The Human Connection: CRL’s World Congress Keeps Focus on What Matters

An intimate group of cell and gene therapy experts examine what's working and what's not  

After years of squinting at each other from behind zoom cameras, the mood is clear: scientists are grateful to be meeting in person once again. That was certainly true at Tuesday’s Fifth Annual World Congress Meeting in Cambridge sponsored by Charles River Laboratories. It met virtually last year and not at all in 2020, thanks to the pandemic.

The focus of this year’s World Congress — Realizing the Promises of Cell and Gene Therapies —was a bookend to last fall’s meeting, which was exclusively focused on the cell therapy revolution. Many of the same topics were back on the table, including viral vectors, CAR-Ts, and regulatory challenges, to name a few. While there was general agreement among the attendees that the field is well past the point of waiting for the first win, it is at a critical point. Put another way, what can realistically be done to expand the limited number of cell and gene therapies that have made it to market?

To address this, the Congress took a decidedly more interactive approach than in previous years, focusing heavily on in-person collaboration and conversation. “Gone are the days of 50-slide PowerPoints,” remarked distinguished Charles River scientist Dr. Lauren Black.

As morning moderator Sam Cheung, Director of Scientific Advisory Services at Charles River said, kicking off the first discussion, the audience and their questions are the “third panelist” for each talk. The collaboration was especially important, because the speakers spent significant amounts of their time outlining, defining, and grappling with the many challenges facing the field, both theoretical and biological, logistical and regulatory — while inviting the audience of experts to weigh. Everything from discussing the relative merits of autologous and allogeneic therapies to strategies for navigating the FDA’s post-COVID backlog were fair game.

Animal models and viral vectors

Several speakers bemoaned the limitations of some of the animal models used to advance therapies forward, remarking that the mismatch between the models and the patients too often means that developed treatments are excellent at treating the mice but are not so good at treating the humans. “We used the same process in the mouse as we did in the human,” says Dr. Ken Olivier, VP of Non-Clinical Development at TScan Therapeutics,d uring his morning talk on cellular programming alongside Ananya Zutshi and Entrepreneur-in-Residence at Cellino Biotech. “A lot of the [measuring] endpoints were very checkbox. Most of that stuff did eventually translate… but I don’t think that’s the best way to go.”

During his afternoon talk about the use of adeno-associated viruses alongside Dr. Luk Vandenberghe, who heads the core lab at the Grousbeck Gene Therapy Center and Dr. Glenn Pierce, Interim Chief Scientific Officer at Voyager Therapeutics cut to the core of the frustrations with the field: “We need to do more… We’ve made some strides over the last 20-25 years, but really insufficient.” Dr. Vandenberghe agrees, noting that “the field has gone through a hype cycle… there is a skepticism. We have to show the world we can build medicines.” Adeno-associated viruses still face many challenges and the two speakers spent significant time talking about toxic and overbroad immune responses to the viral vector, both necessary steps to overcome for the field to expand and produce more wins.

New technologies can help accelerate the field

But while progress has been slow in some areas, there is much to feel optimistic about: “We have so many things now that we didn’t have even 10 years ago,” says Dr. Prem Premsrirut, co-founder and CEO of Mirimus, Inc, in New York which uses both CRISPR and RNAi to engineer animal models. “Learning how to combine new technologies, that’s what’s going to make the most powerful therapeutics in the future.” New knowledge, new ways of collaborating, and new ways to reduce risk are propelling the field forward. With new innovations at every stage of the process, most attendees ‘look to the hope’, and are excited and galvanized about the future possibilities. Dan Leger, Associate Director of Global Marketing for Cell & Gene Therapy at Charles River, encapsulated this, and closed out the conference with a summary: “It’s going to take all of us. It’s through community and communication… that will lead to these breakthroughs. But above all else, what brings us here today is [the desire to] make a difference in someone’s life.”

As the World Congress attendees reflected and unwound over drinks and hors d’oeuvres after a long and thought-provoking day of talks, the most important takeaway of the conference — more than any one fact or talk — was the reminder that the work they do matters to patients. They are literally waiting for solutions. Dr. Rabi Mishra, part of Takeda Oncology, was feeling good about the day.: “It’s easy to get lost in the process… [this event] tells me that what I’m doing is really going to help someone.”