It was Christmas Eve in 2015. The seasonal festive vibe was blocked out from the science lab in UC Berkley, California, where bioengineering doctoral candidate Lee Gun-woo, then 27, was immersed in his research.

Lees heart quickened as he watched the results of his experiment unravel before his eyes. A polymer nanoparticle traveled straight to his intended target genome in a test rat and changed its DNA.

A jolt ran down his spine. This could make CRISPR DNA scissors work in the human body. It could be a game changer in delivering drugs to tackle genetically caused illnesses, he thought.

Lee dialed up his friend and fellow scientist Park Hyo-min.

Hyeong, lets start a company, Lee said.

The conception

The story of Lee Gun-woo, 33, and Park Hyo-min, 41, is truly an American Dream come true.

The duo co-founded the gene therapy company GenEdit in May 2016 with Lee as the chief executive officer leading research and Park as the chief technology officer responsible for validation of development.

What GenEdit does is step up the game for the groundbreaking DNA scissors technology called CRISPR.

The technology snips parts of the human DNA to remove or insert new genetic material. The Nobel Prize for Chemistry in 2020 went to scientists Emmanuelle Charpentier and Jennifer Doudna for their contribution to its discovery.

Lee and Park are not strangers to the leading CRISPR researcher Doudna, who is a professor at their alma mater UC Berkley.

Doudnas name is listed as a contributor to Lee and Parks co-authored article published in Nature in 2017, titled Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair. Lee, Park and scientist Michael Convoy have equal stakes in the piece as main authors, according to the science journal.

CRISPR gene editing is a two-part system comprising a customizable guide RNA and a protein called Cas9.

The guide RNA directs Cas9 to any desired segment of DNA for editing. The Cas9 enzyme then cuts the DNA at that precise location, allowing for genes to be turned on or off or for the removal or insertion of DNA. But editing the DNA of cells in a petri dish -- or even curing a mouse of a disease -- is one thing; making the hot new technology work in humans is a whole other challenge. Sneaking the gene-editing complex into human cells is no easy task, according to American Chemical Societys Chemical & Engineering News introductory article dated 2017.

This is where GenEdit steps in.

Born out of UC Berkleys laboratory, GenEdits polymer nanoparticle delivery technology can make the Cas9 travel to the desired location in human body. This innovative concept has attracted global angel investors such as the likes of Sequoia Capital, Bow Capital, Data Collective Bio and SK Holdings. The Series Seed financing round raised a total of $8.5 million, led by DCVC Bio and SK Holdings.

Before he knew it, Lee was named in Forbes top 30 entrepreneurs under the age of 30 in 2018.

5th anniversary

Now in its fifth year, GenEdit aspires for the future where all hereditary illness can be cured with gene therapies.

There are countless ailments that are passed down the blood line and GenEdit is studying DNA markers for non-viral polymers that will match them.

Speaking to The Korea Herald in a video interview, CTO Park Hyo-min said that GenEdit will soon decide on the first target disease within 2021.

Were not definitive at this point, but there is a good chance we will narrow down our focus to central nervous system diseases, for which we have been able to amass a volume of promising data, Park said.

It is not with absolute certainty I say this, but we may be able to deliver a novel drug for a rare CNS disease within the next six to seven years, said Park, if authorities fast-track their approval for orphan drugs for rare diseases.

Park declined to say if Alzheimers Disease or dementia were research subjects.

We are aware that companies who tackle AD receive tremendous attention from investors. However, conditions like AD or dementia do not have a clear genetic cause, Park said, underlining that GenEdits target indications are only those with high correlation with genetic roots.

Other than CNS indications, the company is also perusing therapies for liver and immune cell diseases.

The liver has comparatively low hurdle for drug delivery mechanisms, Park said, Precisely for that reason there is much competition in the area of liver treatments, but we may still consider to throw our gloves in.

Apart from their main target pipeline, for which GenEdit intends to see through to drug commercialization, the company is open to strategically licensing out other findings, Park said.

When asked what motivates these researches, and what does it feel like to be a young, celebrated scientist, CEO Lee Gun-woo -- who is eight years younger than co-founder Park -- remained modest.

Through this winding long process of life, I dream of serving the society in any way I can. To be able to serve, I believe its imperative to broaden my capabilities, Lee said.

I had the opportunity to listen to astronaut Jonny Kims webinar. He spoke of Martin Luther King and the life of service, that everyone is capable of greatness through the act of giving, Lee said. One of my dreams is to use science to benefit more patients, and I am profoundly grateful that I am on that path.

The young co-founders of GenEdit had not foreseen that they would be leading a gene therapy company in the US when they first set foot on US soil in 2011.

Lee had come straight after his bachelors degree in bioengineering at Korea Advanced Institute of Science and Technology in Daejeon, and Park had come after completing a masters in food science at Korea University in Seoul.

Lee had spent most of his life growing up in his home city of Daegu, while Park had lived in Seoul.

I would like to tell scientists in Korea that they must create reasons to come out to the US. Korea has great science, but one cant deny that its here in the US where all the breakthrough innovations happen, said Park.

If in Korea, every academic novelty would have to be indirectly studied. Here, everything becomes a raw, immediate experience. We get to be in the heart of the research leading scientific progress, shoulder-to-shoulder with Nobel laureates, Park said.

Being in southern California, we are adjacent to companies like Genentech. Remember the time when the only drugs available were swallowable tablets? Genentech is the company that pioneered the protein-based drug shots that revolutionized cancer therapies. We aspire to be the next Genentech in the realm of gene therapies, Lee said.

His source of joy is his parents who send him support from Daegu. Although there are limitations to their comprehension of the intricate science of Lees work, news of their son always fills them with pride, Lee said. Due to the COVID-19 pandemic, Lee has not been able to pay them visits as of late.

As for Park, he is most thankful to Lee and his wife for being a spiritual buttress. As a father of four, with his eldest now 12 years old, Park says he is especially thankful to Lee for clocking in more hours at work, and to his wife for her sacrificial love.

What binds Lee and Park together as partners is the deep trust and camaraderie that has built up in the decade they have known each other. The five years in school and five years in business have united them as near-family and made them an inseparable team.

As of February, GenEdit had 17 full-time employees, of whom 14 were researchers. By the end of the year, GenEdit plans to boost the headcount to 27 full-time workers. Lee and Park said that they have built a culture where it is OK to make mistakes and keep matters transparent. Anyone who wants to try some cool science and do fun researches is welcome to join this science-focused team, they said.

By Lim Jeong-yeo (kaylalim@heraldcorp.com)

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[Herald Interview] GenEdit to hone in on nervous system disorders in 2021 blueprint - The Korea Herald