Cell therapy weekly: association launches to address challenges in advanced cell therapies in Europe
This week: a biotechnology company has been awarded an US$8 million grant to support the clinical development an AAV immune-gene therapy for brain cancers, the US Food and Drug Administration (MD, USA) has approved an allogeneic cell therapy to address graft versus host disease (GVHD) in adult patients with hematologic malignancies. Plus, the seven companies accepted into the FDA’s Manufacturing PreCheck Pilot Program have been revealed, and an association has launched to address key challenges in advanced cell therapies in Europe.
The news highlights:
- US$8 million CIRM grant to support brain cancer AAV gene therapy
- Seven companies selected for the FDA’s Manufacturing PreCheck Pilot Program
- FDA approves precision-engineered allogeneic cell therapy
- Association launches to address challenges in advanced cell therapies in Europe
US$8 million CIRM grant to support brain cancer AAV gene therapy
Following the earlier FDA clearance of Siren Biotechnology’s (CA, USA) Investigational New Drug application for its lead investigational AAV immune-gene therapy, SRN-101, the California Institute for Regenerative Medicine (CIRM; CA, USA) has awarded Siren Biotechnology an US$8 million non-dilutive CLIN2 grant to support the clinical development of SRN-101. SRN-101, now at Phase I/II status, is indicated for high-grade glioma brain cancers.
“High-grade gliomas remain among the hardest cancers to treat, and the patients who live with them cannot wait,” explained Nicole K. Paulk, Founder, CEO and President of Siren Biotechnology. “This funding propels SRN-101 toward the patients who need it, made possible by the vision of the people of California.”
Seven companies selected for the FDA’s Manufacturing PreCheck Pilot Program
The FDA has accepted seven companies, out of 80 requests, into its inaugural Manufacturing PreCheck Pilot Program – a voluntary pilot initiative authorized under Executive Order 14293, designed to allow domestic pharmaceutical and biological manufacturers to engage with the FDA early, before a product application is filed.
Through the program, companies have access to:
- Facility-specific Drug Master File (DMF) reviews, which allows participants to receive early technical guidance from the FDA before manufacturing facilities become operational, enabling enhanced facility readiness prior to drug or biologics application submission.
- Pre-Submission Evaluations of GMP Facilities, providing enhanced engagement with the FDA to support expedited facility evaluation and enable inspections earlier in the review cycle.
- FDA expertise and feedback throughout facility development to help provide early identification of potential facility issues before they result in deficiencies that could delay or prevent application approval
Companies that have been selected to participate in the program with cell and gene therapy priorities are:
- Cellares Corp (Bridgewater, NJ, USA) will manufacture cell-based gene therapy products for oncology and hematology diseases.
- FUJIFILM Biotechnologies (Holly Springs, NC, USA) will support commercial-scale cell culture biomanufacturing.
- Kriya Therapeutics, Inc. (Durham, NC, USA) will manufacture AAV-based gene therapy products to address chronic disease conditions.
Other selected companies for other biologics include Amneal Pharmaceutical (Long Island, NY), Eli Lilly and Company (Lebanon, IN) Kyowa Kirin, Inc (Sanford, NC) and Regeneron Pharmaceuticals, Inc (Saratoga Springs, NY).
FDA approves precision-engineered allogeneic cell therapy
TREGZI™, an allogeneic regulatory T cell immunotherapy developed by Orca Bio (CA, USA), has been approved by the FDA for use in a stem cell transplant to improve survival free of chronic GVHD in the treatment of adults with hematologic malignancies. TREGZI, clinically known as Orc-T®, uses hematopoietic stem and progenitor cells (HSPCs) to reconstitute the immune system, highly purified regulatory T cells to suppress GVHD and conventional T cells to accelerate immune reconstitution and produce graft-versus-leukemia activity.
Based on results from Orca’s Precision-T Phase III study, TREGZI demonstrated favourable results, with patients being able to live with chronic GVHD at one year compared with conventional allogeneic transplant.
“For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukemia effect while minimizing the risk of GVHD and infection,” said Miguel-Angel Perales, medical oncologist and chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center. “The FDA approval of TREGZI signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes.”
Association launches to address challenges in advanced cell therapies in Europe
The T2EVOLVE Association, created by the European T2EVOLVE project, has launched, aiming to provide a long-term framework to help address challenges in advanced cell therapy development. The project aims to support the development, evaluation and implementation of advanced cell therapies by creating a neutral environment where stakeholders can work together on shared challenges and opportunities.
The Association will coordinate activities across five strategic workstreams:
- Patient education and advocacy
- Clinical translation and correlative research
- Frontiers innovation and manufacturing
- Regulatory innovation and policy alignment
- Data reuse and artificial intelligence for advanced therapies
“Our vision is to create a trusted and inclusive platform where scientific excellence, clinical experience, patient perspectives, industrial innovation, and regulatory expertise can converge,” said Carmen Sanges, Executive Director of the T2EVOLVE Association. “By building bridges across sectors, countries, and ongoing initiatives, we aim to accelerate the translation of innovation into better outcomes for patients while ensuring that the momentum created by T2EVOLVE continues to grow into a sustainable European community.”
