Session Details

Plenary Session 4 – Commercial Strategies for Expanding Global CGT Access
Friday, May 31, 2019 01:45 PM - 03:15 PM
Plenary Hall
Chair:
Bill Milligan, Steminent Biotherapeutics, Canada

Over the past two decades, the field of cell and gene therapy product development has emerged as a substantial, promising new platform for the future of health care. Successful strategies to translate and advance CGT candidates from discovery, through clinical development, and to commercialization has validated the CGT business model.  CGT development companies have used , regional capitalization, IPOs, regulatory harmonization and in-license / out-license partnering , regionally and globally, as strategic tools for increasing their access to capital, adding development / manufacturing resources,  extending / accelerating their access to patients globally, and thereby maximizing the value of their CGT products. The speakers in this session will share how they have utilized these strategies in expanding their regional and global expansion of CGT products to build value and access world-wide markets.

Speakers:
Bringing Cellular Medicines to Market for the Treatment of Serious and Life-Threatening Inflammatory Conditions
Silviu Itescu, MBBS, Mesoblast, Australia

Mesoblast is developing allogeneic cellular medicines using its proprietary technology platform to target complex diseases where inflammation plays a critical role. In the US, Mesoblast has three product candidates in Phase 3 and has commenced the Biologics License Application (BLA) process for its allogeneic cellular medicine, remestemcel-L, for the treatment of steroid-refractory acute graft versus host disease (aGVHD) in children. Its other Phase 3 programs are for the treatment of advanced heart failure and chronic low back pain due to degenerative disc disease, with clinical results expected within the next 12 months. Mesoblast already receives revenues from two allogeneic cell therapy products marketed by its licensees in Japan and in Europe. Using industrial-scale proprietary manufacturing processes, Mesoblast anticipates being able to meet the growth trajectory and commercial demand for its therapies as they become approved and are marketed in various jurisdictions.

A Global View of CAR T Cell Therapies from the Academic Launching Pad
Bruce Levine, PhD, University of Pennsylvania, United States
Since the 1990’s, we have conducted clinical trials of gene modified T cells.  Chimeric antigen receptor (CAR) T cells targeting CD19 on B cells leukemias and lymphomas have induced durable complete responses in patients who are relapsed or refractory to all other available treatments.  Transfer of the cell processing and analytics technology from academia to an industry large-scale GMP facility included areas of manufacturing process and analytical technology. Diverse technology transfer team participants from academia, GMP production, Technical development, Quality Assurance, and Regulatory. This allowed the conduct of industry sponsored global multi-center clinical trials in relapsed/refractory acute lymphoid leukemia in children and young adults as well as in diffuse large B cell lymphoma that were the basis for FDA, EMEA, Canada, Switzerland, and Australia approvals, with final action pending in Japan (Kymriah, Novartis).  The road forward for wide patient access to these uniquely personal cellular therapies depends not only on scientific progress in targeting, gene modification and cellular manipulation to enhance specificity and potency, but also on meeting automation, engineering, logistics, clinical site onboarding, and health policy challenges.

Innovating Cancer and Regenerative Disease Treatment in China
Tony Liu, MBA, CBMG, China

Cellular Biomedicine Group, Inc. (NASDAQ: CBMG) develops proprietary cell therapies for the treatment of cancer and degenerative diseases. We conduct immuno-oncology and stem cell clinical trials in China using products from our integrated GMP laboratory. Our GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards.

Cellular Biomedicine Group brings together the world’s latest technology with industry-leading scientists and professors to research and develop safe treatments for those with serious conditions and diseases.

We are proud to have a share in an emerging field that has great potential for a significant positive impact on society. Our directors, scientists, doctors and employees share a sense of responsibility that ensures we maintain stringent international safety and quality control standards and focus on the patients and caregivers who will benefit the most from the application of breakthroughs in stem cell technology and immuno-oncology.