD
Interim Global Treasurer
June 2019 – June 2020
Indiana University School of Medicine
Zionsville, Indiana

Oscar Lee, MD, PhD
Asia, Regional Vice-President 
June 2019 – June 2021
National Yang- Ming University
Taiwan

Ngaire Elwood, PhD
Australia & New Zealand, Regional Vice-President
June 2018 – June 2020
Murdoch Childrens Research Institute
Melbourne, Australia 

Joan Garcia-Lopez, MD, PhD
Europe, Regional Vice-President 
June 2019 – June 2021
Director of Research and Education Banc De Sang i Teixits Barcelona, Spain

 

Lynn O’Donnell, PhD
North America, Regional Vice-President
June 2018 – June 2020
Ohio State University, James Cancer Hospital
Columbus, OH, United States

Patricia Rocco, MD, PhD
South and Central America, Regional Vice-President
June 2019 – June 2021
Federal University of Rio de Janeiro
Rio de Janeiro, Brazil

Daniel J. Weiss, MD, PhD
Chief Scientific Officer

June 2016 – June 2020
University of Vermont
Burlington, VT, United States

Miguel Forte, MD, PhD
Chief Commercialization Officer 
June 2016 – June 2020
Zelluna Immunotherapy
Oslo, Norway

Karen Nichols, Esq.
Chief Regulatory Officer
March 2016 – March 2019
Magenta Therapeutics
Cambridge, MA, United States

Elizabeth Stenger, MD, MSc
Elected Member MD
June 2019 – June 2021
UPMC Children’s Hospital of Pittsburgh 
Pittsburgh, PA, United States

Nadim Mahmud, MBBS, PhD
Elected Member PhD
June 2018 – June 2020
University of Illinois College of Medicine
Chicago, IL, United States

Jeannette Bloom, MBA, MT(ASCP)SBB
Elected Member Technologist
June 2018 – June 2020 
Baylor College of Medicine
Houston, TX, United States

Anne Lamontagne, MSc 
Elected Member Technologist
June 2019 – June 2021
Clinical Cell and Vaccine Production Facility
University of Pennsylvania
Philadelphia, PA, United States 

Donald Phinney, PhD
Senior Editor of the Journal 
October 2018 – Present
Professor at The Scripps Research Institute
Jupiter, FL, United States

Queenie Jang, BSc (Pharmacy), MBA
Chief Executive Officer 
ISCT
Vancouver, BC, Canada

Our Executive Management Committee

The ISCT Executive Management Committee consists of:

  • Chief Executive Officer (Chair of the Executive Management Committee),
  • President
  • President-Elect

 

  • Immediate Past President (Chair of the Strategic Advisory Council)
  • Global Secretary
  • Global Treasurer

The Executive Management Committee oversees the operational and management aspects of the Society.

Chair

Queenie Jang, BSc (Pharmacy), MBA
Chief Executive Officer 
ISCT
Vancouver, BC, Canada

Members

John Rasko, AO, MBBS, PhD, FRCPA, FRACP, FAHMS
President

June 2018 – June 2020
Royal Prince Alfred Hospital
Sydney, Australia 

Bruce Levine, PhD
President-Elect

June 2018 – June 2020
Barbara and Edward Netter Professor in Cancer Gene Therapy
University of Pennsylvania
Philadelphia, PA, United States

Catherine Bollard, MBChB, MD
Chair, Strategic Advisory Council (Immediate Past President)
June 2018 – June 2020
Children’s National Health System
George Washington University
Washington, DC, United States

Lizette Caballero, BS, MT(ASCP)
Global Secretary
June 2019 – June 2022
UCSF Blood and Marrow Transplant Lab
San Francisco, CA, United States

Emily Hopewell, PhD
Interim Global Treasurer
June 2019 – June 2020
Indiana University School of Medicine
Zionsville, Indiana

A Renewed Focus on Laboratories and Technologists

Developed By ISCT Members, For ISCT Members

The ISCT Laboratory Life Line

In 2019, our Lab Practices Committee founded the ISCT Laboratory Life Line, taking a proactive and leading step towards promoting high standards and knowledgeable protocols across our membership in lab settings. This forum is designed as an interactive resource where professionals can engage in shop talk, facilitating collaboration, troubleshooting, and engagement for lab personnel. We encourage interested membership to contribute and learn about best practices for lab practices, leadership, or organization through accessible online discussions at our Life Line.

Our community engagement is the cornerstone of this effort

In today’s ever-changing landscape of cell and gene therapy, it is more important than ever to capitalize on the collective expertise of ISCT members to drive best practices. The Laboratory Life Line is one platform that was developed to encourage lab professionals of all backgrounds to share ideas, collaborate and educate one another to promote excellence across the cell therapy field.
Our community engagement is the cornerstone of this effort.

New Project (10)

Heather Garrity, BSc, MSc
Co-Chair, ISCT Lab Practices Committee
United States

ISCT-FACT Partnership

We began a new partnership over the past year with one of our closest collaborators, the Foundation for Accreditation of Cellular Therapy (FACT), with the goal of spreading educational resources across the membership embedded within a wide network of laboratories. The start of this partnership was exciting not only for the tangible benefits that we worked together to bring to a wider network, but also because through it, we worked to rekindle our roots through the shared heritage of our organizations.

Nancy Collins - Cropped Image

Nancy Collins, PhD
Co-Founder, ISCT
Founding Board Member, FACT

United States

An unprecedented journey of communication, education, and trust between practitioners and regulators

The primary motivators for founding ISHAGE (now ISCT) and ASBMT (now ASTCT), was concern for  development of hematopoietic stem cell transplantation, patient safety protection, and translation of  cutting edge research into clinical application. In the early years, we were well aware that the FDA feared unsafe development in our field, and we feared adoption of a drug development regulatory model that did not fit cell therapy.  

ISHAGE and ASBMT leaders founded FACT in 1996 as a voluntary inspection and accreditation organization to be a single voice for collection, processing, and clinical centers which used the best and safest practices.  The resulting FACT and FDA collaboration has been an unprecedented journey  of communication, education, and trust between practitioners and regulators. It has fundamentally changed both groups, resulting in regulations and guidances which are informed by FACT accredited organizations. This model is even more important today since the expansion of hematopoietic transplantation to include all cell types in the new field of regenerative medicine,

More than ever, it is important that all laboratories have access to educational resources and have tools to educate and train the next generation of cell and gene therapy professionals.

Medical laboratory professionals play a vital role in cellular therapy. Even with the best quality management systems in place, quality results are difficult to ensure without adequately trained staff.

