CRISPR Medicine Insider, November 19, 2019 - CRISPR Medicine


This is our first newletter. It's been a great start for CRISPR Medicine News with a ton of views and thank you for letting us know you enjoy it. A lot more exciting stuff is on the way (c;


Here is what we have been writing.

Setting standards in the CRISPR genome editing revolution

Samantha Maragh from the NIST (US National Institute of Standards and Technology) gives us the lowdown on finding standards and best-practices in the new and quickly evolving field of CRISPR medicine

CRISPR - an exciting gene therapy journey is on the way and it is fine.

Kevin Holden, head of science at Synthego corporation based in Silicon Valley, shares his thoughts on the beginning of CRISPR medicine and on automation and large data-sets as a way to best practices in gene editing.

Safety considerations for delivering CRISPR Cas9 medicines.
It's all about safety - an interview with safety director at AstraZeneca, Roberto Nitsch, about the key safety aspects of delivering potential CRISPR-medicine to patients.

CRISPR revolution enabled by these key breakthroughs, and why TALENs could make a comeback.

Well considered thoughts on "the sharing of science" from Jason Potter of Thermofisher and the breakthroughs that led to the CRISPR revolution.

More good reads

Top picks

  • First step for CRISPR gene editing to fight cancer. The study involved just three patients - two with multiple myeloma and one with sarcoma - and shows only feasibility and safety. Scientists used CRISPR to knock out three genes to enable the patients own immune cells to attack cancer cells more aggressively. Results are very preliminary and data is to be presented at a conference in Florida next month. Also here and here.
  • At The Scientist Emma Yasinski has a good explainer of the new Prime Editing prototype gene editing method. Combining CRISPR-Cas9 and base-editing David Liu and colleagues at the Broad Institute cuts only one strand of DNA to gain better precision and similar efficiency as CRISPR-Cas9 solo. Also here and here.
  • Russian ‘CRISPR-baby’ scientist has started work. David Cyranoski, Nature News, reports that biologist Denis Rebrikov's controversial 'CRISPR-baby' plans have taken a turn from making HIV resistant babies to repair the gene linked to deafness, called GJB2.
  • And meet a deaf woman who considers working with Rebrikov.
  • Cellectis “Smart” CAR T-Cells Proof-of-Concept in Nature Communications study - shows solid tumor potential
  • Promising new gene therapy for Duchenne muscular dystrophy using dystrophin's distant cousin utrophin
  • Geneticists retract study suggesting first 'CRISPR-babies' might die early

Vision and opinion

  • Leading scientists and ethicists call for a global moratorium on heritable genome editing. »Certainly, the framework we are calling for will place major speed bumps in front of the most adventurous plans to re-engineer the human species. But the risks of the alternative — which include harming patients and eroding public trust — are much worse.«
  • STAT news has ingenious David Liu answer your questions on CRISPR, Prime Editing and more.
  • Bill Gates og NIH invests in CRISPR gene therapy for the poor
  • NIH director, Francis S. Collins, lays out inspirational vision for the future of medical science
  • Jennifer Doudna on the gene-editing revolution


  • Gene editing pioneers 1 year-old company, Beam Therapeutics, to go public to develop new treatments based on base editing.
  • Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold (again)
  • Bluebird Bio gets green light from European health regulators for manufacturing blood disease gene therapy
  • 100 infants treated for spinal muscular atrophy with first commercialised gene therapy
  • Novo Nordisk haemophilia gene therapy with Bluebird Bio

Uh ah wow

And that's it

Thank You for now - more good stuff is on the way.

NB The newsletter and blog are always evolving - tell us what you think!
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(c; Rasmus


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