Welcome 2020!

A new decade of exciting discoveries awaits on CRISPR Medicine News. Read on about one of the most remarkable developments in 2019: Prime Editing and discover how Cas12a is being engineered to a superior gene-editing tool. Also, don't miss some of our highlights of 2019, like a new gene-editing platform that could replace CRISPR/Cas9 or CRISPR as a bacteria killer.
And last but not least, we have an opening for a student position!

Here is what we have been writing.

Prime editing is the new versatile 'word processor' for genome editing. Interview: Andrew Anzalone
Prime editing made the Nature top 10 papers of 2019 with a Nature paper describing a new CRISPR tool that enables incredibly versatile genome editing: All possible point mutations as well as deletions and insertions. First-author Andrew Anzalone talks to us about discovering prime editing.

Cas12a: Engineering superior tools for the CRISPR toolbox. Interview: Stefano Stella, Twelve BIO
They solved the CRISPR Cas12a structure, discovered how the unique Cas12a enzyme cuts DNA, and are now engineering superior variants for gene editing and diagnostics. Stefano Stella, CEO Twelve BIO, talks about the work.

More good reads

Top picks

• Great piece in the New York Times on the youngest person ever to get gene therapy. Treatment for sickle cell disease which affects millions of people globally.

• A Chinese court sentenced biophysicist He Jiankui to three years in jail, a fine and a research ban for creating the world's first gene-edited babies. Two other researchers received lesser prison sentences and fines. Also, Scientific American, New York Times, and BBC plus lots of commentaries Conversation, STAT1, STAT2, and Genetics&Society.

• CRISPR fighting disease. The first clinical-trial results are in, and CRISPR gene therapy for a host of diseases is moving closer to reality in 2020.
• CRISPR in space, flu-free CRISPR-chickens, and other good CRISPR stories from 2019.
• After nearly half a century, the concept of genetic medicine has become a reality: nine gene insertion treatments and five gene interference treatments listed by Scientific American.

Research

• Simple strategy increases CRISPR knock-in efficiency by chemically modifying DNA-ends.

• "I envision there being a whole catalog of CRISPR cofactors that can be used to enhance CRISPR activity." On how cofactors can move chromatin packaging out of the way to give CRISPR access to the DNA.

• Temporal control of CRISPR gene editing systems by reversibly masking gRNA.

• Great work. Cell paper provides an ex vivo strategy to treat cystic fibrosis using Cas9 and AAV.

• Researchers develop a method to regulate nucleic acid cleavage and gene editing in live cells.

Industry

• Locus Biosciences initiate the world's first controlled clinical trial for a CRISPR enhanced bacteriophage therapy.

• Promising. Accelerated program for gene therapy treatment of children with rare disease Sanfilippo syndrome type A (MPS IIIA).

• Exciting times for gene editing. Pharma's gene and cell therapy ambitions will kick into high gear in 2020 - despite some significant hurdles.

• $61 million boost to expand the CRISPR tool kit. Emendo Biotherapeutics are synthesizing Cas9 alternatives.

• CRISPR driver behind Synthego's move to adopt GMP standards for producing sgRNA.

Uh ah wow

• Nobel Prize one step closer? Doudna and Charpentier win Israels Wolf Prize.

• CRISPR gene therapy (surprise) in top biotech trends to watch in 2020. Also at The Scientist.

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!
Please send your feedback, tips, and suggestions to editor@crisprmedicinenews.com

(c; Rasmus

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