Fyodor Urnov
@UrnovFyodor
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Gene editor + Professor Molecular Therapeutics @berkeleyMCB + Director for Technology and Translation @igisci + @danahercorp Beacon for CRISPR Cures.
Berkeley, CA
Joined November 2018
A triumph of science, a miracle of medicine - built on decades of public investment into the US basic and translational biomedical research ecosystem. Magnificent effort led by Drs Rebecca Ahrens-nicklas (CHOP) and Kiran Musunuru (Penn) + IGI + Danaher. https://t.co/zzckbvOyNT
innovativegenomics.org
The first on-demand CRISPR therapy for an infant with a rare metabolic disease developed by the IGI and collaborators around the world.
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F1.1 @tazvonrue acquired and edited this gem at Soho Press. ❤️ I am not biased in the slightest. 😀 https://t.co/iKr4sxaWv5
npr.org
Philip Miller's sinister thriller is set in a Great Britain that's lost its bearings. But even when she's terrified, fictional journalist Shona Sandison will always risk everything to get the story.
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Watch. Weep tears of joy. Get inspired.
Official video with a general-audience summary of base editing and prime editing: https://t.co/6WRYBb6W7g
@brkthroughprize
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This. Is. Not. A. Dire. Wolf. Not. Even. Close. The CSO of Colossal has publicly stated as much (she is a scientist). No amount of salesmanship by the business people behind Colossal can change laws of biology. Enjoy video of a cute wolf puppy and savor being science-savvy.
Never-before-seen footage of Khaleesi, the first de-extinct female dire wolf, growing up. Here’s a rare look at months 1 through 3—from socialization to the start of the ranking phase. She’s growing fast, and if she gets any cuter we’re printing calendars.
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"At the end of every hard-earned day People find a reason to believe" (Springsteen) Watch this video. It's data-based reason to believe. Don't forget Kleenex. I watched this video 4 times and cried every single time.
10-month-old KJ Muldoon, who landed in international headlines after receiving a first-of-its-kind gene-editing treatment, was released from the hospital last week. The bespoke therapy was developed by @kiranmusunuru & Dr. Rebecca Ahrens-Nicklas.
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This FDA is an FDA interested in listening and learning and evolving, not just our thinking in regulation, but also our processes at the FDA. Here's more from yesterday's FDA Cell and Gene Therapy Roundtable. https://t.co/0t7owXGbB8
LIVE NOW: FDA's Cell and Gene Therapy Roundtable Captions available here: https://t.co/QyRYlaur9t
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Anyone interested in, or developing, gene editing medicines is well advised to watch this video. In it, HHS, FDA, CMS, and NIH leadership affirm their support for scaling up such medicine development in the US, and the key role NIH-supported biomedical research plays in it.
After this week’s cell and gene therapy roundtable at @US_FDA, @SecKennedy joined @DrOzCMS, @DrMakaryFDA, and @NIHDirector_Jay to deliver a message directly to the American people about investing in cutting-edge cures. Cell and gene therapy holds promise—but it also demands
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.@SecKennedy said yesterday at the FDA: "CRISPR gives the gift of life." He is 100% correct. I appreciate HHS, FDA, CMS, NIH, and CBER leadership devoting their morning to communicate their clear support for CRISPR giving such a gift to all of our nation's children who need it.
Thursday morning, I had the privilege of speaking with the Muldoon family, whose 10-month-old son, baby KJ, was discharged Wednesday from @ChildrensPhila. Baby KJ was diagnosed with a rare urea cycle disorder and received a first-of-its-kind personalized CRISPR gene-editing
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My entire generation grew up on that legendary rotascoped video. Still a sui generis. My CRISPR cures mind now refilms it where he gets rescued by a doctor who gives him a bespoke medicine. https://t.co/vxxT1Z7f9C
nytimes.com
Harket, best known for his band’s infectious 1985 synth-pop hit “Take on Me,” revealed his illness in an interview on a-ha’s website.
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After speaking with Baby KJ's physician and parents Secretary Kennedy said today: "CRISPR gives the gift of life." My remarks to HHS, FDA, CMS, NIH, and CBER leadership are below: we can and we must provide that gift of life to all children who need it. https://t.co/XYiAMhmFaI
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Baby KJ, the world's first patient to receive personalized CRISPR gene editing therapy for a rare metabolic disease, is now home and thriving! This groundbreaking treatment offers hope for other untreatable genetic conditions. #CRISPR #GeneEditing
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No words - only tears of joy. And bow of gratitude to Rebecca Ahrens-nicklas (CHOP) and Kiran Musunuru (Penn) and Sadik Kassim (Danaher) - our amazing partners in designing, derisking, and administering a CRISPR on demand to Baby KJ.
In case you needed a smile this Tuesday: 10-month-old baby KJ, who received a bespoke gene-editing therapy after a six month scientific sprint - "graduated" from the hospital today and went HOME! YAY SCIENCE! Here's his story: https://t.co/bOjDvHu4WL
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Ten years ago a piece in Nature entitled "Do Not Edit the Human Germline" authored by yours truly and colleagues argued against this dangerous and unnecessary application of editing. I thank the august lineup of authors of this timely piece for taking a strong stand on this.
International Call for a 10-Year Moratorium on Heritable Human Genome Editing - Cytotherapy
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.@Jasonmmast provides a clear-eyed summary of how we are getting from N=1 (Baby KJ) to N=many. @statnews
https://t.co/0ISXH8uyqA
statnews.com
The custom-built gene editing treatment for 6-month-old KJ Muldoon could not have come at a more welcome or jarring time for CRISPR-powered biomedicine.
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Interview: Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine -- A timely interview with @kiranmusunuru and @UrnovFyodor @GenomeObserv
https://t.co/7DEuk7MQCi
liebertpub.com
Moving CRISPR-based therapies from discovery to dosing patients in clinical trials and ultimately to approval involves navigating a challenging terrain of highs and lows. In this interview, physici...
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Eric continues to be his incomparable self in lucid, accurate, accessible descriptions of major biomedical advances. (in this case I'm biased since I was on the team)
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A CRISPR therapy produced in record time benefits an infant with an often deadly hereditary disease, with help from @UCBerkeley
https://t.co/JB3iWOwCLx
@ChildrensPhila
news.berkeley.edu
A newborn boy, KJ, was diagnosed at birth with a rare and potentially fatal genetic disease. He received a novel, on-demand CRISPR therapy in record time.
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An incredible feat...making a personalized, clinical-grade CRISPR gene therapy in 6 months that is actually efficacious! As @UrnovFyodor put it, this "is a triumph for the American peoples’ investment in biomedical research" https://t.co/ywPFaWgE0h
nytimes.com
The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.
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Researchers led by teams at Children’s Hospital of Philadelphia and University of Pennsylvania report on the first use of the gene-editing technology CRISPR in a customized therapy designed to treat a patient with a rare disease
time.com
The gene-editing treatment was designed specifically for the infant to correct his liver defect.
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The first person appears to have been successfully treated with a bespoke gene-editing therapy. This breakthrough could allow such treatments to one day become a routine option for children with debilitating genetic diseases
economist.com
Scientists hope more children will benefit
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