GenEdit Uncloaks to Solve Gene Editing “Delivery Problem”

DNA Gene Editing

Since Jennifer Doudna and her team published their CRISPR-Cas9 discovery, gene editing has been all the rage. Unlocking the endless potential for disease curation, the techniques currently in use have one major hang-up delivery.  

After five years of working quietly behind the scenes, Kunwoo Lee has uncloaked his solution to the gene-editing “delivery problem” with $26 million in Series A funds for his company, GenEdit.  

Because traditional approaches use adeno-associated viruses to deliver gene therapies, some patients already have a natural immunity to the virus used, or they will become immune after just one dose. GenEdit’s approach uses polymer nanoparticles to deliver DNA, RNA or CRISPR ribonucleoprotein to add, delete, edit or silence a gene to treat disease. This approach does not trigger an immune response in patients, enabling a “universe of opportunities” for next-gen genetic medicines. 

“Solving the delivery problem is what will truly launch genetic medicines to being an essential therapeutic modality for a wide range of diseases,” said Andrew Adams, VP of New Therapeutic Modalities at Eli Lilly and board observer at GenEdit. Lilly was a new investor for the Series A round.  

While GenEdit has been quiet about specific targets, the company identified diseases of the nervous system. Funds from Series A will support the development of GenEdit’s NanoGalaxy platform while identifying a candidate to take on IND-enabling trials.  

GenEdit is also building out the team. Wednesday, the biotech announced two additions to its C-suite. Romuald Corbau joins as the chief scientific officer. With two decades of experience, Corbau recently led gene therapies from innovation to clinical development at Spark Therapeutics and Freeline. Aaron Mishel also joined as chief financial officer. 

Two more startups announced their funding rounds on opposite coasts today. In Cambridge, Garuda Therapeutics is up and running with $72 million Series A to develop the “world’s first, off-the-shelf hematopoietic stem cell platform that will eliminate dependency on donor or patient cells.” 

With self-renewing blood stem cells, patients would have easy access to long-lasting, consistently matched blood stem cell therapies. This is especially important for minority and mixed-race patients who often face greater challenges to finding a match.  

Garuda believes their technology has the potential to address and potentially cure more than 70 diseases by transforming blood stem cell transplants and “revolutionizing the landscape of medicine.” The current pipeline includes programs for hematologic malignancies, sickle cell disease, β-thalassemia, bone marrow failure diseases and more.  

“Eliminating the need for donor or patient cells while gaining the ability to exploit stem cell engineering would democratize blood stem cell transplants, ensuring this vital, and often curative, therapy can be made accessible to any bone marrow transplant eligible patient in need,” said Dhvanit Shah, president and CEO of Garuda. 

Across the country in Menlo Park, California, Hexagon Bio is freshly stocked with $61 million to fuel its microbial genomes platform. Twelve months after its $47 million launch, the company is focused on turning “nature’s hidden molecules” into first-in-class drugs.  

Proceeds from this latest raise will funnel into expanding the genomics database to accelerate drug discovery efforts for “high-value anti-infective and oncology targets.” The biotech company is applying modern-day tech to age-old approaches. Penicillin and other antibiotics were discovered through fungi and other microbes. Using genetic sequencing and AI, Hexagon is determined to uncover more opportunities. 

“Microbial genomes are an incredibly rich source of diverse, potent and drug-like compounds that, as a result of millions of years of evolution, provide elegant solutions to human therapeutic targets that have eluded traditional screening approaches. To date, the discovery of these small molecules has been limited to a tiny fraction of the earth’s microbes and hampered by a lack of mechanistic understanding of the intended drug targets,” said Maureen Hillenmeyer, Ph.D., Hexagon’s co-founder and CEO. 

“Hexagon exists to overcome these challenges and unearth new medicines to combat human disease. We are excited to embark on the next phase of growth to discover and develop unique and potent therapeutic compounds for a broad spectrum of intractable diseases.” 

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