Gene-modified cell therapies – often also referred to as ex vivo gene therapies – consist of specific cells that are genetically modified outside the body being administered as a therapeutic in order to help the patient fight a particular disease. In the context of gene therapy new genetic material can be introduced to repair an existing defect or to supplement a missing or deleted piece of genomic information. The most recent example falling into this category is the FDA approved gene therapy Hemagenix (which consists of a viral vector carrying a gene for clotting Factor IX), a one-time gene therapy product given as a single dose by IV infusion for a type of hemophilia. It gives people with the inherited disorder a treatment option that could potentially keep their bleeding in check for years while also allowing them to skip the infusions that have been the standard of care.

In the related but distinct treatment paradigm of cellular therapies the actual therapeutic is of functional cellular nature. Most prominent among these are the chimeric antigen receptor T-cell (CAR T-cell) therapies for cancer with six clinical approvals to date. More recently, a similar approach based on natural killer cells directed against molecular targets on cancer cells (CAR-NK) have entered clinical development.

While the various technologies are progressing at a fast pace, there are still many challenges that need to be overcome to make gene-modified cell therapies a more routine treatment opportunity, including improvements in safety profiles, the design and execution of clinical trials, educating and mentoring the next generation of early-career professionals, working with regulatory agencies, patient education and awareness, and last but not least, the question of pricing and reimbursement.

“Take as an example: It’s hard to effectively convey the difference between somatic and germline gene editing to people outside the field. It’s hard to explain how and why we’re only using a disabled version of HIV as a Trojan horse to deliver the genetic material – not real HIV!” –Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, UPenn & PMWC 2023 Luminary Awardee (January 25-27, 2023 PMWC Program)

The importance of the educational aspect is nicely depicted in the recently released documentary “Of Medicines and Miracles” which tells the story of Carl June’s effort to reprogram T-cells to fight cancer and Emily Whitehead’s fight to beat leukemia, now cancer-free for ten years. The hope is that many people in the public will see the film and will come to understand a little bit more not only about the technology, but also what patients go through and the factors scientists and clinicians have to consider regarding the development of these therapies.

“I hope the film will raise awareness, increase participation in clinical trials, and support additional funding and greater acceptance of CAR T and other advanced therapies.” –Bruce Levine

We are excited to have world class experts from the field of gene-modified cell therapies, including Carl June (University of Pennsylvania, prior PMWC Luminary Honoree for developing CAR-T therapy, and just recently Awarded the Inaugural Maria I. New International Prize for Biomedical Research), join the Gene Modified Cell Therapies Track (Track 1 – January 26th), chaired by Sharon Benzeno (Adaptive Biotechnologies), at PMWC 2023 Silicon Valley January 25-27. This track will not only touch on the latest advancements of cell therapies, but it will also focus on various therapeutic applications in oncology and autoimmune disease, how data sets can inform future drug discovery, and why innovations in cell manufacturing are needed to help mitigate the high production costs. Lastly, we will honor Bruce Levine (UPenn) with the PMWC 2023 Luminary Award for his pioneering work in cancer immunotherapy that led to the first FDA-approved gene therapy for leukemia and lymphoma.

See below for highlights of the evolving Track 1, January 26th program chaired by Sharon Benzeno (Adaptive Biotechnologies):

• PMWC 2023 Luminary Award Ceremony which will honor Bruce Levine (University of Pennsylvania)
• Advance and Optimize Cell Therapies in Oncology – panel chaired by Sharon Benzeno (Adaptive Biotechnologies) with Ira Mellman (Genentech), Lewis “Rusty” Williams (Walking Fish Therapeutics), Loïc VINCENT (Affini-T Therapeutics), Sneha Ramakrishna (Stanford) and Nick Haining (Arsenal)
• Pioneering Cell Therapies in the Treatment of Autoimmune Disorders – a panel chaired by Carl June (UPenn), Lenny Dragone (Sonoma Bio), James Chung (Kyverna), David Chang (Cabaletta), and Cristina Musselli (Abata Therapeutics)
• Leveraging Data Sets to Inform Future Drug Discovery and Development – a panel chaired by Jason Coloma (Maze Therapeutics), with Peter DiLaura (Sonoma Bio), Nick Haining (Arsenal), Lacey Padron (Parker Institute) and Ulrik Nielsen (Sanofi)
• Patentability of Immunotherapies – Latest Developments – a talk by Janet Xiao (Morrison & Foerster LLP)
• Innovations in Cell Therapy Manufacturing – panel chaired by Bruce Levine (UPenn), Chris Holt (BMS), Rahul Singhvi (Resilience), Heidi Hagen (Sonoma Bio) and Snehal Patel (Sana Biotechnology)
• Investment in Cell Therapies- a panel chaired by Karen Tkach Tuzman (BioCentury). Panelists include Rajul Jain (Vida Ventures), Yvonne Yamanaka (venBio), Brian Daniels (5am Ventures), Abraham Bassan (Samsara Capital) and Robert Lin, UPMC.

Take advantage of PMWC January 25-27’s expiring registration rate now to not miss out on this unique opportunity to engage with the high-caliber contributing speakers assembled together to discuss and advance the various aspects of Gene-Modified Cell Therapies, and Precision Medicine as a whole.