Spun out of MIT, the company is engineering a new class of biomolecules at 10x lower cost and drastically increased scale to advance gene editing toward cures for complex diseases.

August 12, 2024 05:00 AM Pacific Daylight Time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Kano Therapeutics, a biotechnology company pioneering single-stranded DNA (ssDNA) as a safe, efficient and flexible biomolecule for gene insertions, today announced it has secured an oversubscribed $5 million seed round. The Engine Ventures and VSquared Ventures co-led the round, with Taihill Venture and Metaplanet also participating. The round brings Kano’s total funding to date to $7.1 million. It will be used to develop Kano’s good laboratory practice (GLP) standards, design a process for clinical-grade production of kilobase ssDNA, and advance the company’s internal and external programs through collaborative drug development.

“Existing genetic medicines can change a letter – or maybe a word – of genetic information, but Kano is introducing the ability to replace entire sentences and even paragraphs”

“We believe curing genetic diseases isn’t just a biology problem; it’s an engineering problem,” said Dr. Floris Engelhardt, co-founder and CEO of Kano. “The materials we need to deliver on the full promise of genetic therapies are incomplete. This is why we’re building a ‘one-stop shop’ product development platform for safer DNA payloads at scale. By accessing a new class of gene repair templates, Kano can expand the capabilities of today’s editing techniques, and correct longer stretches of DNA effectively and flexibly.”

Advances in gene and cell therapy, including the 2023 FDA approval of the first CRISPR drug, are paving the way for treating a broad range of genetically-driven diseases. Currently, nearly 3,000 cell and gene therapies are in active development. Despite this momentum, challenges remain when it comes to inserting genetic information into the genome for therapeutic purposes. Current techniques typically rely on double-stranded DNA (dsDNA) donors or viral vectors; however, dsDNA donors are highly toxic and do not insert efficiently, while viral vectors can introduce safety concerns, and are associated with manufacturing and redosing issues.

To cure genetic diseases – the majority of which have no available treatment options – targeted, non-viral gene insertions via ssDNA are key. Pairing the stability of dsDNA with the flexibility of RNA, ssDNA is better suited to efficiently replace whole genes and safer, as it is less likely to trigger the immune system. Kano is enabling the precision-manufacturing of ssDNA in custom lengths and sequences, and at scales suitable for drug development. The company’s novel platform bridges the gap between editing genes and replacing them, empowering scientists, labs and companies with the building blocks they need to devise treatments for genetic disorders, cancers and other high unmet need conditions.

Kano employs bioreactor-based production to manufacture long, circular ssDNA repair templates. The company’s platform connects manufacturing, application and computational tools, serving as a comprehensive, end-to-end solution that:

• Breaks down long-standing manufacturing bottlenecks by delivering kilobase ssDNA at a 10x lower cost and drastically increased scale
• Enables gene constructs with low toxicity and high insertion efficiency for therapies that were previously inaccessible
• Builds a first-of-its-kind, real-world computational knowledge base to understand 3-dimensional structure-to-function mechanisms that improve drug delivery and safety

“Existing genetic medicines can change a letter – or maybe a word – of genetic information, but Kano is introducing the ability to replace entire sentences and even paragraphs,” said Ann DeWitt, General Partner at The Engine Ventures. “The company’s platform is positioned to have an incredible impact on gene and cell therapy, providing the biomedical ecosystem with an entirely new category of technology that will make targeted, non-viral therapeutic gene insertions a reality,” added Lise Rechsteiner, General Partner at VSquared Ventures.

Spun out of the Bathe BioNanoLab at MIT’s Laboratory for Nucleic Acid Nanotechnology, Kano is founder-led and indication-, disease- and technology-agnostic. Its team holds backgrounds in DNA origami, synthetic biology, bioinformatics and biotech corporate strategy. Co-founders Engelhardt and John Vroom, Kano’s Chief Business Officer, met through Nucleate, a collaborative, student-led organization that facilitates the formation of pioneering life sciences companies, and together won the MIT Sloan Healthcare Innovations Prize.

In the coming months, Kano will begin internal pipeline development of ex vivo genetic medicines based on kilobase gene insertion, expand its existing therapeutic collaborations to initiate externally driven pre-clinical programs, and scale its production capacity. To learn more about Kano, or to inquire about partnership opportunities, visit: https://kanotherapeutics.com/

About Kano

Founded at MIT, Kano is bridging the gap between editing genes and totally replacing them, unlocking the broad use of single-stranded DNA payloads for cell and gene therapies. The company is developing and collaborating on therapies that safely, effectively and flexibly correct gene-length stretches of DNA inaccessible to today’s editing techniques. For more information, visit kanotherapeutics.com/ or follow the company on LinkedIn.

Contacts

Media
Kerry Walker
kerry@engineventures.com

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