Personnel at FACT-accredited labs are required to be knowledgeable about FACT Standards and federal regulations. An ISCT Laboratory Membership can provide technologists access to ISCT-sponsored meetings and ongoing online educational resources aimed at providing solutions for quality, operational, and regulatory challenges in the Cell and Gene Therapy Lab at a discounted rate.

FACT has partnered with ISCT to provide a special Laboratory Membership offer to FACT-accredited laboratories. This offer includes 3 complimentary technologist memberships with a 2020 ISCT Lab Membership. To ensure all staff have access to ISCT resources including education, forums, the
Telegraft, and Cytotherapy, additional technologist memberships are highly subsidized!

New Project (37)

Linda Miller
Chief Executive Officer, FACT 
United States

Engaging Industry Labs

As we work to advance the field of Cell and Gene Therapy, labs dedicated to industrial manufacturing have been on the forefront of our strategic vision. Since our last annual report, we worked to engage laboratories working under the umbrella of our industry partners, to assist them in identifying key personnel requirements, in recruiting highly qualified personnel to meet these requirements, and to train technicians where there are gaps in the pool of available applicants.

Bauer (1)

Gerhard Bauer, PhD
Laboratory Director, GMP Facility, UC Davis
United States

They don’t need master’s degrees; they need to receive appropriate manufacturing training and learn how to work within a GMP environment

Good Manufacturing Process (GMP) manufacturing of approved, commercialized cell and gene therapy products, and late phase clinical trial products slated for the market, has already become a major part of our industry.

Over the last three years we have been able to produce overwhelming evidence that such products can save lives of patients who did not have a great chance of survival only a few years earlier. We do face, however, a challenge in bringing a sufficient quantity of such products to market while keeping the price within an affordable range. We therefore need to find better ways of manufacturing cell and gene therapy products.

For this purpose, what is still needed is a larger work force of cell and gene therapy hands on product manufacturers; they don’t need master’s degrees; they need to receive appropriate manufacturing training and to learn how to work within a GMP environment, faithfully executing tasks under supervision proficient in Quality Control and Quality Assurance. If we want to get more life-saving products to patients, then we need to develop educational programs for such qualified personnel, and will need to coordinate our efforts so students can have access to such programs at several centers of excellence.

An Allied Perspective – CCRM’s Strategy to Support Commercialization

CCRM is accelerating the translation of promising technologies, processes and therapies into life-changing health outcomes for patients.

We are making this possible by providing strategic funding, dedicated infrastructure and specialized business and scientific expertise to support the commercialization of cell and gene therapies. We have partnered with leading research institutions to launch new ventures, and we enable industry by providing innovative contract development manufacturing organization (CDMO) services and scaling emerging companies by catalyzing investment. Supporting and enabling training, to ensure we have a skilled workforce, is another area we are tackling. The industry has many challenges to overcome and we have made it our purpose to solve the big problems in regenerative medicine.

New Project (43)

Michael May
Chief Executive Officer, CCRM
Canada

Rekindling our roots

ESP Mentorship as a Model for Global Expertise

A Mentor’s Perspective

Diseases know no borders. Global problems need global and new solutions. Developing innovative therapies requires the involvement of committed individuals whose talents combine scientific excellence, awareness of unmet medical needs, and entrepreneurial spirit.

New Project (12)

Christian Chabannon,
MD, PhD
Professor
Aix-Marseille Université 
France

photo GramignoliR(2)

Roberto Gramignoli,
MS, PhD 
Senior Researcher
Karonlinska Institutet 
Sweden

A Mentee’s Perspective

This reinvigorating experience had allowed us to stand ‘upon the shoulders of giants’ to achieve a shining, yet practical vision on contributing to the future of cell and gene therapies. 

New Project (12)

Christian Chabannon, MD, PhD
Professor
Aix-Marseille Université 
France

A Mentor’s Perspective

Diseases know no borders. Global problems need global and new solutions. Developing innovative therapies requires the involvement of committed individuals whose talents combine scientific excellence, awareness of unmet medical needs, and entrepreneurial spirit.

After all, cell and gene therapies are paradigm-shifting. They promise curative efficacy for diseases often unmanageable by existing pharmaceuticals. The ISCT has a core mission in overseeing the translation of proven novel therapies into clinical practice, made possible thanks to its tremendous capacity to gather preeminent experts across all fields of cell and gene therapies worldwide, from academic institutions to regulatory agencies and industry partners. Thanks to visionary educational programs such as the ISCT Mentoring Program, early stage professionals like myself had the terrific opportunity to connect with such experts as mentors to share experience and develop expertise in a collaborative, friendly network. 

New Project (29)

Roberto Gramignoli,
MS, PhD 
Senior Researcher
Karonlinska Institutet 
Sweden

A Mentee’s Perspective

This reinvigorating experience had allowed us to stand ‘upon the shoulders of giants’ to achieve a shining, yet practical vision on contributing to the future of cell and gene therapies.

The ISCT Mentoring Program was designed to bring new blood to the fast-growing field of cell and gene therapy. As a mentor, I was lucky enough to connect with two junior investigators who ticked all the boxes; it is an extremely rewarding experience to be part of a global initiative that helps accelerate biomedical progress worldwide.

Our group’s work resulted in a peer-reviewed publication that identifies some major hurdles along the developmental path of new treatments manufactured through cell or tissue engineering.

The ISCT Mentoring Program was designed to bring new blood to the fast-growing field of cell and gene therapy. As a mentor, I was lucky enough to connect with two junior investigators who ticked all the boxes; it is an extremely rewarding experience to be part of a global initiative that helps accelerate biomedical progress worldwide.

Our group’s work resulted in a peer-reviewed publication that identifies some major hurdles along the developmental path of new treatments manufactured through cell or tissue engineering.

After all, cell and gene therapies are paradigm-shifting. They promise curative efficacy for diseases often unmanageable by existing pharmaceuticals. The ISCT has a core mission in overseeing the translation of proven novel therapies into clinical practice, made possible thanks to its tremendous capacity to gather preeminent experts across all fields of cell and gene therapies worldwide, from academic institutions to regulatory agencies and industry partners. 

Although a decade has passed since the approval of the first ATMP therapy in Europe, and half of a century since the first successful cell transplant, established ATMP products can be counted on one’s fingers. In our editorial in Cytotherapy, my colleagues and I put the perspective of early stage professionals in dialogue with long-term expert vision, to summarize our discussions throughout the 6 months of the ISCT Mentoring Program towards identifying the main challenges in driving cell and gene therapies to the clinic.

