Tag Archives: CRISPR

The Man Who Wrote the Book on CRISPR and What He Taught Me

Kevin Davies literally wrote the book on CRISPR, the revolutionary new gene editing technology that earned Jennifer Doudna and Emmanuelle Charpentier a Nobel Prize a few months ago. Today, I had the wonderful opportunity to attend a talk he gave on that superb book, Editing Humanity.

If you’re not familiar, CRISPR is a technology based on the immune systems of bacteria. Bacteria find a particular genetic sequence they recognize from past infections and cut the genes in order to protect themselves. Scientists have harnessed this primordial system to cut and splice genes.

In today’s talk, Davies highlighted how accessible this revolutionary new technology really is. The equipment is not expensive and many labs could potentially use it. This presents great opportunities but also very real risks of misapplication.

CRISPR has been used successfully to treat sickle cell anemia in early trials. Davies noted that it has also been used to treat progeria in mice, which might some day bring an end to this deadly disease that ages children before their time.

I even got the opportunity to ask Davies a question, and inquired which other applications of CRISPR excite him most. He mentioned possible applications for cystic fibrosis and cancer therapy. He also said that as a graduate student in genetics, the idea of precisely editing genes seemed like science fiction, but today is a reality. It amazed me to think of how much the field has evolved.

Another interesting tidbit from the talk: due to COVID, Doudna accepted her Nobel Prize in the backyard of her home in Berkeley! I found that image to be quite a beautiful one.

One great silver lining of COVID has been how much easier it’s become to attend talks like this! In the past, one might have had to be in Cambridge, MA to attend, but now it’s open to everyone. I hope we continue to offer a remote option for these discussions even once in person events are possible again.

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Photo: “File:Emmanuelle Charpentier and Jennifer Doudna.jpg” by For Emmanuelle Charpentier portrait, credit Bianca Fioretti of Hallbauer & Fioretti. For Jennifer Doudna portrait, credit User:Duncan.Hull and The Royal Society. is licensed under CC BY-SA 4.0

I Just Went to a Meeting With Two Experts on Gene Editing. Here’s What I Learned

Photo: Shengfang Jin, PhD, Vice President and Head of Discovery Biology, Editas Medicine

This morning, I attended an online presentation by two experts on gene editing and CRISPR. Shengfang Jin, VP of Editas Medicine, and Andrew Lin, who heads the Rare Disease Center at WuXi AppTec, presented a lot of great info, but what stuck out to me most was the trial Editas is doing to cure a rare eye disorder.

Leber congenital amaurosis type 10 generally causes people to be born blind or lose their sight by age 10. I can only imagine how difficult it must be. A new therapy from Editas targets this disease with a one-and-done treatment. This therapy is the first use of CRISPR in a human being, as opposed to a cell culture, and is currently in Phase 1/2 clinical trials.

Listening to Dr. Jin speak, I was put in mind of this passage from the Bible:

“the blind can see, the lame can walk, those who suffer from dreaded skin diseases are made clean, the deaf hear, the dead are brought back to life, and the Good News is preached to the poor. “

Matthew 11:5

I increasingly suspect we are at the dawn of a golden age in medicine. The rapid pace of COVID vaccine development or this first in vivo use of CRISPR may be the first signs of things to come.

P.S. The presentation should be up on the website of Genetic Engineering & Biotechnology News here soon.

What I Just Learned From a Discussion With Nobel Laureate Jennifer Doudna

Today I attended a fascinating discussion of the movie Human Nature, a documentary on CRISPR gene editing. Nobel Prize winner Jennifer Doudna, the co-inventor of CRISPR, gave us her perspective on the technology. Author Walter Isaacson and the filmmaker, Adam Bolt, also gave valuable insights.

Isaacson framed the moment well, saying that CRISPR is part of the 3rd great scientific revolution. The first was in the first half of the 20th century, in physics. The 2nd, in information technology, consumed the second half of the century. And in the 21st century, the revolution is and will be in the life sciences.

Because CRISPR can make DNA and RNA programmable like computer code, there’s a strong parallel between CRISPR and the IT revolution. What if biology and medicine progressed the way software has in the last few decades?

Doudna is particularly excited about the applications of CRISPR to cure cancer. CRISPR can be used to program the patient’s immune cells to attack tumors. This echoes what the co-founders of BioNTech said earlier this week at a call I attended.

Bolt and Doudna also noted that CRISPR was a scientific backwater at first. This really emphasizes the importance of funding basic science with no clear application. We never know where it will lead!

