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Gene Therapy

Making Gene Therapy Faster And Better With Fuse Vectors



Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes blindness. The toddlers were treated using gene therapy, through which patients are given a harmless virus containing healthy versions of a defective gene in order to remedy the problem.

“Gene therapy sounds like science fiction, but it is beginning to have a huge impact,” says Benjamin Blaha, co-founder of the gene therapy-focused Danish biotechnology start-up Fuse Vectors, which is today announcing a $5.2 million pre-seed fundraising round. “We desperately need to develop more of these treatments as quickly as possible, because they can save lives and transform patient outcomes.”

Fuse hopes to play a significant role in doing exactly that. Blaha and co-founders Jordan Turnbull and Henrik Stage believe that the bottleneck for gene therapy developers today lies in the manufacturing process, rather than in the science itself. “If we want to develop new treatments faster, more safely and more cheaply, we need to solve the manufacturing problem,” Blaha adds. “Traditional methods of manufacturing gene therapies are akin to asking a blacksmith to build a jet plane.”

Fuse is focused on the mechanics of getting treatments into the human body – the “vector” for transmission. Currently, gene therapies make use of human cells – a technique developed for other types of drug delivery – but working with live cells in this way is inefficient and difficult to control. Fuse’s innovation is a technique that enables cell-free production of the viral vectors required; using protein and DNA as building blocks, it constructs tailor-made vectors for delivering the treatment.

“This allows for greater precision, more efficiency and increased scalability,” explains Turnbull. “You get higher drug quality, improving safety and efficacy by eliminating unwanted by-products, increased development speed from research to clinical application, and reduced production costs that make advanced therapies more affordable.”

The potential gains are significant. Fuse believes that its finished product is so tightly controlled that patients will need only a fifth of the dose currently required from gene therapies, which should be far safer. It says it can develop a new viral vector for testing in just four hours – compared to the four weeks it takes to develop a cell in the traditional way. And it thinks costs could be dramatically lower.

All of which is hugely exciting for healthcare professionals and the patients they want to treat. “There are thousands of addressable diseases that we need these therapies for,” adds Blaha. “The opportunity is to transform people’s lives.”

That said, the company is at a relatively early stage – particularly in terms of commercialisation. Fuse is pre-revenue, though it has signed up around a dozen partners, ranging from pharmaceutical companies to academic institutions, which will trial its technology in the coming months. The two founders hope to convert some of those partners into paying customers before the end of the year.

The company is pursuing two business models, with plans both to develop its own gene therapies and to give access to its manufacturing process to other developers. “The key is just to develop these treatments as quickly as possible,” says Turnbull.

New funding for the business should help in this endeavour. Today’s $5.2 million round is led by HCVC, an early-stage venture capital firm with investments in both Europe and North America. “With its unique cell-free viral vector solution, expert founding team and strong business model, Fuse Vectors has the potential to overcome significant challenges in the field,” says HCVC managing partner Alexis Houssou. “We believe in their ability to bring transformative treatments to patients.”

The funding round has also won support from BioInnovation Institute, an international non-profit foundation supported by the Novo Nordisk Foundation, and Denmark’s Export & Investment Fund. At the former, chief business officer Trine Bartholdy, says: “Fuse’s approach has the potential to make gene therapy much more interesting for the industry to develop as well as to increase its accessibility to patients.”

gene therapy, genetic engineering, genome editing, CRISPR, gene modification, genetic disorders, DNA repair, viral vectors, non-viral delivery, somatic gene therapy, germline editing, gene expression, targeted therapy, biotechnology, precision medicine, RNA therapeutics, cell therapy, molecular medicine, hereditary diseases, biomedical research

#GeneTherapy #GeneticEngineering #GenomeEditing #CRISPR #GeneModification #GeneticDisorders #DNARepair #ViralVectors #NonViralDelivery #SomaticGeneTherapy #GermlineEditing #GeneExpression #TargetedTherapy #Biotechnology #PrecisionMedicine #RNATherapeutics #CellTherapy #MolecularMedicine #HereditaryDiseases #BiomedicalResearch

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