MIT spinout leverages single strand DNA to ‘bridge the hole’ between gene modifying and gene substitute.
Gene modifying expertise firm Kano Therapeutics has closed an oversubscribed $5 million seed funding spherical to advance its platform designed to allow focused, non-viral gene insertions. The corporate was spun out of MIT’s Bathe BioNanoLab and describes itself as “disease-agnostic” – aiming to deal with a variety of advanced ailments by bridging the hole between gene modifying and gene substitute.
Present gene modifying strategies usually depend on double-stranded DNA (dsDNA) donors or viral vectors. In line with Kano, dsDNA donors face toxicity and effectivity challenges, whereas viral vectors can introduce security considerations and complicate manufacturing and redosing processes.
Kano is targeted on advancing gene modifying expertise via the event of single-stranded DNA (ssDNA) as a brand new class of biomolecules, which the corporate claims present a safer, extra environment friendly and versatile technique for gene insertions. Whereas a typical dsDNA molecule is shaped from two complementary strands of nucleotides, an ssDNA molecule consists of solely a single strand, permitting for the environment friendly substitute of whole genes with lowered danger of immune response.
Kano is targeted on precision manufacturing of ssDNA in customized lengths and sequences at scales appropriate for drug growth. The corporate employs a bioreactor-based manufacturing system to fabricate lengthy, round ssDNA restore templates, successfully breaking down long-standing manufacturing bottlenecks by delivering kilobase ssDNA at a tenth of the standard price and at considerably elevated scales.
“We consider curing genetic ailments isn’t only a biology drawback; it’s an engineering drawback,” mentioned Dr Floris Engelhardt, co-founder and CEO of Kano. “The supplies we have to ship on the total promise of genetic therapies are incomplete. For this reason we’re constructing a ‘one-stop store’ product growth platform for safer DNA payloads at scale. By accessing a brand new class of gene restore templates, Kano can develop the capabilities of at the moment’s modifying strategies, and proper longer stretches of DNA successfully and flexibly.”
The seed funding, which brings the full raised by Kano to $7.1 million, was co-led by The Engine Ventures and VSquared Ventures, with participation from Taihill Enterprise and Metaplanet. The funds will likely be allotted to additional develop Kano’s laboratory practices, design processes for the clinical-grade manufacturing of kilobase ssDNA, and advance each inner and exterior drug growth applications.
“Present genetic medicines can change a letter – or perhaps a phrase – of genetic data, however Kano is introducing the flexibility to switch whole sentences and even paragraphs,” mentioned Ann DeWitt, Common Companion at The Engine Ventures.
Trying forward, Kano plans to start growing its personal pipeline of ex vivo genetic medicines based mostly on kilobase gene insertion. The corporate additionally intends to develop its present therapeutic collaborations and scale up its manufacturing capabilities to provoke pre-clinical applications pushed by exterior companions.
