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In 2020, the FDA approved the third CAR-T cell treatment for cancer, Tecartus to treat mantle cell lymphoma, but it also sent multiple developers of gene and cell therapies back to the drawing board—demanding additional data to support approval of their products. These high-profile rejections signaled a new approach to the regulatory review of gene and cell therapies. 

The FDA has hired more than 50 new reviewers in anticipation of receiving 200 applications per year from companies hoping to start clinical trials of gene and cell therapies and issuing 10 to 20 new approvals a year by 2025. This represents both an opportunity and great challenge for gene and cell therapy developers, who will need to learn how to navigate the regulatory process effectively. 

The potential impact of regulatory surprises on biopharma developers and their investors was perhaps best demonstrated by Biomarin. After years of developing a gene therapy to treat hemophilia, it saw its shares plummet in August 2020 on news that the FDA refused to approve the product without an additional two years’ worth of data on its durability. 

We’ll review the latest developments in the regulatory review process for gene and cell therapies, offering advice that companies can use to streamline their interactions with regulators, both in the U.S. and overseas.  

Topics include: 

  • Understanding the FDA’s new guidance documents covering product development, accelerated approval pathways and other topics specific to gene and cell therapies. 
  • How to apply the lessons learned from Biomarin’s setback and other approval delays to improving development strategies for future gene and cell therapies. 
  • Comparing and contrasting the FDA’s policies for gene and cell therapies to those of regulatory bodies in other large global markets. 

Featured Speakers

Kazuhiro Fukuta

Senior Consultant, Head of Regenerative Medicine Group, Consulting Div.
CMIC Co., Ltd.

Dr. Kazuhiro Fukuta is a Senior Consultant and the Head of Regenerative Medicine Group, Strategy & Regulatory Affairs Consulting Department, Consulting Division in CMIC. He received his Ph.D. in agricultural chemistry from Kyoto University. He is specialized in science, regulation and clinical development of regenerative medicine, including cell and gene therapies, based on his experiences as a researcher of biochemistry, molecular biology and molecular regenerative medicine at university and his 10 years’ career at a biotech company. He is currently supporting various Japanese and overseas companies in developing new cell and gene therapy products in Japan.

Geoff MacKay

President & CEO
AVROBIO

Geoff is the president and CEO of AVROBIO, a leading clinical-stage lentiviral gene therapy company. A pioneer in cell and gene therapy, he brings a track record of successful leadership at innovative biotechs. He is the former CEO of Organogenesis Inc., the world’s leading cell therapy company. During his tenure at the helm, the company treated 1 million patients with living cell therapies, received the first approval of an allogeneic cell therapy from the FDA’s Center for Biologics Evaluation and Research and led the field of regenerative medicine. Geoff is also the founding CEO of eGenesis, a biotech dedicated to applying CRISPR Cas-9 gene editing to xenotransplantation. Earlier in his career, Geoff spent 11 years at Novartis in senior leadership positions within the global transplantation and immunology franchise. Geoff sits on the boards of Talaris Therapeutics and Satellos Bioscience. Past activities include chairman of the board of MassBio, chairman of the board of the Alliance of Regenerative Medicine, and a member of the advisory council to the Health Policy Commission for Massachusetts.

Paul Hastings

CEO
Nkarta

As CEO, Paul has guided Nkarta Therapeutics through a successful IPO and positioned the company to become a leader in cell therapy. Nkarta’s mission is to discover, develop and deliver novel off-the-shelf natural killer (NK) cell therapy product candidates that have a profound impact on cancer patient. In an important step, Nkarta last year launched clinical trials of its first Car-NK therapy, NKX101, targeting NKG2D ligands. As a former cancer patient, Paul sees his role as CEO as more than just a business position. He is outspoken about today’s societal issues, including promoting racial equity and making medicines affordable for all patients.

Mark McClung

Chief Business Officer
Sangamo Therapeutics

Mark McClung has served as Sangamo’s Executive Vice President and Chief Business Officer since May 2020. Prior to joining Sangamo, from 2015 through 2019, Mr. McClung was Vice President and General Manager of Global Oncology Commercial at Amgen, which he joined from Onyx Pharmaceuticals where he had served as Senior Vice President & Chief Commercial Officer. For two decades prior, Mr. McClung held roles of increasing responsibility at GlaxoSmithKline in marketing and sales, commercial operations, clinical development and product strategy, and general management in Canada, the United States, and Europe, including as Vice President and Head of Global Commercial for GSK Oncology from 2009 – 2013.

Mr. McClung completed graduate coursework at York University in Toronto and Wharton Business School at the University of Pennsylvania, and he received a bachelor’s degree in Human Kinetics and Biomedical Sciences from the University of Guelph in Ontario, Canada.

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