(LaCasadeGoethe, Pixabay)
11 Dec. 2018. A start-up company developing nanoscale particles to deliver gene-editing enzymes, including those for Crispr, is raising $8.5 million in its seed funding round. GenEdit Inc. in Berkeley, California is a spin-off enterprise from University of California in Berkeley, founded by a biomedical engineering professor and 2 recent Ph.D. graduates leading the company.
GenEdit is developing a delivery technology for gene editing processes, with the best known technique being Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The actual editing of genomes with Crispr in most cases today uses an enzyme known as Crispr-associated protein 9 or Cas9. RNA molecules guide the editing enzymes to specific genes needing repair, making it possible to address root causes of many diseases.
Up to now, most Crispr processes use benign viruses, such as adenoviruses, to deliver the editing enzymes to DNA in the target cells. Adenoviruses, while relatively harmless to most people, can be troublesome for people with weakened immune systems, or people with respiratory or cardiac disorders. As a therapeutic technology, Crispr would need to find a way of removing risks of adverse effects from viruses that deliver editing enzymes, or use a method other than viruses for delivery.
GenEdit is commercializing research from the lab of UC-Berkeley professor Niren Murthy that studies delivery of small molecule and biologic therapies with micro- and nanoscale particles. Murthy, with doctoral students at the time Kunwoo Lee and Hyo Min Park, demonstrated the use of gold nanoparticles to deliver Cas9 enzymes with Crispr to edit the relevant genes in lab mice induced with the inherited diseases Duchenne muscular dystrophy and fragile X syndrome, a common single-gene form of autism spectrum disorders.
In an August 2018 publication, Murthy, Lee, and Park demonstrated delivery of the more precise Crispr Cpf1 enzyme with nanoscale particles made of polymers instead of gold. GenEdit’s technology offers this polymer nanoparticle method to deliver enzymes for Crispr, as well as the other genome editing techniques zinc finger nucleases and TALENs. The company says it’s creating a library of polymer nanoparticles to deliver these enzymes and proteins designed to interact with specific cells and tissue in nerves, muscle, liver, and blood.
GenEdit was founded by Lee, now the company’s CEO, with Park and Murthy in 2016. While GenEdit gained initial funds for its launch, the company now raised another $8.5 million in seed funds, led by technology investment companies Data Collective in Palo Alto and San Francisco, and SK Holdings in Seoul, South Korea. Joining the seed round were GenEdit’s earlier funders Sequoia Capital and Bow Capital.
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