Kano Therapeutics, a biotechnology company creating single-stranded DNA (ssDNA) as a safe, efficient, and flexible biomolecule for gene insertions, announced it has secured an oversubscribed $5 million seed round. The Engine Ventures and VSquared Ventures co-led the round, and Taihill Venture and Metaplanet also participated.
This funding round brings Kano’s total funding to date to $7.1 million. And 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.
The 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. And currently, nearly 3,000 cell and gene therapies are in active development. Despite this growth, challenges remain when it comes to inserting genetic information into the genome for therapeutic purposes.
The current techniques typically depend 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. And to cure genetic diseases – the majority of which have no available treatment options – targeted, non-viral gene insertions via ssDNA are key.
Combining 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. And 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, enabling scientists, labs and companies with the building blocks they need to devise treatments for genetic disorders, cancers and other high unmet need conditions.
Kano utilizes bioreactor-based production to manufacture long, circular ssDNA repair templates. And the company’s platform connects manufacturing, application and computational tools, serving as a comprehensive, end-to-end solution that:
1.) Breaks down long-standing manufacturing bottlenecks by delivering kilobase ssDNA at a 10x lower cost and drastically increased scale
2.) Enables gene constructs with low toxicity and high insertion efficiency for therapies that were previously inaccessible
3.) 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
Spun out of the Bathe BioNanoLab at MIT’s Laboratory for Nucleic Acid Nanotechnology and Kano is founder-led and indication-, disease- and technology-agnostic. And 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 next few 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.
KEY QUOTES:
“We believe curing genetic diseases isn’t just a biology problem; it’s an engineering problem. 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.”
– Dr. Floris Engelhardt, co-founder and CEO of Kano
“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.”
– 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.”
– Lise Rechsteiner, General Partner at VSquared Ventures
