While the 2024 Meeting on the Mesa may have moved to the desert of Phoenix, Arizona, the annual gathering of the cell and gene therapy community feels like it is truly starting to find fertile soil. The Alliance for Regenerative Medicine continues to convene a wonderful cross-section of this emerging sector, showcasing thought leaders building unity across the ecosystem, while still showing the competitive entrepreneurialism and innovation that brought this industry into being.

While there were many great topics and conversations had during those 100+-degree days and among the omnipresent, autonomous Waymo vehicles, I’d like to highlight three themes that really seemed most pressing and relevant to the year ahead.

Patient Access: It felt like no topic loomed larger than the ability for patients to access these cutting-edge therapies, particularly those gene editing treatments such as Casgevy, recently commercialized by Vertex Pharma and CRISPR Therapeutics for sickle cell disease. While manufacturing and CMC challenges still exist, improvements in those areas have allowed the industry to shift focus to new challenges on how to get these beneficial, and often curative, therapies to patients. Those challenges include:

• The regulatory requirements are to remain within 60 minutes of the treating center for up to 4 weeks. Since these are typically large biomedical institutions in urban areas, many patients are geographically encumbered from these treatments,
• The intensive patient delivery process is much more than a one-time infusion and may include bone marrow transplants, lymphodepleting chemotherapy, or other more invasive medical procedures
• The high cost (i.e., Casgevy’s $2.2M list price), which, as one industry panelist noted, “is not even making the company much money.”

A highlight of the week was Luke Timmerman’s interview with Jimi Olaghere. Olaghere is a patient advocate and one of the first patients clinically cured of sickle cell disease, having been on the clinical trial for Casgevy after years of desperation. Olaghere’s entire story is well worth listening to, but his final plea was for the industry to keep the diversity of clinical trials in focus and not leave out Africa and those of African descent in the research.

Collaboration: Closely tied to the patient access challenge outlined above was the resounding theme of collaboration. More specifically, collaboration among the various therapeutics brands, as well as, between the public and private sectors. While there are advances being made, including at the Bespoke Gene Therapies Consortium, more work needs to be done if we plan to reach more patients in need. Two points that really hit it home for me were:

• Debbie Drane from CSL Behring (who I am compelled to quote every year now) noted that the industry’s challenge is not products, it’s a shared problem of access. The sector needs to find the will to learn from each other to help optimize manufacturing and other areas where all can benefit and then compete in the market downstream. A prior panelist stated that “if your process is the product, you are not likely to be successful.” in the context of scalability. I see this being akin to the electronic vehicle industry looking for standards of charging devices and technologies to unlock the larger market.
• During a gene editing panel, Fyodor Urnov, PhD who leads the Innovative Genomics Institute at UC Berkeley eloquently wove a storyline with his panelists at two gene editing companies and Rachel Anatol PhD of the Office of Therapeutic Products within CBER at the FDA about future possibilities of the agency working with sponsors to more efficiently review “platforms” where minor components (such as guide RNA sequences) might be modified to support smaller frequency mutations of a common disease where it might not be commercially viable to run entirely new clinical studies when it impacts much smaller fractions of the community.

Rare Diseases: The cell and gene therapy community is uniquely positioned to offer hope to the rare disease community. Danaher’s Sadik Kassim, Ph.D., introduced a panel on the subject by highlighting the prevalence of rare diseases (3.5%–5.9% of the world’s population). There are an estimated 10,000 rare diseases, with 250 novel diseases identified annually, and approximately 80% of them have a genetic component.

While the opportunity exists to significantly reduce the burden of these diseases, new business models, better public/private/payor alignment, and innovative new in vivo therapies will make these hopes a reality for millions of patients around the world.