Cell therapy weekly: initiative to expand APAC cell and gene therapy development
This week: TechBio company BioCurie (DE, USA) announced that the Advanced Research Projects Agency for Health (ARPA-H; DC, USA) has awarded it up to US$9.3 million to support the development of its AI-driven gene therapy platform and Lonza (Basel, Switzerland) and Genetix Biotherapeutics (MA, USA) extended their commercial agreement to further advance the manufacturing of a beta-thalassemia gene therapy. Meanwhile, Terumo Blood and Cell Technologies (Terumo BCT; CO, USA) unveiled a new initiative aimed at supporting cell and gene therapy developers in the Asia-Pacific (APAC) region.
The news highlights:
- ARPA-H awards US$9.3 million towards AI-driven platform
- Initiative to expand APAC cell and gene therapy development
- Extended manufacturing agreement for in-demand gene therapy
ARPA-H awards US$9.3 million towards AI-driven platform
The ARPA-H has awarded up to US$9.3 million in funding to accelerate the development of BioCurie’s scalable, data-driven genomic medicine production. Under the award, the TechBio company will design, create and validate an AI-powered digital platform that replaces trial-and-error process development with computational modelling and simulation. The platform integrates advanced modelling, machine learning and real-world manufacturing data, aiming to reduce development timelines and improve process robustness across viral and non-viral gene therapy production.
“AI will fundamentally reshape how advanced therapies are developed and manufactured,” said Irene Rombel, CEO of BioCurie and Principal Investigator on the ARPA-H project. “This ARPA-H award validates our vision and accelerates our ability to deliver a scalable, digital-first manufacturing platform to therapy developers and manufacturers. Our mission is clear: bring disease-modifying and curative genomic medicines to patients faster and more affordably.”
Initiative to expand APAC cell and gene therapy development
Terumo BCT has announced the launch of its cell and gene therapy Ecosystem Blueprint, aimed at supporting cell and gene therapy developers in the Asia-Pacific (APAC) region.
“The APAC [cell and gene therapy] landscape is maturing, with innovators increasingly building for both local impact and global expansion,” commented Wenyan Leong, Director for APAC Commercial and Global Strategic Partnerships. “Terumo BCT is helping these developers future-proof their operations by providing the same world-class automation and standardization used in the world’s leading markets.”
The Blueprint focuses on how critical capabilities, workflows and partnerships must align to simplify complex workflows and support more cost-efficient, scalable cell and gene therapy development and manufacturing, helping developers move from early development to scalable, repeatable delivery. Terumo BCT will be implementing the Blueprint in collaboration with regional partners and cell and gene therapy developers across various real-world applications. Instances will include and are not limited to, co-developing standardized induced pluripotent stem cell workflows and enhancing regional Good Manufacturing Practice training and implementation capabilities.
Furthermore, the blueprint involves partnerships with clinical-stage cell and gene therapy developers to address challenges in current execution models, providing practical validation of clinical readiness, workflow robustness and scalability under real-world operating conditions.
“Our partners understand that navigating the path to commercialization and clinical delivery requires moving beyond manual, fragmented processes toward integrated, high-quality manufacturing solutions,” explained Leong. “Our Ecosystem Blueprint acts as the operational foundation, offering the pre-validated pathways and partner networks that transform regional progress into global commercial reality in a practical, scalable way.”
Extended manufacturing agreement for in-demand gene therapy
Lonza and Genetix have expanded their long-term commercial manufacturing agreement to meet the increasing demand for Genetix’s ZYNTEGLO™, the first and only FDA-approved gene therapy for pediatric and adult patients with transfusion-dependent beta-thalassemia. The partnership also includes plans to scale up manufacturing for future Genetix therapies. Originally established in 2013, this collaboration facilitated the commercial approval and rapid uptake of the gene therapy in 2022.
“Our long-standing partnership with Lonza reflects our shared commitment to commercial excellence and operational discipline to bring curative therapies to patients impacted by rare disease,” said Genetix’s Chief Technology Officer Brian Riley. “Their scientific rigor, quality focus and consistent execution have been important in enabling a reliable supply of ZYNTEGLO™. In the last year, patient demand for Genetix’s therapies has grown consistently and rapidly. Extending the agreement with Lonza and expanding capacity provides a strong foundation as we invest to meet the growing patient demand and broaden our long-term manufacturing strategy.”