Authored by A. Rotolo, C. Chabannon and R. Gramignoli, this editorial in Cytotherapy 22.2 arose out of productive discussions that started within the 2018-2019 ISCT Mentorship Program, and provides a useful and succinct overview of some of the challenges facing the CGT field today.

Doubling Down on Mentorship

The sophomore year of our ISCT Mentoring program has been met with immense interest across both mentor and mentee groups, almost doubling involvement from our 2018-2019 cycle. With over 20 mentors and 55 mentees for the 2019-2020 roster, our mentorship program is looking to grow even further to promote compassionate and competent expertise across our membership.

Our program is structured to foster a true community of experts, not only serving to connect mentors to mentees, but to create an enduring, collaborative network of mentees who will grow together as they advance both their careers and the field. Each mentor is paired with a small working group of two to three mentees, who work together for a span of at least half a year through monthly teleconferencing. Each mentorship group is determined by areas of interest, including Regulatory, Quality and Operations, Basic Science, Clinical, and Commercialization, as well as by geographical location.

The next cycle of the ISCT Mentoring Program will be recruiting in Q3 2020. 

“Truly Borderless, Truly Global”

Amanda Medcalf
Sr. Director of Manufacturing and QC
Fate Therapeutics
Anthony Ting
VP of Regenerative Medicine
Athersys, Inc
Bruce Levine
Professor
Director of the Clinical Cell and Vaccine Production Facility
University of Pennsylvania
Catherine Bollard
Professor of Pediatrics and Immunology,
Children’s National Health System and George Washington University
Conrad Russell Cruz
Assistant Professor
Children’s National Hospital
Cornelia Kasper
Professor
Department for Biotechnology, BOKU
Daniel J. Weiss
Professor of Medicine
University of Vermont
Dominic Wall
Director Pathology, Operations
Peter MaCallum Cancer Center
CSO
Cell Therapies Pty Ltd
Dominic Clarke
Global Head of Cell Therapy
HemaCare Corporation
Elizabeth J. Shpall
Professor
MD Anderson Cancer Center
Fermin Sanchez-Guijo
Head, Cell Therapy Area,
IBSAL-University Hospital of Salamanca, Spain
Jacques Galipeau
Director of the Program for Advanced Cell Therapy
University of Wisconsin
Janet Macpherson
Product Specialist – Cell & Gene Therapy
GE Healthcare Life Sciences
Kim Raineri
Vice President of Operations
Nikon CeLL Innovation Co., Ltd.
Kurt Gunter
Chief Medical Officer
Kuur Pharmaceuticals
Ngaire Elwood
Director
BMDI Cord Blood Bank
Orestis Argyros
Biology Manager,
GSK
Sandeep Soni
Associate Professor of Pediatrics
Stanford University
J. Wade Atkins
Quality Assurance and Regulatory Affairs Specialist
National Institute of Health
William Janssen
Director of Human Applications  Laboratory
St Jude Children’s Research Hospital

ISCT recognizes the growing need for qualified personnel and leaders in the field of cell and gene therapy. To attract and support emerging professionals within this industry, ISCT’s Early Stage Professionals (ESPs) Committee provides a networking and educational forum for new cell and gene therapy professionals from around the globe that helps to establish leaders of not only ISCT, but also the field at large. The ESP Committee is composed of 25 members from 4 countries who develop programming for their fellow ESPs, including specialized sessions at the ISCT Annual Meeting, Early Stage Professionals Abstract awards, and a group mentoring program.


ESP/CellCAN Job Market Survey

 

During the 2019 ISCT North America Regional Meeting, ISCT’s Early Stage Professionals (ESP) Committee, in partnership with CellCAN, conducted a joint session focused on examining one key bottleneck for the ongoing development of the cell and gene therapy field: A shortage of Highly Qualified Personnel (HQP).

The session made use of live polling to collect data to expands upon the ESP Committee’s 2018 Job Market Survey, sparking an ongoing broader campaign to gather data on the knowledge gaps within the field, as well as the realities of ESPs as they seek career progression.

Take a look at some of the results below:

New Project (40)

Head-to-Head: Employees vs. Employers

Training Prior to Employment

A Key Disparity

ISCT recognizes the growing need for qualified personnel and leaders in the field of cell and gene therapy. To attract and support emerging professionals within this industry, ISCT’s Early Stage Professionals (ESPs) Committee provides a networking and educational forum for new cell and gene therapy professionals from around the globe that helps to establish leaders of not only ISCT, but also the field at large. The ESP Committee is composed of 25 members from 4 countries who develop programming for their fellow ESPs, including specialized sessions at the ISCT Annual Meeting, Early Stage Professionals Abstract awards, and a group mentoring program.


ESP/CellCAN Job Market Survey

 

During the 2019 ISCT North America Regional Meeting, ISCT’s Early Stage Professionals (ESP) Committee, in partnership with CellCAN, conducted a joint session focused on examining one key bottleneck for the ongoing development of the cell and gene therapy field: A shortage of Highly Qualified Personnel (HQP).

The session made use of live polling to collect data to expands upon the ESP Committee’s 2018 Job Market Survey, sparking an ongoing broader campaign to gather data on the knowledge gaps within the field, as well as the realities of ESPs as they seek career progression.

Take a look at some of the results below:

New Project (40)

Head-to-Head: Employees vs. Employers

Training Prior to Employment

A Key Disparity

NEW THIS YEAR:

Training the Next Generation

With the dramatic growth in the cell and gene therapy field, there is an urgent need to train the next generation of professionals. ISCT is playing a key role in developing the careers of Early Stage Professionals (ESP) as they enter the CGT field in record numbers. Our ESP membership has grown from 303 in 2018 to 395 in 2019, reflecting a growing interest and engagement within the field.

In 2019, we introduced student registration to our Annual Meeting, as well as to our NA Regional Meeting. This registration category, available to medical, pharmacy, and nursing students hoping to attend ISCT Annual Meetings, includes complimentary pre-conference day registration, in addition to main conference registration at a highly subsidized price of $50. Throughout 2019, 42 students were able to make use of this.

Leading into 2020, ISCT plans to launch a series of exciting training and professional development opportunities. Among these are the European Cell Therapy Training Course, planned for 2021, and a partnered scholarship program for Master’s degree programs offered at the University of Granada and sponsored by Miltenyi Biotec. 

Starting Cycles of Growth

John-Barrett-Headshot

John Barrett, MD
Co-Founder, ISCT-ASTCT Cell Therapy Training Course
USA

Cell therapy is an upcoming and unique discipline still forging its trajectory.

