Ten years ago the first child received CAR T-cell therapy to treat acute lymphoblastic leukemia, the most common childhood cancer. Today she is 10 years cancer free.

While these cell- and gene-therapies have shown tremendous promise through successful outcomes in the cases known to date, the use of new cell- and gene-based therapies has been slower than expected due to several key factors, including the high cost of treatment and the difficulty to obtain coverage and reimbursement. Famous examples include cancer CAR T-cell therapies, Kymriah (~$373K/treatment), Yescarta (~$475K/treatment), or spinal muscular atrophy treatment Zolgensma (~$2.1M/patient). Interestingly, the FDA is currently evaluating about 900 new gene therapy-based drugs, and it is anticipated that by 2025, the agency will approve an additional 10-20 new cell- and gene-based therapies per year.

“We will see multiple clinical studies reading out over the next few years in the area of gene and cell therapies – these products are expensive and the health care community as a whole needs to think about pricing and reimbursement with the patient in mind.”
Yael Weiss, CEO Mahzi Therapeutics & Track Chair Gene and Cell Therapies at PMWC Silicon Valley January 25-27, 2023 (Program)

While gene therapies are expensive there are also numerous challenges that still need to be overcome, one of which is the gene delivery method. Viral vectors are among the most widely studied vectors for gene delivery – approaches include novel vectors with tissue‐specific tropism, genetically engineering the capsid through directed evolution, and altering the route of administration, to name a few. Furthermore, clinical translation of viral gene therapies is facing manufacturing, biological, and regulatory challenges that require significant efforts from the preclinical, clinical, and commercial sides.

At PMWC 2023 Silicon Valley, January 25-27, we have a 2-day dedicated track for Gene and Cell Therapies touching upon exactly these hot topics and challenges: cost, reimbursement, delivery methods, ethical implications, clinical translation, and commercialization. In addition, we are excited to also include in this important discussion the latest observed trends in the wider context of the gene and cell therapy sector which include parents getting actively involved with fundraising and working with doctors to develop a therapy for their sick child. These are just a handful of exciting themes we will visit with leaders in the field. See the developing tracks below:

January 25th – Day 1: Patient-Centric Therapeutics Track
Patient-Driven Therapies – The Challenges and Successes: Presentations followed by a panel chaired by Yael Weiss with Allyson Berent (Angelman Foundation & Parent), Krystoff Bankiewicz (PTC Bio), and Terry Pirovolakis (Parent).
From Bench to Bedside – Biomarkers, Natural History Studies, Animal Models: Panel chaired by Peter Marks (FDA) with Emil Kakki (Ultragenyx) and Allyson Muotri (UCSD).
Commercialization – Moving from Rare to Common: Panel chaired by John Maraganore (Alnylam) with Rahul Singhvi (Resilience), Andrew Lo (MIT), Shankar Ramaswamy (Kriya), and Mark Harrison (University of British Colombia).
Next Generation Gene Editing / Future Modalities: Presentations followed by a panel chaired by Shannon Muir (CIAPM) with Nadav Achituv (USCF), and Amber Salzman (Epic Bio).
Bioethics & Ethical Implications in Call and Gene Therapies: With presentations by Hank Greely (Stanford University) and Hana El-Samad (UCSF).

January 26th – Day 2: Gene Modified Cell Therapies Track
Advance and Optimize Cell Therapies in Oncology (PANEL): Chaired by Sharon Benzeno, Adaptive Biotechnologies
Pioneering Cell Therapies in the Treatment of Autoimmune Disorders (PANEL): Chaired by Carl June, UPenn
Leveraging Data Sets to Inform Future Drug Discovery and Development (PANEL)
Innovations in Cell Therapy Manufacturing: Chaired by Bruce Levine, UPenn who will also be honored with the PMWC 2023 Luminary Award

I hope the above clearly illustrates the unique opportunity to get an updated view into this exciting and fast-evolving field of gene and cell therapy that we bring to you as a special track at PMWC Silicon Valley January 25-27, 2023 – sign up today and take advantage of the discounted off the regular ticket price.

Tal Behar
President & Co-founder, PMWC
PMWC 2023 Silicon Valley – January 25-27, 2023


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