Cell therapy weekly: encouraging data from base-edited cell therapy for sickle cell
This week: Beam Therapeutics (MA, USA) presented encouraging data from its BEACON trial assessing its base-edited cell therapy for severe sickle cell disease and SolasCure (Cambridge, UK) received Fast Track Designation for its Aurase Wound Gel (AWG). Plus, Celltrio (CA, USA) secured US$15 million in investment to propel its flagship automation platform.
The news highlights:
- Fast Track Designation for chronic wounds
- Encouraging data from Phase I/II trial for sickle cell disease
- US$15 million invest to facilitate scalable, sterile automation platform
Fast Track Designation for chronic wounds
The US Food and Drug Administration (MD, USA) has granted Fast Track Designation to AWG for the indication of calciphylaxis ulcers. AWG is a specialized hydrogel developed by SolasCure to treat chronic wounds and currently being assessed in a Phase II trial for venous leg ulcers.
AWG contains Tarumase, a recombinant enzyme that targets fibrin, elastin and collagen in wounds to promote healing though debridement (removal of dead, damaged or infected tissue from a wound) and wound bed preparation. This investigational hydrogel may provide an alternative solution for patients with calciphylaxis ulcers, which stems from the calcification of blood vessels in fat and skin tissues due to calciphylaxis – a rare condition with limited treatment options.
Encouraging data from Phase I/II trial for sickle cell disease
Beam Therapeutics announced encouraging data from it Phase I/II trial of BEAM-101, an investigational base-edited autologous hematopoietic stem cell therapy for severe sickle cell with severe vaso-occlusive crises. At the European Hematology Association 2025 Congress (12–15 June 2025; Milan, Italy), Beam Therapeutics presented new data, detailing the promising findings from the BEACON trial. In the trial, 17 patients were treated with BEAM-101, where all patients achieved significant improvements, including:
- Significant increases in Hemoglobin F Induction of more than 60%
- Reduction in sickle hemoglobin
- Rapid neutrophil and platelet engraftment
- Normalized or improved markers of hemolysis and oxygen delivery
- Resolution of anemia durable for up to 15 months
- Improve red blood cell health and function
“We remain highly encouraged by the potential of BEAM-101, with today’s data further building on its potential to deliver a transformative treatment for patients with [sickle cell disease],” expressed John Evans, CEO of Beam. “We continue to see growing evidence of differentiated outcomes for BEAM-101 and base editing in severe [sickle cell disease], now observed across 17 patients with the longest follow-up of over one year.
US$15 million invest to facilitate scalable, sterile automation platform
Celltrio has secured US$15 million in investment, which will help accelerate the global commercialization of RoboCell®, a modular platform for automated, sterile cell culturing and cell therapy manufacturing.
“We are highly impressed by Celltrio’s customer traction and its differentiated intellectual property in cell culturing and cell & gene therapy automation,” said Jay Song, CEO of Premier Partners (Seoul, South Korea), an investment company that led the investment round. “We believe Celltrio is poised to become a category-defining leader, delivering scalable, sterile automation to laboratories and manufacturing facilities worldwide.”
Celltrio aims to expand commercialization of RoboCell across North America, Europe and Asia.