It is attracting a broad spectrum of scientists, with both technical and clinical backgrounds who are urgently demanding  training in all the aspects of expertise required. This includes not only the basic science behind cellular therapies, but also comprehension of practical steps that have to be taken to make a clinical-grade cell product, as well as knowledge of the regulatory framework covering cell production in both academic and commercial laboratories. 

There is a growing need to train future cell therapists, including experts in cell production as well as those involved in clinical trial development  and the translation of cell production from academic institutes to the commercial world. Dave DiGiusto and I conceived of this training course to meet that need. It is a joint effort by the ISCT and the American Society for Cell Therapy (TCT), a connection mirrored in the diversity in backgrounds of attending scholars, many of whom have developed their interest in cell therapy through stem cell transplantation.  

In the 5 years since we began, the field has matured, and we have striven to adapt the curriculum to reflect emerging areas of development.  We have steadily improved the course, adapting it responsively through feedback from the scholars. 

In October 2019 we held the third CTTC in Philadelphia hosted by Dr Bruce Levine. By all accounts, this was our most successful CTTC yet. One shortcoming is that we cannot meet the continually growing demand for training. We can take only a fraction of all the applicants who might benefit from the CTTC and we run the course only on alternating years. We hope that the success of the style and contents of the CTTC will inspire local training courses to develop within ISCT.

John-Barrett-Headshot

John Barrett, MD
Co-Founder, ISCT-ASTCT
Cell Therapy Training Course
USA

Cell therapy is an upcoming and unique discipline still forging its trajectory.

It is attracting a broad spectrum of scientists, with both technical and clinical backgrounds who are urgently demanding  training in all the aspects of expertise required. This includes not only the basic science behind cellular therapies, but also comprehension of practical steps that have to be taken to make a clinical-grade cell product, as well as knowledge of the regulatory framework covering cell production in both academic and commercial laboratories.

There is a growing need to train future cell therapists, including experts in cell production as well as those involved in clinical trial development  and the translation of cell production from academic institutes to the commercial world. Dave DiGiusto and I conceived of this training course to meet that need. It is a joint effort by the ISCT and the American Society for Cell Therapy (TCT), a connection mirrored in the diversity in backgrounds of attending scholars, many of whom have developed their interest in cell therapy through stem cell transplantation.  

In the 5 years since we began, the field has matured, and we have striven to adapt the curriculum to reflect emerging areas of development.  We have steadily improved the course, adapting it responsively through feedback from the scholars. 

In October 2019 we held the third CTTC in Philadelphia hosted by Dr Bruce Levine. By all accounts, this was our most successful CTTC yet. One shortcoming is that we cannot meet the continually growing demand for training. We can take only a fraction of all the applicants who might benefit from the CTTC and we run the course only on alternating years. We hope that the success of the style and contents of the CTTC will inspire local training courses to develop within ISCT.

A World-Class Standard

Our Cell Therapy Training Course, designed in partnership with the ASTCT and held for the third time in 2019, brought together global leaders and rising stars in the field of cell and gene therapy to advance the field at large. Participating scholars were competitively selected and fully sponsored for the week-long course. Through CTTC, selected scholars were provided with world-class technical, clinical, and leadership training, and connected to an elite network of contacts in the field.

The third biennial 2019 Cell Therapy Training Course was held this fall in Philadelphia, hosted by the University of Pennsylvania. Select lecture recordings from the course can be found here (members only), in addition to the full course program.

If you are interested in enrolling as a scholar, keep an eye out for CTTC 2021. 

Cell Therapy Training Course 2019

In Partnership With: 


Hosted By: 


In Partnership With: 


ASTCT Logo

Hosted By: 


Penn Medicine

Sponsored in part by: 


Novartis
Legend Biotech
Tmunity
Emily Whitehead Foundation
Fresenius Kabi
Kite

CO-CHAIRS

John Barrett, MD

David DiGiusto, PhD

PLANNING FACULTY

Catherine Bollard, MD, MBChB

Colleen Delaney, MD, MSc

Bruce Levine, PhD

Krishna V. Komanduri, MD

SPEAKING FACULTY

Usman Azam, MD

Tmunity Therapeutics

Veronika Bachanova, MD, PhD 

University of Minnesota

Joseph A. Fraietta, PhD

Center for Advanced Cellular Therapies
University of Pennsylvania
Perelman School of Medicine

Philip J. Cross, MS

Philip J. Cross & Associates, Inc.

 

Anna Gilbert, ASQ CQA

BDO USA

Noelle Frey, MD, MSCE

Cell and Transplant Therapy Program
University of Pennsylvania

Elizabeth Hexner MD, MSTR

Abramson Cancer Center
University of Pennsylvania

Whitney Gladney, PhD

Center for Cellular Immunotherapies
University of Pennsylvania

Megan Kasimatis Singleton
JD, MBE, CIP

Office of Human Subjects Research
Johns Hopkins University

Wei-Ting Hwang, PhD

Department of Biostatistics, Epidemiology
and Informatics (DBEI)
University of Pennsylvania

Peter Marks, MD, PhD

Center for Biologics Evaluation and Research (CBER)
U.S. Food & Drug Administration

Lester Lledo, MSN, CRN P

Penn Medicine, Center for Cellular Immunotherapies
University of Pennsylvania

Jos Melenhorst, PhD

Pathology & Laboratory Medicine
University of Pennsylvania

Shannon Maude, MD, PhD

Medical Director, Center for Cellular Immunotherapies
University of Pennsylvania

Donald M. O’Rourke, MD

The Abramson Cancer Center and
Perelman School of Medicine

Doug Olson, PhD

BUHLMANN Diagnostics Corp.
Cancer survivor and patient two in the initial CART 19 clinical trial

Johannes van der Loo, PhD

Center for Cellular & Molecular Therapeutics
The Children’s Hospital of Philadelphia

Elizabeth J. Shpall, MD

The University of Texas MD Anderson Cancer Center

 Gabriela Plesa, MD, PhD

Center for Cellular Immunotherapies
University of Pennsylvania

SCHOLARS

Gabor Foldes
MD, PhD

Giulia Golinelli
PhD

Andrea Henden
MBBS(Hons), FRACP, FRCPA

Gaurav Sutrave
BSc(Med)/MBBS(Hons I), FRACP, FRCPA

Robert Myles Wright
MBBS

Saurabh Dahiya
 MBBS

Shoba A. Navai
MD

Shabnum Patel
PhD

Irene Scarfo
PhD

Frederico Simonette
MD, PhD

Mauro Castellarin
PhD

Saba Ghassemi
PhD

Philipp C Rommell
Dr.rer.nat

A Masterclass in CGT

The Grassroots Footprint of our Regional Model

In 2019, we revised our regional engagement practices across the globe, both to widen our footprint in the world and to engage specific needs in specific places within the field of Cell and Gene Therapy.

