Cell therapy weekly: Leveraging CRISPR-Cas3 to treat epidermolysis bullosa

Written by Megan Giboney

This week: Sanguine Biosciences (MA, USA) has secured US$10 million in credit financing from Catalio Capital Management (NY, USA), Kite Pharma (CA, USA), a Gilead company, and Epic Bio (CA, USA) have announced a research collaboration and license agreement and Healiva (Lugano, Switzerland) and C4U Corporation (Osaka, Japan) have formed a strategic alliance to develop CRISPR-Cas3-based gene therapy for epidermolysis bullosa.

The news highlights:


US$10 million financing to accelerate apheresis-derived product development

Sanguine Biosciences has secured US$10 million in credit financing from Catalio Capital Management through Catalio’s Structured Opportunities Fund.

Sanguine will receive an initial $6 million, with an option for an additional $4 million. The company plans to use this capital to support its newly established apheresis donation center, advance the development of crucial cell and gene therapy products originating from apheresis, including GMP-grade and CD34+ mobilized leukopaks, and fuel its commercial growth efforts.

“Our partnership with Catalio is an important step forward for Sanguine as we bridge the gap between researchers and patients, leading to advanced therapies and likely better outcomes,” said Brian Neman, CEO of Sanguine. “This financing will enable our business to continue to innovate, grow, and align with our customers’ needs.”

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Kite and Epic Bio collaborate to develop new cell therapies for cancer

Kite, a Gilead company, and Epic Bio have announced a research collaboration and license agreement utilizing Epic Bio’s proprietary gene regulation platform to advance the development of next-generation CAR T-cell therapies to treat cancer.

The agreement includes an upfront payment from Kite to Epic Bio, with additional performance-based payments tied to development, regulatory milestones, and sales. Epic Bio also stands to receive tiered royalties for any approved products stemming from this collaboration.

“Cell-based cancer immunotherapies have reshaped modern cancer care, but we have still only scratched the surface of their potential benefit for patients. At Kite, we are committed to developing next-generation CAR T-cell therapies with the goal of reaching more patients with cancer who could benefit,” said David Barrett, Vice President of Cell Biology and Translational Medicine at Kite. “We look forward to collaborating with Epic Bio with the goal of further advancing today’s cell therapies and potentially expanding their reach and impact on patients.”

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Leveraging CRISPR-Cas3 to treat epidermolysis bullosa

Healiva and C4U Corporation have formed a strategic alliance to develop CRISPR-Cas3-based gene therapy for epidermolysis bullosa (EB). EB is a rare genetic skin disorder that causes severe skin blistering and erosion upon minimal friction or trauma.

Akimitsu Hirai, President & CEO of C4U, said, “By utilizing its proprietary CRISPR-Cas3 technology, a new generation of gene editing technology, C4U’s focus is to develop alternative and complete cures to genetic diseases. Skin diseases, especially EB, are relevant targets for the CRISPR-Cas3 platform, and we believe that we will bring a bright future to EB patients throughout the world.”

Priyanka Dutta Passecker, Co-Founder and CEO of Healiva, stated: “This collaboration integrates gene therapy prospects with our comprehensive cell therapy portfolio, leveraging our expertise in autologous (Epidex®) and allogenic therapy technologies and the capabilities of our manufacturing infrastructure.”

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