MIT spinout leverages single strand DNA to ‘bridge the gap’ between gene editing and gene replacement.
Gene editing technology company Kano Therapeutics has closed an oversubscribed $5 million seed funding round to advance its platform designed to enable targeted, non-viral gene insertions. The company was spun out of MIT’s Bathe BioNanoLab and describes itself as “disease-agnostic” – aiming to address a wide range of complex diseases by bridging the gap between gene editing and gene replacement.
Current gene editing techniques typically rely on double-stranded DNA (dsDNA) donors or viral vectors. According to Kano, dsDNA donors face toxicity and efficiency challenges, while viral vectors can introduce safety concerns and complicate manufacturing and redosing processes.
Kano is focused on advancing gene editing technology through the development of single-stranded DNA (ssDNA) as a new class of biomolecules, which the company claims provide a safer, more efficient and flexible method for gene insertions. While a typical dsDNA molecule is formed from two complementary strands of nucleotides, an ssDNA molecule consists of only a single strand, allowing for the efficient replacement of entire genes with reduced risk of immune response.
Kano is focused on precision manufacturing of ssDNA in custom lengths and sequences at scales suitable for drug development. The company employs a bioreactor-based production system to manufacture long, circular ssDNA repair templates, effectively breaking down long-standing manufacturing bottlenecks by delivering kilobase ssDNA at a tenth of the traditional cost and at significantly increased scales.
“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.”
The seed funding, which brings the total raised by Kano to $7.1 million, was co-led by The Engine Ventures and VSquared Ventures, with participation from Taihill Venture and Metaplanet. The funds will be allocated to further develop Kano’s laboratory practices, design processes for the clinical-grade production of kilobase ssDNA, and advance both internal and external drug development programs.
“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.
Looking ahead, Kano plans to begin developing its own pipeline of ex vivo genetic medicines based on kilobase gene insertion. The company also intends to expand its existing therapeutic collaborations and scale up its production capabilities to initiate pre-clinical programs driven by external partners.
