Takeda and Carmine Enter $900 Million Non-Viral Gene Therapy Collaboration

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Cambridge, Massachusetts-based Carmine Therapeutics inked a research collaboration deal with Japan’s Takeda Pharmaceutical to develop and commercialize non-viral gene therapies for two rare diseases.

Under the terms of the deal, Takeda will pay Carmine an undisclosed upfront payment and research funding support. Takeda has committed to a $5 million convertible loan to support the development of Carmine’s REGENT technology platform. Carmine is also eligible for more than $900 million in milestone payments and tiered royalties. Takeda has an option to license the programs after preclinical proof-of-concept studies are concluded and would then take over clinical development and commercialization.

Typically, gene therapies rely on viruses as vector for delivering gene therapies to target cells, usually adeno-associated viruses (AAV). Carmine’s REGENT technology platform uses red blood cell extracellular vesicles (RBCEVs). Broadly speaking, the technology uses engineered O-type blood cells to carry the RNA payload to the target cells.

One advantage over AAV-based gene therapies is the potential for repeat dosing, a larger payload capacity, and improved bio-distribution in selected tissues by modifying the surface molecules of the RBCEV.

“We are pleased to enter this collaboration with Takeda, a recognized global leader in rare disease therapies, slightly more than a year since Carmine was created and incubated by Esco Ventures X,” said XQ Lin, founding chief executive officer of Carmine. “This provides Carmine with significant funding to further develop our REGENT platform and advance our wholly-owned programs.”

Carmine was founded in 2019 by Esco Ventures X, Harvey Lodish with the Whitehead Institute for Biomedical Research and MIT, and Singaporean researchers Minh Le and Jiahai Shi. So far, the company has raised more than $9.4 million in seed equity financing led by Esco Ventures and Takeda Ventures.

In December 2019, Carmine won the Bristol Myers Squibb 2019 Golden Ticket for LabCentral. LabCentral is a shared laboratory launchpad for life sciences and biotech startups in Cambridge. Bristol Myers Squibb, as part of the Golden Tickets program, could choose up to two companies per year to underwrite the cost of one lab bench for one year in LabCentral’s facility in Kendall Square.

At the time, Ronne Yeo, vice president of Discovery at Carmine, said, “Nucleic acid therapeutics will take center stage in the 21st century, however their delivery has been a huge technical challenge. The beauty of Carmine’s REGENT platform is that we are able to harvest large quantities of extracellular vesicles very economically, and load them with nucleic acid payloads to be delivered in various sites in the body, overcoming several limitations of today’s gene therapy vehicles.”

Takeda has been building its pipeline in gene therapy and protein replacement technology. In March, Takeda entered a multi-target partnership with Evox Therapeutics to develop protein replacement and mRNA therapies for rare diseases. The deal involved five new therapies, including Evox’s preclinical program for Niemann-Pick disease type C (NPC). That deal included $44 million in upfront, near-term milestone payments and research funding in addition to $882 million in various milestone payments.

“Collaborating on the Evox exosome platform also complements our expanding capabilities in cell and gene therapies,” said Madhu Natarajan, Takeda’s Rare Diseases Drug Discovery unit head, “particularly with the potential to develop new delivery approaches in addition to our cutting-edge adeno-associated virus platform, to provide transformative therapies or functional cures for people living with rare diseases.”

And in September 2019, Takeda and Evotec partnered on at least five drug discovery programs across a broad range of indications. Then in April of this year, the two companies initiated a long-term research deal in support of Takeda’s gene therapy discover programs. Under the alliance, Evotec supports various Takeda programs aligned with Takeda’s four therapeutic areas: oncology, rare diseases, neuroscience and gastroenterology.

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