Our regional model is unique within the field of cell and gene therapy. Our membership benefits from the passionate involvement of local leadership across the globe, with independent initiative that is supported by our head office. Our committees conduct region-focused research, create partnerships with other organizations on a global scale, and host scientific meetings and sessions that continually push the field forward.

This is accomplished through the hard work of our five autonomous regional committees, and the support of ISCT’s global network. Throughout the year, the efforts of each committee have resulted in a series of productive scientific meetings, partnered joint sessions, and growth both in our membership and in our connection to the field. 

Events created by our regional committees are vital towards our efforts to coordinate scientific advancement, regulatory oversight, and innovative commercialization strategies at a global level. 

Here are some of the highlights from our regional committee events this year:

Steve Oh1

Steve Oh, PhD
Past Regional Co Vice-President, ISCT Asia
Singapore

Excitement and Challenges in Asia

The growth of immunotherapies has resulted in excitement and challenges in Asia. As these breakthrough therapies become widespread, challenges like the cost of goods, manufacturing, and critical quality attribute (CQA) issues will have to be addressed.

At the SCSS-ISCT Joint Meeting, “Frontiers in Cell Therapy,” presentations from ISCT experts like Ohad Karnelli, and Bruce Levine certainly drew delegate attention. Company presentations from partners like RoosterBio, BioSpherix, and X-Therma also provided practical and timely solutions for the field.

Three programmes from Singapore, funded at a total of S$80 million, were also highlighted at the event. In 2020, we expect to see some industry engagements being announced as a result of this significant investment by the Singapore government.

Showcasing Science Down Under

The ISCT ANZ-ASSCR-AGCTS Joint Scientific Meeting provided me with the wonderful opportunity to present our group’s work in targeted T cell immunotherapies to an engaged and dynamic group of peers. The conference size and format facilitated discussion between participants across disciplines. The scientific connections and discourse generated by presenting at this focused meeting will be invaluable to my professional development and future work.

I was honoured to be the recipient of an ISCT ANZ travel grant and award for highest scoring abstract. Regional opportunities such as the this grant are so important to early career researchers to facilitate the sharing of innovative work and continue to grow our community of expert, passionate scientific minds.

Img

Wei Jiang, PhD
Westmead Cellular Therapies Group
Australia

Our Regional Leaders

Oscar Lee, MD, PhD Regional Vice-President ISCT Asia

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Ngaire Elwood, PhD Regional Vice-President ISCT ANZ

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Joan Garcia-Lopez, MD, PhD Regional Vice-President ISCT Europe

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Lynn O’Donnell, PhD Regional Vice-President ISCT North America

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Patricia R.M. Rocco, MD, PhD Regional Vice-President ISCT SCA

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Oscar Lee, MD, PhD
Regional Vice-President
ISCT Asia

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Ngaire Elwood, PhD
Regional Vice-President
ISCT ANZ

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Joan Garcia-Lopez, MD, PhD
Regional Vice-President
ISCT Europe

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Lynn O’Donnell, PhD
Regional Vice-President
ISCT North America

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Patricia R.M. Rocco, MD, PhD
Regional Vice-President
ISCT SCA

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Boots on the ground

Cytotherapy, Rebranded

New Project (8)

Donald Phinney, PhD
Senior Editor, Cytotherapy
United States

Cutting edge research in the rapidly expanding fields of cell and gene therapy

Cytotherapy has undergone remarkable changes in aesthetic and content and celebrated a record impact factor in 2019. Building on this momentum, the Editorial Team is focused on continuing to increase the global influence of the journal by publishing cutting edge research in the rapidly expanding fields of cell and gene therapy. They are also working to further strengthen the relationship between the journal and ISCT by developing new initiatives, collaborations, and special features that highlight the important work done by its members. Through these efforts, we hope to see unprecedented rates of growth for both the journal and the society over the next few years.

New Look. New Editorial Board. New Impact.

2019 has been an exciting year for Cytotherapy. In June, our publication was rebranded on several levels. First, the look and feel of the journal has been updated to reflect its expanding focus. Photographs of cells, sourced from our society’s membership through the Insta-Your-Cells photo challenge, speak to a continuing commitment to engage with our community. 

The expertise of Cytotherapy has also shifted, with new additions to our editorial staff, and the clarification of the journal’s scope and submission guidelines. Cytotherapy, in line with the society’s vision towards more cohesive and committed communications, has developed upon its respectable foundations. 

In 2019, Cytotherapy‘s record impact factor of 4.297 was also reported for 2018. This number, derived from ScienceDirect, Elsevier’s online portal, measures “the average number of citations received in a particular year by papers published in the journal during the two preceding years” (Clarivate Analytics, 2019). 

Cytotherapy Cover

The 2019 Cytotherapy Editorial Board

Cytotherapy, the official journal of the  ISCT, publishes novel and innovative results from high quality scientific and clinical studies in the fields of cell and gene therapy. Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease and describing important advances in cell/gene-based product manufacturing and validation are welcomed. Results of clinical studies evaluating the safety and efficacy of cell and gene therapies in early and late phase trails are also of interest. In addition to short reports and full-length articles, the journal also accepts editorials addressing emerging trends and potential controversies in the field, and review articles summarizing bodies of work that have made lasting impacts in the field.

Senior Editor

Donald G. Phinney, PhD

Scripps Florida
Jupiter, Florida
United States

Associate Editors

Massimo Dominici, MD

University of Modena and Reggio Emilia, Modena, Italy

Oscar Lee, MD, PhD

National Yang-Ming University, Taipei, Taiwan

Luis Ortiz, MD

University of Pittsburgh, Pittsburgh, Pennsylvania, United States

John Rasko, MBBS, PhD

Royal Prince Alfred Medical Centre, Newtown, Australia

Sowmya Viswanathan, PhD

University Health Network, Toronto, Ontario, Canada

Editorial Board Members

2019 marked the start of the ISCT Talking With Giants series.

Featured at the start of several issues of Cytotherapy, this inspirational series provides both a celebration of the contributions of scientific leaders in cell and gene therapy, and an opportunity for readers to learn more about the passion, motivations, and visions of these giants within an informal question and answer format. 

