Manufacturing Scale, Safety Profiles, and Reimbursement Reform Are Critical Hurdles for Type 1 Diabetes Cell Therapy

In the final segment of BioPharm International®’s interview with Steve Harr, MD, president and CEO of Sana Biotechnology (Sana), Dr Harr outlines the 4 questions that will define whether Sana's gene-modified islet cell therapy can achieve meaningful global impact in type 1 diabetes (T1D). He also addresses the manufacturing safety challenges inherent to stem-cell-derived therapies at scale.

Dr Harr frames the company's development path around 4 sequential questions: Does it work? Can it be scaled? Can it be commercialized? And how will it be paid for? On efficacy, he says definitive data on whether the therapy can achieve insulin independence at a therapeutic dose, beyond the low-dose immune evasion already demonstrated, should be available within a year.

Why is manufacturing scale the most formidable challenge facing curative islet cell therapy for type 1 diabetes?

Dr Harr places the scaling challenge in stark epidemiological context. With approximately 10 million people globally living with T1D and roughly 500,000 new diagnoses annually,¹ producing 100,000 doses per year at perfect efficacy and durability would reduce the global T1D growth rate only modestly, from approximately 5% to approximately 4%, he explains. This example illustrates the magnitude of the manufacturing capacity required to generate meaningful population-level impact, he states.

On stem cell manufacturing safety profiles. specifically, Dr Harr identifies 2 priority risks, genomic mutations arising during cell culture and off-target cell differentiation, which may give rise to the unintended presence of non-islet cell types in the final product.

"If you have a stomach that's growing in your muscle for 20 years, it becomes more than a nuisance," Harr emphasizes, illustrating why off-target cell control is a non-negotiable safety requirement.

On reimbursement, Dr Harr stresses that a curative medicine must work financially for patients, payers, providers, and developers simultaneously. He notes that a therapy utilized by only some of these stakeholders will likely never achieve broad clinical adoption regardless of its efficacy.

For more insights from Dr Harr on islet cell therapy as a cure for T1D, watch these other interview clips: segment 1, segment 2, and segment 3.

About the speaker

Steve Harr, MD, President and CEO, Sana Biotechnology

Prior to co-founding Sana, Dr Harr served as chief financial officer and head of Corporate Development for Juno Therapeutics until its acquisition by Celgene in early 2018. He was a member of the board of directors of Loxo Oncology prior to its acquisition by Eli Lilly and Company in early 2019. Dr Harr also was a co-founder and member of the board of directors of JW Therapeutics, a cell therapy company in China. Prior to joining Juno, Dr Harr was at Morgan Stanley, as managing director and head of Biotechnology Investment Banking, and previously as a biotech research analyst and co-head of global healthcare research. He obtained his MD from Johns Hopkins University School of Medicine. He was an internal medicine resident at the University of California, San Francisco, and performed research at Harvard Medical School and Massachusetts General Hospital.

Reference

1. Gregory GA, Robinson TIG, Linklater SE, et al. Global incidence, prevalence, and mortality of type 1 diabetes in 2021 with projection to 2040: a modelling study. Lancet Diabetes Endocrinol. 2022;10(10):741-760. doi:10.1016/S2213-8587(22)00218-2