CGT In 2023 With Cartesian Therapeutics, Poseida Therapeutics, REGENXBIO, And Spark Therapeutics

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

As 2022 winds down, all eyes in CGT are focused on what’s in store for the cell and gene therapy space in the coming years. In a recent article, I shared what will inevitably be some of 2023’s most pressing issues in the cell and gene therapy space. From PDUFA VII’s impact on CGTs and the capacity crunch to moving beyond oncology in cell therapy and more, there are seemingly countless topics clamoring for our attention in the short term. Knowing this, I sought the expert commentary from a few of the industry’s best and brightest to get their takes on what they anticipate 2023 will bring. Here’s what Cartesian Therapeutics’ Murat Kalayoglu, Poseida Therapeutics’ Mark Gergen, REGENXBIO’s Nina Hunter, and Spark Therapeutics’ Ron Philip had to say.

“2023 will be known as the year that cell therapy has finally entered a new landscape beyond oncology.  Already we are seeing unprecedented responses to cell therapy in patients with autoimmune diseases such as myasthenia gravis and systemic lupus erythematosus, and I anticipate that 2023 will bring more mature data for these indications.  Unlike oncology, indications beyond oncology will require safer cell therapies.  As such, I expect that the field will shift towards cell engineering approaches that do not involve irreversible modifications at the genomic (i.e. DNA) level, do not rely on exponential cell proliferation, and do not require preconditioning lymphodepletion.”
— Murat Kalayoglu, CEO, Cartesian Therapeutics

“As we look toward 2023, we expect to see several themes emerge in the cell and gene therapy industry. Regarding cell therapy, we think leading allogeneic (allo) approaches will begin to declare themselves more fully as learnings from autologous and early allo programs give rise to a new class of treatment options which build on those learnings from their pioneers. As for gene therapy, we will see the emergence of new approaches and technologies which can overcome the limitations of AAV approaches and start to unlock a new era in patient benefit for rare diseases.

Recently, the cell and gene therapy industry have been on a roller coaster, going from a period of unprecedented and unparalleled excitement with dramatic levels of company creation to a historical period of valuation lows, many negative events and company restructurings we have never seen before. We look for the industry to soon stabilize and hopefully establish a solid foundation for a new period of growth and innovation in the coming year.

Looking beyond 2023, the cell and gene therapy industry will need to navigate the financial and financing markets as well as develop strategies to manage the current headwinds in pharmaceutical reimbursement and regulatory environments along with our colleagues in the broader biopharma industry – with the ultimate goal of broadening patient access to novel treatments for cancer and rare disease.”
— Mark Gergen, CEO and Director, Poseida Therapeutics

“It is an exciting time to be part of cell and gene therapy development. These innovative technologies offer hope for patients and their families. It is amazing to see so many stakeholders - regulators, cell and gene therapy developers, patient advocacy groups, academics - working together to move this field forward to give patients much needed therapeutic options. Although there are and will be regulatory challenges to address as science and technology advance at such a rapid pace, the recent approvals in this area show that these novel approaches can make a real difference for patients. I think the coming year will have many more examples of success.”
— Nina Hunter, VP Corporate Strategy, REGENXBIO

“I continue to be inspired by the breakthroughs and discoveries in genetics that are unlocking new disease targets, which are resulting in more gene therapy development opportunities. While these new pathways are being uncovered, we are also seeing our collective experience in gene therapy R&D and manufacturing continue to advance as more assets are entering later stages of clinical development.  As an industry, one of our biggest challenges will remain the significant and consistent long-term investment needed to advance the technology while simultaneously bringing novel therapies to patients. I anticipate we’ll see more partnerships to offset the risk and costs and enable continued growth of the sector. I believe this diversity of approach and thought can help us address the evolving challenges so that we collectively can achieve the full potential of gene therapy.”
— Ron Philip, CEO, Spark Therapeutics