ISCT Talking With Giants 2019

DARWIN J. PROCKOP

MALCOLM BRENNER

NANCY COLLINS

ROBERT PRETI

HAL E. BROXMEYER

Inaugural Insta-Your-Cell Photo Challenge

IYC-1

1st Place
Imam Rosadi (Indonesia)

IYC-2

2nd Place
Ahmad Galuta (Canada)

IYC-3

3rd Place
Saba Ghassemi
 (United States)

The Insta-Your-Cells photography competition showcased images taken in laboratory environments by our members. This competition featured over 50 unique and fascinating photos taken at the cellular level, and served to visualize some aspects of the intricate work that make scientific advancement possible in our field. 

Winners are featured on the front covers of Cytotherapy from June 2019 onwards, with the 1st place winner being featured in January 2019. 

Additional prizes included complimentary conference registration (1st place), and pre-conference day registration (2nd and 3rd place)
Making an impact… factor

Our Three Pillars

ISCT’s Committees represent an invaluable network of dedicated scientific leaders that work to develop and deliver papers that advance the field of cell and gene therapy. Below are a featured series of their publications throughout this year. 

For more information on contributing to one of our committees, take a look at our Committee Directory.


Co-Chaired By:


Rachele Ciccocioppo, MD
University of Verona, Verona, Italy
Giuseppe Orlando, MD, PhD
Wake Forest University, Winston-Salem, NC, USA

Perspectives of the International Society of Cell & Gene Therapy Gastrointestinal Scientific Committee on the Intravenous Use of Mesenchymal Stromal Cells in Inflammatory Bowel Disease.

Authors: Ciccocioppo R, Baumgart DC, Dos Santos CC, Galipeau J, Klersy C, Orlando G.


Chaired By:


Jacques Galipeau, MD
University of Wisconsin-Madison, Madison, WI, United States

Mesenchymal stem versus stromal cells: International Society for Cellular Therapy Mesenchymal Stromal Cell committee position statement on nomenclature.

Authors: Viswanathan Y, Shi Y, Galipeau J, Krampera M, Leblanc K, Martin I, Nolta J, Phinney DG, Sensebe L.

Manufacturing mesenchymal stromal cells for clinical applications: A survey of Good Manufacturing Practices at U.S. academic centers.

Authors: Phinney DG, Galipeau J.

Challenges for mesenchymal stromal cell therapies.

Authors: Martin I, Galipeau J, Kessler C, Leblanc K, Dazzi F.


Co-Chaired By:


Sai-Kiang Lim, PhD
A*STAR Institute of Medical Biology,
Singapore

Bernd Giebel, PhD
University of Duisburg-Essen
Essen, Germany

Defining mesenchymal stromal cell (MSC)-derived small extracellular vesicles for therapeutic applications

Authors: Witer K, Van Balkom, B, Bruno S, Choo A, Dominici M, Gimona M, Hill A, De Klejin D, Koh M, Lai R, Mitsialis S, Ortiz L, Rohde E, Asada T, Toh W, Weiss D, Zheng L, Giebel B, Lim S.

Co-Chaired By:


(2018-2020) Kenneth Micklethwaite, MD The Westmead Institute for Medical Research Sydney, Australia

(Jan 2018 – Present) Patrick Hanley, PhD Children’s National Medical Center Washington, DC, United States
Championed by Patrick Hanley and Mark Lowdel, the March 2019 issue of Cytotherapy featured a series of focused papers by ISCT’s Immuno-Gene Therapy Committee.
Featuring a series of unique publications that provide a novel focus on both testing and manufacturing cell therapy products, this issue of Cytotherapy provides timely and useful scientific discourse on the state of translational science in cell and gene therapy. 
 
The field of cell and gene therapy has rapidly grown from a number of small, isolated centers 20 years ago to specialized cell and gene therapy centers worldwide. Nonetheless, despite the recent high-profile acquisitions of small companies developing cell therapies such as chimeric antigen receptor (CAR)-T products, the bulk of developments in this field remain led by academic drug developers, most of whom are focused on a treatment for their group of patients with local production and supply and are not aware of the challenges of supplying the national or global patient need.

Critical testing and parameters for consideration when manufacturing and evaluating tumor–associated antigen-specific T cells

Authors: Tanna J, Ulrey R, Williams K, Hanley P. 

The past year has seen remarkable translation of cellular and gene therapies, with U.S. Food and Drug Administration (FDA) approval of three chimeric antigen receptor (CAR) T-cell products, multiple gene therapy products, and the initiation of countless other pivotal clinical trials. What makes these new drugs most remarkable is their path to commercialization: they have unique requirements compared with traditional pharmaceutical drugs and require different potency assays, critical quality attributes and parameters, pharmacological and toxicological data, and in vivo efficacy testing. What’s more, each biologic requires its own unique set of tests and parameters…

Mesenchymal stromal cell therapy: progress in manufacturing and assessments of potency

Authors: Robb K, Fitzgerald J, Barry F, Viswanathan S.

Mesenchymal stromal cell (MSC) therapies have been pursued for a broad spectrum of indications but mixed reports on clinical efficacy have given rise to some degree of skepticism regarding the effectiveness of this approach. However, recent reports of successful clinical outcomes and regulatory approvals for graft-versus-host disease, Crohn’s disease and critical limb ischemia have prompted a shift in this perspective. With hundreds of clinical trials involving MSCs currently underway and an increasing demand for large-scale manufacturing protocols, there is a critical need to develop standards that can be applied to processing methods and to establish consensus assays for both MSC processing control and MSC product release…

Tumor-infiltrating lymphocytes: Streamlining a complex manufacturing process

Authors: Hopewell E, Cox C, Pilon-Thomas S, Kelley L.

Adoptive cell therapy of tumor-infiltrating lymphocytes has shown promise for treatment of refractory melanoma and other solid malignancies; however, challenges to manufacturing have limited its widespread use. Traditional manufacturing efforts were lengthy, cumbersome and used open culture systems. We describe changes in testing and manufacturing that decreased the process cycle time, enhanced the robustness of critical quality attribute testing and facilitated a functionally closed system. These changes have enabled export of the manufacturing process to support multi-center clinical trials.

Characterization of human natural killer cells for therapeutic use

Authors: Wagner A, Alici E, Lowdell M. 

As a part of the innate immune system, natural killer (NK) cells are cytotoxic lymphocytes that can exert cytotoxic activity against infected or transformed cells. Furthermore, due to their expression of a functional Fc receptor, they have also been eluded as a major effector fraction in antibody-dependent cellular cytotoxicity. These characteristics have led to multiple efforts to use them for adoptive immunotherapy against various malignancies.  There are now at least 70 clinical trials testing the safety and efficacy of NK cell products around the world in early-phase clinical trials…

Manufacturing chimeric antigen receptor T cells: issues and challenges

Authors: Roddie C, O’Reilly M, Dias Alves Pinto J, Vispute K, Lowdell M. 