The first patient to be treated for sickle cell anemia using CRISPR is doing quite well a year later. Doudna and others are hard at work on further applications of this technology. As an investor and as a human being, I am eagerly anticipating these breakthroughs.

“Jennifer Doudna” by Koninklijke Nederlandse Akademie van Wetenschappen is licensed under CC BY 2.0

An Outstanding Science Movie and Your Chance to Talk to a Nobel Prize Winner

I just finished watching the superb documentary Human Nature, which details the origins and applications of CRISPR gene editing. The filmmakers interview all the leading people in the field and produce a fascinating and highly accessible narrative.

I found particularly striking how one of the co-inventors of CRISPR, Jennifer Doudna, enjoyed being in an unknown field at first but also wondered whether the field was neglected because it was a dead end. It shows us what we can accomplish when we overcome our self-doubt!

You can see the movie and attend a talk tomorrow at 1pm EST with Nobel Laureate Jennifer Doudna here, both free of charge. It’s not often a person gets the chance to ask questions of a Nobel prize winner. I’ll be there!

P.S. If you’re interested in CRISPR, I recommend this book.)

“Micah’s DNA” by micahb37 is licensed under CC BY-SA 2.0

How We Could’ve Known Theranos Was a Fraud

Investors lost over $9 billion on Theranos, the diagnostics testing company run by Elizabeth Holmes that was exposed as a fraud. Worse yet, patients received inaccurate test results, imperilling their health. But what if asking one simple question could’ve prevented all this?

In the book Editing Humanity, Keith Davies notes that Theranos’ technology had no foundation in peer-reviewed, published research. The always-astute John Ioannidis pointed this out as early as 2015. Theranos published a single paper in 2018, after the company was already exposed as fradulent and was near bankruptcy. Theranos made incredible claims for its technology, but without peer review by respected journal, there was no one to check if those claims were actually true. (Theranos had filed patent applications, but that doesn’t necessarily provide the same level of detail and review.)

This stands in contrast to many other biotech companies, such as several in the CRISPR area (Editas Medicine, Intellia Therapeutics, etc.) whose approach grows out of research published in major journals. Also, such companies have major scientists as co-founders, not a 19 year old unknown.

I am considering getting involved in early-stage biotech investment, so asking “What papers can you point me to that underlie your technology?” is a question I plan to ask in the future. That may be able to weed out some weaker companies and potential frauds.

For more background on Theranos, I strongly recommend the outstanding book Bad Blood by John Carreyrou. I listened to it as an Audible audiobook. It was read by Will Damron, whose voice is outstanding.

I also hear excellent things about the ABC News podcast series The Dropout, which details Holmes’ path from Stanford student to billionaire to accused criminal awaiting trial today. I haven’t listened to it yet, but I look forward to getting a chance soon!

“File:Elizabeth Holmes 2014 cropped.jpg” by Photo by Max Morse for TechCrunch TechCrunch is licensed under CC BY 2.0

How We Can Rewrite Our Genetic Blueprint

We may soon be able to edit our own genes using a new technology called CRISPR. It uses a bacteria to change data in the genetic code and could one day cure rare genetic diseases. There is also potential in editing genes to prevent cancer and heart disease.

In the new excellent book Editing Humanity by Kevin Davies, we learn how the technology was developed and how it has been applied and misapplied. CRISPR has cured a patient of sickle cell disease already, but was also used inappropriately to sloppily modify the genes of several babies in China. It’s tool with enormous potential, but that includes potential for abuse.

I am intrigued to see if this tool could one day be used to reverse the DNA errors that come with aging.

As an investor, it struck me that Beam Therapeutics, a company founded by David Liu and Feng Zhang, two of the best scientists in the field, went public at a valuation of only $180 million. If CRISPR doesn’t revolutionize health, perhaps it’s worth zero. But what if it succeeds? It could be the most valuable company in history. Indeed, since this book came out in mid-2020, I see the share price has increased nearly fivefold.

I also think we would benefit greatly from increasing our investment as a society in basic research. I was surprised to learn that the Howard Hughes Medical Institute supports the lab of one of the co-inventors of CRISPR, Jennifer Doudna, at a rate of $1 million per year. While HHMI’s work is outstanding and to be commended, I imagine Doudna could use a lot more than that. What would she do with $10 or $100 million a year? Our government and we as individuals (through crowdfunding) could get it to her and other leading scientists. The benefits we could reap can only be imagined.

Davies’ book is entertaining, readable, and informative throughout. I’d highly recommend it to anyone with an interest in CRISPR, gene editing, or health in general!