CRISPR Medicine Insider, December 23, 2019 - CRISPR Medicine

Happy holidays!

We have been busy on CRISPR Medicine News - Read on to discover how CRISPR cause high levels of AAV integration, how SNIPR BIOME is turning CRISPR into a bacteria killer, a new platform that could replace CRISPR/Cas9 and how to use CRISPR to cure severe hereditary immunodeficiences.


Here is what we have been writing.

CRISPR cause virus vector to integrate at high level. Interview: Bence György.
Safe and efficient delivery tools are crucial for successful gene therapy. Adeno-associated virus vectors (AAV's) has so far been accepted by the FDA in two trials with CRISPR. However in a new study Bence György and collaborators have shown that CRISPR-Cas9 cause AAVs to integrate into the chromosomal DNA of the target cells in high frequency. This potentially raise safety concerns.

Turning CRISPR into a bacteria killer. A new principle in antibiotics. Interview: Christian Grøndahl.
Bugs are winning: WHO has issued dire warnings of 'the post antibiotic era' in which any person regardless of age or country risk to suffer and die of intreatable bacterial infections. Multi-drug resistance is spreading fast, and we are running out of ways to kill bacteria not least because of a decades long discovery void of almost no new antibiotics.
This is where SNIPR BIOME may change things...

Move over CRISPR: a new platform, TGEE, could soon replace everybody's favorite gene editing tool. Interview: Yoel Shiboleth.
While CRISPR has taken the world by storm a small Israeli company, TargetGene Biotechnologies, has a much more precise genome editing platform they invented and developed under the radar. It could be a game-changer for gene therapy.

Using CRISPR to cure severe hereditary immunodeficiences. Interview: Rasmus O. Bak.
A new CRISPR/Cas9 research center, PASCAL-MID, aims to lay the foundation for future gene therapies in Denmark. In short the bigger plan is to extract stem cells from the patient, correct the genetic mutation outside the patient, and infuse the corrected stem cells back into the patient. Rasmus O. Bak, one of five PI's, gives us insights on the strategy to develop the CRISPR tools to be used for gene editing at clinical scale in patients.

More good reads

Top picks

  • CRISPR Babies: MIT's Technology Review reports exclusive excerpts from He Jiankui’s unseen original research manuscript on creating the gene-edited twins Lulu and Nana.
  • Big news for CRISPR and safe gene therapy! First data from CRISPR-based therapies to treat sickle cell disease and beta thalassemia shows promising results. Still, early days though. And here and here.
  • And also meet Victoria Gray, the first CRISPR sickle cell patient and listen to her story in this podcast.
  • Great feature on the huge challenges facing gene therapy not just technologically but also ethically.
  • Prime editing selected among Nature top 10 papers of 2019 - CRISPR tool enables precise genome editing by copying RNA into the genome.


  • Great review on advances in genome editing through control of DNA repair pathways by leading CRISPR scientist Jacob E. Corn.
  • The ins and outs of base editors. Excellent feature by Sandeep Ravindran, Nature.
  • CRISPR gene drive could counter rising antibiotic resistance crisis. A CRISPR-based system targets amplified antibiotic-resistant genes - demonstrates 100-fold improved efficiency in targeting antibiotic-resistant bacteria.
  • Insight from GEN on in vivo CRISPR delivery options - viral vectors, lipid-based nanoparticles, and polymer formulations.
  • A new gene therapy strategy, courtesy of Mother Nature - Scientists turn a natural cellular process into a drug-delivery system

Vision and opinion

  • How Far Are We from (Accurately and Safely) Editing Human Embryos?
  • CRISPR this, and CRISPR that. Why is CRISPR constantly in the headlines but RNAi, so much farther ahead, relatively unknown?
  • And this very nice Milestones in antisense RNA research from Nature.
  • And now amidst CRISPR baby concern: Bioethicist argues that we need a CRISPR baby industry ASAP. Full argument in bioethics paper.
  • A moratorium on heritable human genome editing is not enough argues Jennifer Doudna in Science one year after chinese CRISPR babies.
  • Russian consensus that human genome editing on embryos and germ cells is premature, 15 geneticists, clinicians and bioethicists writes in a letter to Nature.


  • Thermo Fisher Unveils Viral Vector Manufacturing Expansion.
  • Astellas Pharma has agreed to acquire Audentes Therapeutics for approximately $3 billion cash, in a deal the companies say is intended to create a top-tier gene therapy developer.
  • GenEdit and Editas Medicine signed a deal.
  • »Can a non-profit change the economics of drug development in a time of ever-rising drug costs for the healthcare system?« Harvard consortium to build $50M cell and gene therapy center. And here.
  • New gene therapy bladder cancer drug fase III results to be presented 5 Dec. Ferring and Blackstone invest $570MPress release
  • Gene therapy global boom, $2 billion spending spree in production facilities for future gene therapies

Uh ah wow

  • 60 minutes episode: George Church's lab at Harvard Medical School is working to make humans immune to all viruses, eliminate genetic diseases and reverse the aging process.
  • CRISPR the movie - Human Nature - showcases biology’s hottest tool up to the point when things went awry, review by Amy Maxmen.
  • And speaking of when things went awry: Where is He? AP News takes a look at the fate of He Jiankui and the CRISPR babies 1 year after He shocked the world. It's a mystery. No one seems to have heard from him or them since early 2019.

And that's it

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

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Happy holidays!
(c; Rasmus


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