Clinical trials of adoptively transferred CD19 chimeric antigen receptor (CAR) T cells have delivered unprecedented responses in patients with relapsed refractory B-cell malignancy. These results have prompted Food and Drug Administration (FDA) approval of two CAR T-cell products in this high-risk patient population. The widening range of indications for CAR T-cell therapy and increasing patient numbers present a significant logistical challenge to manufacturers aiming for reproducible delivery systems for high-quality clinical CAR T-cell products. This review discusses current and novel CAR T-cell processing methodologies and the quality control systems needed to meet the increasing clinical demand for these exciting new therapies.

T-cell receptor gene-modified cells: past promises, present methodologies and future challenges

Authors: Rego R, Morris E, Lowdell M.

Immunotherapy constitutes an exciting and rapidly evolving field, and the demonstration that genetically modified T-cell receptors (TCRs) can be used to produce T-lymphocyte populations of desired specificity offers new opportunities for antigen-specific T-cell therapy.
Overall, TCR-modified T cells have the ability to target a wide variety of self and non–self targets through the normal biology of a T cell. Although major histocompatibility complex (MHC)–restricted and dependent on co-receptors, genetically engineered TCRs still present a number of characteristics that ensure they are an important alternative strategy to chimeric antigen receptors (CARs), and high-affinity TCRs can now be successfully engineered with the potential to enhance therapeutic efficacy while minimizing adverse events…

Chemistry, manufacturing and controls for gene modified hematopoietic stem cells

Authors: Soni S, Kohn D. 

Gene modification of hematopoietic stem cells is increasingly becoming popular as a therapeutic approach, given the recent approvals and the number of new applications for clinical trials targeting monogenetic and immunodeficiency disorders. Technological advances in stem cell selection, culture, transduction and gene editing now allow for efficient ex vivo genetic manipulation of stem cells. Gene-addition techniques using viral vectors (mainly retrovirus- and lentivirus-based) and gene editing using various targeted nuclease platforms (e.g., Zinc finger, TALEN and Crispr/Cas9) are being applied to the treatment of multiple genetic and immunodeficiency disorders. Herein, the current state of the art in manufacturing and critical assays that are required for ex vivo manipulation of stem cells are addressed. Important quality control and safety assays that need to be planned early in the process development phase of these products for regulatory approval are also highlighted.

Proposal for the International Society for Cell & Gene Therapy position statement on assays for the quality control and potency assessment of adoptive cellular immunotherapies

Authors: Weil B, Hanley P, Lowdell M. 

Translation of cell and gene therapies from pre-clinical experiments to clinical trials and final drug licensing brings requires the development, verification and even validation of the assays essential for the definition of the drug product. The technical and scientific challenges in doing this are far greater than they seem at first and are compounded by a lack of approved standards for assays used to support (c)GMP manufacture. This paper highlights some of those challenges and proposes solutions based on the experience of our colleagues using similar assay platforms in regulated pathology laboratories.


Organized by the ISCT Immuno and Gene Therapy Committee
Chaired by Sandeep Soni, MD, Stanford University, Palo Alto, CA, United States

The ISCT Scientific Signature Series offers the unique opportunity to gather key opinion leaders in the chosen field to discuss, present and develop position statements to move the field forward. These events are aimed at driving thought leadership by providing content and building collaboration around concept papers, consensus statements, clinical networks, regulatory proposals, and recommendations for investment in clinical and basic research in cell and gene therapy medicine.

ISCT’s North America Regional Committee 2019 Meeting in Madison, USA featured our annual scientific signature series – a full-day symposium event that gathered key opinion leaders in Gene Therapy to drive the field forward through the development and discussion of position statements in a public forum. 

This year’s Scientific Signature, “Novel Ex-Vivo Gene Therapy Approaches and Emerging Gene Editing Technologies” focused on perspectives from translational science, manufacturing, and regulation. The symposium consisted of four sessions, each consisting of three topic presentations, followed by a thirty minute open discussion:

    1. HSPC Selection, Conditioning, and the Synthetic Biology Platform
    2. The CRISPR-CAS Gene Editing System
    3. Advances in Gene Therapy Technologies and Applications
    4. Regulatory & Manufacturing Considerations

Sponsors of ISCT’s Scientific Signature Series 2019

Crispr-Logo-1-300x200


The International Society for Cell and Gene Therapy (ISCT) and the AABB have an ongoing series of projects overseen by the ISCT-AABB Joint Working Group. The main project team managed by the Joint Working Group focuses on research on pooled Human Platelet Lysate (pHPL).

In 2019, the project team released an inaugural jointly-published manuscript across both societies’ peer-reviewed journals: Cytotherapy and Transfusion.

Alongside this exciting progression, 2019 marks a year of expansion for the working group, as a second project team, focusing on biopreservation, has been established to advance cellular therapy product stability. For more information, read Lizette Caballero’s article in the February 2019 issue of Telegraft and the AABB’s update document.

ISCT’s scientific community operates on a global scale, providing expert review and support in the interest of proven and safe research practices.

Through liaison work with standards organizations, a broader scientific community, and with investigatory stakeholders, ISCT conducts thought leadership in the field of cell and gene therapy on a global scale. Below is a snapshot of some actions taken by ISCT committees over the past year:

TOTAL

0
Scientific Consultations and Endorsements

INTERNATIONAL

International Standards Organization
MSC and Stromal Cell Definitions

International Standards Organization*
Standards for UC-MSC Banking
Standards for BM-MSC Banking

*ISCT has provided ongoing liaison work for two biobanking standards documents, providing consultation around nomenclature for the international standards organization
0
Consultations for the International Standards Organization

NORTH AMERICA

Re: Fetal Tissue Research Amendment
Letter of Support to Mark Pecan of the HHS 

Re: NIH Requirements for Fetal Tissue Research
ISSCR-led Scientific Coalition Letter 

Re: NIH Requirements for Fetal Tissue Research
US Senate Member-led Letter 

0
Signed Letters of Endorsement

ISCT offers regulatory review and consultation from cell and gene therapy experts for regulatory authorities worldwide.

