Cell therapy weekly: partnership for scalable automation
This week: Cellular Origins (Cambridge, UK) and Fresenius Kabi (Bad Homburg, Germany) have joined forces to enhance automation for cell and gene therapy manufacturing, AvenCell Therapeutics (MA, USA) has secured US$112 million Series B funding to support ongoing clinical validation for its CAR-T platform. Plus, the US Food and Drug Administration (FDA; MD, USA) granted Rare Pediatric Disease designation to an experimental therapy for Danon disease.
The news highlights:
- Partnership for scalable automation
- Accelerating clinical validation
- Rare Pediatric Disease designation for Danon disease therapy
Partnership for scalable automation
Cellular Origins and Fresenius Kabi have announced a collaboration to enhance the automation process in cell and gene therapy manufacturing. By integrating Fresenius Kabi’s Cue® Cell Processing System with Cellular Origins’ Constellation™ robotic platform, the collaboration aims to deliver fully automated, around-the-clock manufacturing at scale. This integration optimizes manufacturing space, reduces the need for personnel and requires no changes to existing processes.
Christian Hauer, President MedTech at Fresenius Kabi, stated:
“Our cell therapy technologies are designed with automation at their core, to provide new levels of precision and efficiency. By working closely with the team at Cellular Origins, we hope to advance the [cell and gene therapy] industry by helping therapy developers embrace automation during the production process with the goal of ultimately benefitting patients.”
Accelerating clinical validation
Clinical-stage cell therapy company, AvenCell Therapeutics, has secured US$112 million in Series B funding to support ongoing clinical validation of company’s universal CAR-T cell therapy platform.
The funding was led by Novo Holding (Hellerup, Denmark) and includes investments from Blackstone Life Sciences (MA, USA), Eight Roads Ventures Japan (Tokyo), F-Prime Capital (MA, USA), Piper Heartland Healthcare (MN, USA) and NYBC Ventures (NY, USA).
This innovative platform produces switchable CAR-T cells, both before and after patient administration, offering a significant improvement in safety and efficiency over conventional therapies for treating various blood cancers. Current trials are focused on evaluating therapeutic products, including AVC-101 and AVC-201, which utilize the platform for relapsed/refractory acute myeloid leukemia.
“AvenCell’s switchable CAR-T platform represents a paradigm shift in cell therapy, offering unprecedented control over treatment dynamics,” explained Nihal Sinha, Partner at F-Prime Capital and recent member of AvenCell’s Board of Directors. “The ability to modulate CAR-T cell activity post-infusion could address critical safety and efficacy challenges in current therapies. We look forward to supporting AvenCell’s journey in advancing these promising cell therapies through clinical development, with the potential to address significant unmet needs in the treatment landscape.”
Rare Pediatric Disease designation for Danon disease therapy
The US FDA has granted Rare Pediatric Disease designation to PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell therapy for Danon disease, a rare X-linked genetic disorder. Developed by Papillon Therapeutics (CA, USA), PPL-002 is designed to restore functional Lamp-2 protein, which is severely deficient in individuals with Danon disease. Mutations in the LAMP2 gene result in weakened heart and skeletal muscles, as well as intellectual disabilities, with symptoms typically emerging in adolescence or early adulthood.
“We are pleased that the [US] FDA has granted a second Rare Pediatric Disease designation to our pipeline,” commented Carter Cliff, CEO of Papillon Therapeutics. “This designation offers an additional opportunity to receive a priority review voucher, which will enable us to accelerate our pipeline and better serve patients and families affected by Danon disease.”
Preclinical studies have demonstrated that PPL-002 improves disease phenotype across affected tissues by targeting multiple organ systems simultaneously, offering the potential to modify and reverse disease progression. The research is partially funded by the California Institute for Regenerative Medicine (CA, USA).
