Cell therapy weekly: progress in iPSC-based therapies
This week: Applied StemCell (CA, USA) submitted a Type II Drug Master File (DMF) to the US Food and Drug Administration (FDA; MD, USA) for its GMP-grade human induced pluripotent stem cell (hiPSC) line, Fate Therapeutics (CA, USA) received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its iPSC-derived CAR T-therapy for systemic lupus erythematosus, and Cellino (MA, USA) and Karis Bio (Seoul, South Korea) announced a strategic partnership to manufacture an iPSC-derived cell therapy for cardiovascular disease.
The news highlights:
- DMF submitted for GMP-grade hiPSC line
- CAR T-therapy for systemic lupus erythematosus gets RMAT designation
- Strategic partnership to manufacture iPSCs for cardiovascular disease
DMF submitted for GMP-grade hiPSC line
Applied StemCell, a company specializing in iPSC technology and gene editing, announced it has submitted a Type II DMF to the FDA for its ActiCells™ GMP-grade hiPSC line. This regulatory milestone aims to streamline the path for researchers developing cell therapies, providing them with a high-quality, FDA-compliant iPSC platform.
Alongside the DMF, Applied StemCell has launched a new version of its hiPSC line that has been pre-engineered with its patented TARGATT™ landing pad at the H11 safe harbor locus. This system allows for the precise insertion of large DNA fragments (up to 20 kb) at a safe genomic location, enabling stable and consistent gene expression, making it easier for developers to create customized, gene-edited cell lines for specific therapeutic applications.
“Securing a DMF for our ActiCells GMP hiPSC line, along with introducing our ActiCells GMP TARGATT hiPSC Knock-in Kit, represents a major advancement for the cell therapy industry,” said Ruby Tsai, CEO of Applied StemCell. “By providing a fully characterized, regulatory-ready iPSC platform, we empower researchers to accelerate development while reducing manufacturing and regulatory hurdles.”
CAR T-therapy for systemic lupus erythematosus gets RMAT designation
Fate Therapeutics has received RMAT designation from the FDA for its off-the-shelf CAR T-cell therapy, FT819, which is being developed to treat moderate to severe systemic lupus erythematosus, including lupus nephritis. FT819 is derived from iPSCs and is currently in a Phase I trial evaluating its safety and efficacy using a chemotherapy-free conditioning regimen.
Early trial data supported the RMAT application, and Fate is now testing FT819 at higher doses in up to 10 patients. The therapy’s development is also backed by a US$7.9 million grant from the California Institute for Regenerative Medicine. Fate plans to share more clinical results in 2025.
“RMAT designation recognizes the unique therapeutic potential of our off-the-shelf CAR T-cell therapy to address the unmet need of a wide range of lupus patients,” said Bob Valamehr, President and CEO of Fate Therapeutics. “We believe our current development strategy for FT819, which is designed to provide CAR T-cell therapy on-demand in a cost-effective manner and alleviate patient burden associated with intense conditioning chemotherapy and extended hospitalization, may enable treatment in the community setting and access to patients in underserved areas. With this designation, we look forward to working closely with the FDA as we seek to accelerate development of FT819 to bring this unique treatment to patients across the continuum of care.”
Strategic partnership to manufacture iPSCs for cardiovascular disease
Cellino and Karis Bio have announced a strategic partnership, which aims to industrialize the world’s first clinical-stage autologous induced pluripotent stem cell (iPSC)-derived cell therapy for peripheral artery disease (PAD) and coronary artery disease (CAD).
Karis Bio is currently running a first-in-human trial in South Korea using patient-specific iPSCs to regenerate blood vessels, offering a non-invasive alternative to stents or bypass surgery without the risk of immune rejection. Cellino’s Nebula™ platform — an AI-powered, automated biomanufacturing system — will be used to support production of these personalized cells. This collaboration will initially focus on manufacturing in South Korea and later expand into Phase II trials in the US through Karis Bio’s US branch.
“Our inaugural first-in-human study using an autologous iPSC-derived therapy in South Korea is a pivotal advancement in regenerative medicine,” said Young-sup Yoon, CEO of Karis Bio. “This collaboration with Cellino will mutually leverage the strengths of both organizations, using Cellino’s Nebula platform and Karis Bio’s vessel regeneration technology to pave the way for the next generation of patient-specific cardiovascular disease therapies to be delivered worldwide.”