Review and recommendations are made by our global task force, as well as by regional Legal and Regulatory Affairs Committees in our North America, Europe, and Australia and New Zealand regions. Each consultation consists of significant comments, suggested revisions, and edits for regulatory documentation. Below are a series of major guidance reviews from 2019:

TOTAL

0
Regulatory Consultations and Supports

INTERNATIONAL

Stakeholder Consultation for PIC/S GMP Guide

Annex 2A – MANUFACTURE OF ADVANCED THERAPY MEDICINAL 3 PRODUCTS FOR HUMAN USE

Annex 2B – MANUFACTURE OF BIOLOGICAL MEDICINAL 4 SUBSTANCES AND PRODUCTS FOR HUMAN USE

0
Consultations

NORTH AMERICA

ISCT-Endorsed Letter
Letter of Recommendations to the HRSA regarding the Zika Virus

0
Signed Letter of Recommendations

EUROPE

European Medicines Agency
Guideline on quality, non-clinical and clinical requirements for investigational ATMP in CT (EMA-CAT-852602-2018)

European Medicines Agency
Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (EMA/CAT/GTWP/671/2008)

0
Regional Consultation​s

AUSTRALIA AND
NEW ZEALAND

Australian National Gene Technology Scheme
Implementing Recommendations of the Third Review of the Gene Technology Scheme: Phase 1 Consultation 

0
Regional Consultations

ISCT hosts liaison meetings for stakeholders in the field of cell and gene therapy to coordinate with national regulatory authorities. Each of these liaison meetings consists of a series of focused presentations on the recent developments and urgent concerns that concern stakeholders in the community and industry of cell therapy. These presentations are directed both at regulatory authorities and at fellow stakeholders in attendance, and are designed to promote up-to-date and comprehensive policy development. 

Throughout 2019, two series of liaison meetings held their latest iteration:

Since 2004, ISCT has led over twenty invited stakeholder organizations in an annual face-to-face meeting with the FDA Cell Therapy Liaison Meeting (CTLM). 

This year’s presentations consisted of:

Regulation of Biobanking Material for Future Products
Mahendra Rao, MD, PhD

Collection of MNCs for Immune Effector Cells
Jay Raval, MD

Minimum Characterization Criteria for Clinical Grade iPSC Products
Aisha Khan, MS, MBA

Early Interaction Mechanisms for Tool/Device Developers
Michael Mendicino, PhD

Since 2014, the Health Canada Biologics and Genetic Therapies Directorate has hosted regular bilateral meetings with the Cell Therapy Stakeholder Group to promote regulatory dialogue that identifies critical information in easing the translation of cell therapy and regenerative medicine innovations into Canadian healthcare. 

Co-Chaired By:


Karen Nichols, Esq. 

VP, Regulatory and Quality, Magenta Therapeutics, United States

Dominic Wall, PhD, FFSc(RCPA)

CSO Cell Therapies Pty Ltd, Peter MacCallum Cancer Centre, Australia

The ISCT Global Regulatory Perspectives (GRP) workshop represents the diverse activities and partnerships between international regulatory bodies, industry, clinicians and academia. Steered by representatives from the Society’s Legal and Regulatory Affairs Committees from Asia, Australia, Europe, North America, and South and Central America, the GRP workshop takes place once every year as a featured full-day event during the ISCT’s Annual Scientific Meeting. With regulators in attendance from every region of ISCT membership, GRP programming targets a truly global perspective while maintaining focus on key emergent issues in the cell and gene therapy field.

Each GRP Workshop consists of alternating speaker sessions and case study-based panel discussions. 

During ISCT 2019 Melbourne, the GRP Workshop covered topics including the following:

  • CMC Considerations (including testing) for Pediatric Treatments
  • Risk Assessment for Administration of Non-conforming Products
  • Clinical Strategies for Treatment of Pediatric Diseases
  • Regulatory Convergence

Catered to the industry community within the field of cell and gene therapy, the ISCT Commercialization Signature Series provides an overview and focused discussion of the state of market access and adoption for the sector. Each signature event is designed to bring not only leading positions and information into discussion, but also to facilitate networks across key peers in academic and industry positions. 

The third ISCT Commercialization Committee Signature Series was held this past January at the Phacilitate Cell & Gene Therapy World 2019 event in Miami. Titled “Patients First: Achieving Global CGT Adoption,” the session focused on strategies and positions tailored to leveraging the increasingly robust investments, interest, and scientific advancement in CGT to successful manufacturing, distribution, and legislative approval. 

This timely conference session focused on three general areas: 

1. The business of cell & gene therapy: state of the financial markets
2. Getting the product to the patient: global manufacturing strategies
3. Overcoming cell & gene therapy adoption barriers: positioning clinical evidence and pricing models for success

Sponsored by our 2019 Industry Members

Membership: 49% outside of the United States from 60+ countries

Regional Forum and Meeting Partners

ISCT ANZ-ASSCR-AGCTS
Joint Scientific Meeting


 

ISCT SCA Regional Forum at SOMICET Annual Congress


 

SCSS-ISCT
Joint Symposium 


 

ISCT ANZ-ASSCR-AGCTS
Joint Scientific Meeting


 

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ISCT SCA Regional Forum at SOMICET Annual Congress


 

SCSS-ISCT
Joint Symposium 


 

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March 27


Akron Biotech
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GE Healthcare

October 30


 

Akron Biotech
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November 21


 

IQVIA

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Atvio Biotech
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Joint Session Partners

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A Legacy of Engagement

Since 1992, ISCT has had connected more than 17,000 delegates through our Annual Meetings and communicated with more than 30,000 cell and gene therapy professionals at cutting-edge meetings, events, webinars and seminars to translate the advancement of research into clinical adoption and standard of care over the past 26 years.

ISCT 2019 Melbourne

Chaired By:

Ngaire Elwood

Kunihiko Suzuki

Siok Tey

Ngaire
Elwood

Kunihiko
Suzuki

Siok
Tey

 

Every year, the ISCT Annual Meeting delivers a highly curated scientific program showcasing the latest clinical results and technologies expanding global access to new and emerging cell and gene therapies.

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TELEGRAFT LIVE

Making Connections Before the Meeting

The Meeting is Underway!

Scientific, Industry, and ESP Networking

The Celebrations Continue

“THE CELLFIE”

ISCT2019_SELFIE
ISCT 2019 Melbourne

ISCT Regional Meetings 2019

ISCT NA Regional Meeting
September 13-14 – Madison, WI, USA

“Engineering the Future of Cell and Gene Therapies”

 

ASSCR-ACGTS-ISCT Joint Scientific Meeting:
November 13 – Brisbane

“From Stem Cells to Genes to Therapies”
 

ISCT-SCSS Joint Scientific Meeting
November 13-15 – Singapore

“Frontiers in Cell Therapy”
 
 

ISCT SCA At 5th SOMICET Congress
October 21-23 – Mexico City

“5th International Meeting on Stem Cells and Regenerative Medicine”