Making HSC gene therapy bloom through automated, scalable solutions

Orchard Therapeutics is grounded in the past, but growing toward the future. Founded in 2015, Orchard is playing a critical role in developing hematopoietic stem cell (HSC) gene therapy, advancing this life saving technology from a visionary idea to a promising reality. Carrying forward this mission, Orchard was successfully acquired by Kyowa Kirin, a Japan-based global speciality pharmaceutical company, earlier this year. This expanded, integrated business is increasing its focus on meeting the needs of people living with some of the world’s most rare genetic diseases. 

To build on the decades of research and advancements in the field, Orchard is prioritizing the automation of manufacturing processes. “Historically, we’ve always been on old school paper based systems. Having to analyze and draw together all of the data from different sources is really challenging, and it's not a nice process”, reflects Emma Chan, Director of Technical Development. With a renewed focus on process development, the implementation of bioreactors and automation solutions has enabled effective models for replicating the manufacturing process, ultimately leading to fewer manual errors. 

Central to this solution is the adoption of Benchling, the benefits of which are pervasive throughout every lab based team at Orchard, from the translational research team to the process development team. “We have an open system so that everyone can access all of the data across teams,” shares Emma. “It really helps when we transition from early research to process development, giving everyone insight into the continuity of data.” 

Their team supported the move to Benchling with a Benchling Success Package, which provides tailored support and actionable tasks to help Orchard achieve their data goals. Benchling partners with Orchard to ensure they stay on track amidst other priorities, resulting in optimized solutions at every stage of the transition. The Success Package also includes comprehensive user training on Benchling, ensuring team members are up-to-date on the latest Benchling features and best practices for data collection and analysis. This training streamlines processes, enhances efficiency, and frees up scientists’ time to accelerate Orchard's progress. 

Benchling’s templates offer further efficiencies, helping teams to achieve standardization for experiments, between individuals, teams, and departments. “By using the non-modifiable template, we can rely on Benchling for Good Laboratory Practice (GLP) compliance, as well as keep track of who made the changes,” explains Ana Rubio, Scientist II, Technical Development. With an emphasis on setting up templates and sub-templates, Orchard has been able to standardize workflows, improve data sharing, and increase data integrity and transparency. 

Harnessing such efficiencies, Orchard has had noteworthy therapy progress, with the FDA approval of the first gene therapy treatment for juvenile metachromatic leukodystrophy (MLD) in March, 2024. MLD, caused by a faulty gene that inhibits affected children from producing an important metabolic enzyme called ARSA, has devastating deteriorating effects on the brain and nervous system. The introduction of the gene therapy Lenmeldy, derived from a patient’s own hematopoietic stem cells (HSCs) and delivered in a single-dose infusion, marks important advancements in effective treatments. 

Looking toward the future, the evolving world of cell and gene therapy calls for scale. “With the field moving toward larger indications, and with more and more patients needing treatment, the ability to scale up and automate is critical”, shares Ana. By achieving added degrees of digitalization, Orchard is not only able to capture and centralize data from multiple sources, whether it’s internal data or outside research institutions, but they are also able to handle and analyze that data. Harnessing science and technology, Orchard is continuing to make a profound difference in the lives of those affected by certain rare, inherited diseases by transforming processes that make data powerful.