Recent advancements of gene and cell therapy offer a glimpse of what’s to come. Be it previously approved CAR-T therapies (Yescarta, Kymriah, Tescartus), AAV (adeno-associated virus) transgene delivery (Luxtuma, Zolgensma), or investigational gene editing (in vivo EDIT-101, ex vivo CTX001, or allogeneic T cells) and universal donor cell therapies (allogeneic cell products, such as engineered iPSCs), the potential for personalized gene and cell therapies for common disease is huge. Applying novel, next-generation technologies such as base and prime editing, and epigenome editing are further expanding the impact of these therapeutic approaches on treating human disease. Challenges still exist of course, with the major one being the interplay with the patient’s immune system to achieve durable “acceptance” by the organism. Recent advances and efforts include methods of temporarily clearing antibodies from circulation, immunosuppression regiments, or the engineering of modified AAV capsids that evade neutralizing antibodies.
Some of the key players in today’s gene and cell therapy market include Kite Pharma, Novartis, Spark Therapeutics, Allogene Therapeutics, Poseida Tx, 4D Molecular Tx but also companies in the base and gene editing sector, including Mammoth Biosciences, Editas Medicine, CRISPR Therapeutics, Graphite Bio, Beam Tx, and Intellia Tx.
Given the promising developments in this field we have created a packed “Gene and Cell Therapies” track for June 28-30th’s PMWC 2022 Silicon Valley that focuses on and dives into the latest therapy advancements, challenges to overcome as a research and pharma community, and showcases companies that are destined to change the outcome of rare disease treatment via innovative products. Gene and Cell Therapy sessions include:
• An overview of the advances in Gene and Cell Therapies with Peter Nell (Mammoth Biosciences)
• Advances in Gene and Cell Therapies – chaired by Jane Grogan (Graphite Bio) with Amy Simon (Beam Tx), Eric Ostertag (Poseida Tx), and Laura Sepp-Lorenzino (Intellia Tx)
• Translating CGT to the Clinic – a panel discussion chaired by Nicole Paulk (UCSF) with Matthew Porteus (Stanford University), Eric Crombez (Ultragenyx), Peter Francis (4D Molecular Tx), Erandi De Silva (Forge Biologics), and Patrice Hugo (Q2 Solutions)
• Bioethics & Ethical Implications in Cell and Gene Therapies – with Fyodor Urnov (UC Berkeley), and Sandy Macrae (Sangamo Tx)
• Past, Present, and Future of CRISPR: Where will CRISPR Take Us and How is the Technology Evolving to Realize its Full Potential – a panel discussion chaired by Janice Chen (Mammoth Biosciences) with Fyodor Urnov (UC Berkeley), Kiana Aran, (Cardea Bio), Laura Sepp-Lorenzino (Intellia Tx), and Joseph Lehrer (Graphite Bio)
• A series of Company Showcases of exciting, new and established companies in the field, including Mission Bio (Yan Zhang), Quest Diagnostics (Yuri Fesko), and Mekonos (Anil Narasimha).
Don’t miss this most timely and informative session at PMWC 2022 Silicon Valley, taking place at the Santa Clara Convention Center, June 28 – 30.
Co-founder & President, PMWC
Precision Medicine World Conference – June 28-30, 2022 Silicon Valley