From The Editor | October 30, 2018

ARM's CEO Recaps 2018 Meeting On The Mesa


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

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


Cell & Gene Meeting on the Mesa took place last month in La Jolla, CA, and it’s no surprise that ARM delivered meaningful data and addressed cutting-edge research to more than 1,200 attendees. I caught up with ARM’s CEO Janet Lambert after the Meeting to get her take on top highlights, the most informative panel discussions, as well as a look ahead to next year’s event. Here’s what she had to say.

Harris: Recap Cell & Gene Meeting on the Mesa’s top highlights.

Lambert: Cell & Gene’s Meeting on the Mesa’s growth mirrors the growth of the sector. We started in 2006 with the Scientific Symposium, which expanded in 2011 to include the regenerative medicine community’s first-ever investor and partnering forum. Now the Cell & Gene Meeting on the Mesa is well known as the sector’s can’t-miss premier annual event — a three-day conference bringing together senior executives and top decision-makers in the industry with the scientific community to advance cutting-edge research into cures. The meeting has more than quadrupled in attendance since its founding, growing from 350 attendees to attracting 1,200+ attendees, fostering key partnerships through more than 2,000 one-on-one meetings, highlighting the significant clinical and commercial progress in the field.

There is excitement, energy, and optimism sector-wide. With the arrival of Kymriah, Yescarta, and Luxturna to the market, a well-stocked late-stage clinical pipeline, the field is no longer addressing commercialization from a hypothetical standpoint. Despite the very real commercialization hurdles ahead, there was palpable energy and optimism throughout the meeting; there is very much the sense that this sector has arrived, and stakeholders are ready to collaborate to address issues relating to value-based pricing and reimbursement models; regulatory convergence within the U.S. and EU/Europe; industrialization, scale-up, and manufacturing; development and implementation of standards sector-wide, etc.

And, we announced that the meeting will move from the Estancia (where it has long been held):

  • Cell & Gene Meeting on the Mesa — New Location in 2019
  • Park Hyatt Aviara – Carlsbad, CA | Oct 2-4, 2019
  • Just 20 minutes north of the Estancia
  • Full buy-out of the property, enabling more than twice as many attendees to stay on site
  • 78,000 sq. ft. of meeting and event space — more than two times the size of the Estancia

Harris: What were the biggest topics discussed at the Meeting, and why?

Lambert: Many valuable topics were discussed. Here’s a list of some of the most important data shared with attendees:

  • Reaching the market is a significant achievement, however, the sector must successfully address commercialization challenges: implementation of value-based pricing and reimbursement models, manufacturing, industrialization/ability to scale.
  • Creating and maintaining a supportive policy environment (RMAT, FDA, EMA)
  • Clinical progress and efforts being made to accelerate patient access — accelerate approval pathways, regulatory convergence; value-based payment and reimbursement models and arrangements.
  • The role large pharma and large-cap biotech currently and will hold in the sector, as an R&D and commercial partner in developing and marketing cell and gene therapies globally
  • Cell and gene therapy pipeline, especially in regard to meeting the widespread unmet medical needs of rare diseases, as well as oncology.

Harris: What were some of the most informative presentations or sessions? Why?

Lambert: Here is a list of some of the most valuable presentations, including videos of each panel.

  • Initial plenary session: Charting the Path – Lessons from the Pioneers of Cell and Gene Therapy Commercialization Moderated by Robert Preti, Ph.D., President and CEO, Hitachi Chemical Advanced Therapeutics Solutions; GM, Hitachi Chemical Regenerative Medicine Business Sector (and current ARM Chairman), executives from three of the companies who have been at the forefront of cell and gene medicine (David Lennon, Ph.D., President, AveXis; Ron Philip, SVP, Head of Global Commercial, Spark Therapeutics; and Pascal Touchon, SVP and Global Head, Cell and Gene, Novartis Oncology). The shared insights on the recent significant scientific, regulatory and technical developments have contributed most to the sector’s current success.
  • Peter Marks, U.S. FDA CBER Director: FDA’s Efforts to Advance the Development and Approval of Cellular and Gene Therapies
  • The panel on RMAT Regulatory Convergence, an interactive conversation with regulators and regulatory experts focused on expediting regulatory convergence as well as identifying actions that can be taken to further facilitate global convergence.

Topics included ongoing efforts and priorities for regulatory convergence by focus area including CMC, nonclinical and clinical. Chaired by Melody Eble, Pharm.D., Director, Global Regulatory Affairs, Scientific Innovation Projects – Regenerative Medicine and Digital Technology, Janssen R&D; with speakers: Antony Appleyard, Ph.D., Technical Director Regulatory, Diamond Biopharm; Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA); Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA); Jiwen Zhang, Ph.D., President, Standards Coordinating Body (SCB); Executive Director, Regulatory Affairs, Tmunity Therapeutics.

  • Opportunities and Challenges in Rare Disease. Cell and gene therapies have moved from promise to reality in 2018, with rare diseases often in the vanguard of these remarkable new living medicines. Many firms have built substantial franchises in rare diseases, but there is still great unmet need and the potential to displace and disrupt existing therapies. This panel explored the opportunities and challenges in rare diseases across cell and gene therapy modalities as these therapies come to market; exploring technical challenges, licensing, adoption and reimbursement in healthcare systems and public or patient attitudes to this medical revolution. Chaired by Keith Thompson, CEO, Cell and Gene Therapy Catapult, speakers: Max Colao, Chief Commercial Officer, Abeona Therapeutics; Geoff MacKay, CEO, AVROBIO; Matthew Patterson, President and CEO, Audentes Therapeutics; Alvin Shih, M.D., CEO, Enzyvant.

Harris: Share some of the cutting-edge research addressed at the Meeting.

Lambert: The third and final day of the event took place at the Salk Institute for Biological Studies. The Scientific Symposium, now in its 13th year, examined the imminent scientific and ethical challenges facing cell and gene therapy research. The format was designed around panel discussions exploring topics ranging from basic science to revolutionary techniques and included keynote presentations from world renowned researchers.

Panels and featured talks covered the following:

  • Self-Organizing Synthetic Human Embryos and Organoids Towards Curing Huntington’s Disease
  • Ex Vivo Gene Therapy: Using Blood Stem Cells to Treat Genetic Disorders, highlighted the potential of this approach in different disorders illustrating the technologies for hematopoietic stem cell gene-correction and the engagement of stem cells to prevent tissue degeneration.
  • Advanced Therapies for Skeletal Muscles focused on the central role of muscle stem cells in skeletal muscle homeostasis and repair.
  • Gene and Antisense Therapy for Neurodegeneration was an in-depth update on clinical and preclinical programs of AAV9 gene therapy for spinal muscular atrophy, antisense oligonucleotide therapy for ALS and growth factor gene therapy for Alzheimer’s disease.
  • Using Stem Cells to Study Neuropsychiatric Disorders focused on the ability to generate neural derivatives from accessible somatic cells from patients with mental disorders (and appropriate controls) is beginning to make these heretofore “mechanistically-unapproachable” complex conditions amenable to rigorous molecular and cellular interrogation.
  • Gene Editing in Human Embryos

All videos from this year’s Scientific Symposium: