DEAR ISCT COMMERCIALIZATION COMMITTEE AND INDUSTRY MEMBERS,

The ISCT Commercialization Committee (CC) has established itself as a pillar of the International Society for Cellular Therapy (ISCT) and a key player in influencing the translation of cellular therapy products into therapeutic opportunities. This Committee was pioneered under the leadership of Dr. Robert Deans (Athersys, Inc) for the last four years, and following his leadership has been an honour.

Working through in the CC, we will continue to follow his legacy forward in the years to come.

My personal vision is to establish the ISCT CC as a leading body addressing the access of cellular therapy products to patients and the market. To achieve that leadership, the CC will focus on three key aspects:

• Product and process development/manufacturing • Generation of clinical evidence • Market access

Cell therapy faces an immediate challenge in achieving robust, transferable and comparable manufacturing processes together with developing business models, regulatory practice and reimbursement strategies that will enable market access. The generation of clinical evidence in cell therapy may be seen as less divergent from traditional drug development but will need to address aspects of feasibility and compliance with medical practice expectations. As key stakeholder in ISCT with both academic and industry membership, the CC is well positioned to address these challenges. The aim is to achieve this together, reaching out to partner organizations representing academia, industry and patient associations. The ultimate goal of the CC is to drive value for key stakeholders in the cell therapy field, including investors, pharma industry, regulators, payers, and above all the end consumers, the physicians and patients.

THE YEAR IN REVIEWThe last 12 months have been particularly active, with multiple achievements from our subcommittees:

Process and Product Development (PPD) Subcommittee (Chair: Ohad Karnieli – Pluristem Therapeutics)

The PPD subcommittee has provided leadership in aspects relating to cell therapy product development with tangible deliverables. Several white papers have been published or are in development:

• Cryopreservation: Champion: Erik Woods (Cook Medical) and Aby Mathew (BioLife Solutions)

• Process Development: Champion: Tom Brieva (Celgene) and Andy Campbell(Life Technologies)

• Cell Delivery: Champion: Knut Niss (Novartis)• Stability: Champion: Ohad Karnieli (Pluristem Therapeutics)• Angiogenesis: Champion: Claudia Zylberberg (Akron Biotech),

joint with ARM Potency Group

This group has also established links and activities with other organisations:

• ECI Manufacturing of Cell Based Therapies Conference (Sponsored Plenary, January 2015) Society for Cryobiology joint session at the ISCT 2014 Annual Meeting

• Alignment with United States Pharmacopoeia (USP), leading to invitation of ISCT as a Voting Organizational Member of USP

Over the next year the PPD subcommittee will continue to strive to sustain its leadership on process and product development through dissemination of publications, education events and partnerships with other organizations in the field, while ensuring maintenance and expansion of the sub-committee membership.

Business Models and Cost of Goods Subcommittee (Chair: Bill Milligan – Steminent Biotherapeutics)

The Cost of Goods (CoGs) sub-committee has completed a cell manufacturing survey among ISCT member companies, which have highlighted the state of the industry with regard to cell

YEAR END REPORTISCT Commercialization Committee

2014

manufacturing and CoGs management. The objectives are to increase awareness and educate through discussion opportunities via webinars, presentations and a published road map white paper on CoGs considerations (Q2 2015). The activity of this group is very relevant as the thoughtful management of CoGs throughout development is critical to achieve sustainable business models with broad market access for successful cell therapy product candidates. These are recognized as significant challenges in bringing cell therapy products to the market as documented by some well-known recent examples. I see in particular a great opportunity for more interaction internally with the reimbursement sub-committee and externally with other key stakeholders like ARM.

Reimbursement Subcommittee (Chair: Richard Maziarz – Oregon Health Sciences University)

Indeed, the topic of reimbursement is key to market access and a successful delivery of cell therapy products to patients. A revitalization of this team will be the main focus over the next few months, while maintaining dialogue with relevant partners like ARM, but also Payers and HTA organizations. Planned activities include a survey to identify and confirm areas of focus.

The Commercialization Committee has also engaged external stakeholders for projects and has recently submitted an Ancillary Materials white paper (Champion: Lynn Csontos/Jennifer Solomon, STEMCELL Technologies) in conjunction with the Foundation for the Accreditation of Cellular Therapy (FACT).

STRENGTHENING OUR PRESENCEA particular strength of the CC has been the interaction and cross-over of activities with key opinion leaders in the ISCT Scientific Committees. This has included successful exchanges with the MSC, Immunotherapy and Pulmonary committees. In particular the Pulmonary Committee, which delivered the first Scientific Signature Series on Tracheal Bioengineering.

The CC continues to be a forum where Industry and Academic members have an opportunity to be informed, discuss and impact areas relevant to their common interest and professional activity. This has included the opportunity to influence regulation and legislation, including the ATMP guidelines in Europe, ISO and FACT standard setting, aspects of intellectual property and several opportunities to exchange and disseminate information at the ISCT Annual Meeting. I believe that the CC continues to add value to their members and to

the cell therapy field.

Going forward the objective is to sustain and reinforce the CC as a value-adding forum for its members, for cell therapy and for patients. In addition to the publications and dissemination of information, the CC will focus over the next year in three critical areas:

• Expanding the interactions with significant partner organizations through a constant effort to identify and engage through partnerships. These actions are seen as important to enhance the presence of ISCT but also to align activities, share perspectives and collaborate in influencing relevant key stakeholders. The geographic span is global with strengthening of current activities in the US and Europe with a growing focuses on China, Japan and Asia in general.

• Ensure that the CC remains a forum where leaders in cell therapy, from Academia and Industry, continue to share their views and experiences to foster activities from translation to market access. Here the focus will naturally be on the current product development challenges and the late steps of market access with business models and reimbursement.

• The third and most relevant will be the focus on the final objective of all cellular therapies, the patient. The CC is already identifying and interacting with patient associations. This will ensure that efforts to produce, document and bring to market cellular therapy products reflects the needs, concerns and expectations of those for whom they are being developed.

JOIN USI believe that the CC represents a unique opportunity to stay informed and influence the challenges and successes in cellular therapy. At the end of each year it is time to ensure the continued interest and expanded membership in ISCT and its groups and activities. It is important that the CC addresses the needs of its individual and corporate members. Without Industry Members, there would not be a Commercialization Committee and we look forward to welcoming you to ISCT in 2015.

In ISCT (and particularly the CC) there is an enormous amount of knowledge and expertise in cell therapy. This expertise is coupled with amazing individual and organizational passion for the field to achieve new treatments for patients. I believe that the CC, as a pillar of ISCT, is well positioned to deliver and achieve the ISCT mission of driving the translation of cellular therapies for the benefit of patients worldwide.

Sincerely,

MIGUEL FORTE

Chair, ISCT Commercialization CommitteeSVP Clinical and Regulatory – Chief Medical Officer • TxCell SA

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