The constellation of gene editing modalities in the clinic has grown! - arithmetically by 1, but conceptually by >>1.
RNA-defined repair of mutations and installation of longer DNA stretches could have an outsized impact on clinical use of CRISPR.
Gregorspeed, Prime Medicine!

... says AAV to LNP CRISPR.

[I will be hiding in the cold room behind stacks of expired LB-kan plates]

It is Saturday and my daughter and I decided to make the national breakfast dish * of #Egypt : ful mudammas.
Question: are the jalapeños mission-critical?
* I read this statement in this lovely book and apologize if the above is incorrect. themediterraneandish.com/cookbooks/

Cleese's work for Python ('this is an ex-parrot!') and Fawlty Towers ('please try to understand before one of us DIES') is sublime.
What a deep shame to see him advocate for snake oil.
Ubi nihil vales, ibi nihil velis, Mr Cleese.
H/t Paul Knoepfler
theguardian.com/science/2024/a…

Pfizer is _commercializing_ this therapy.
It was _developed_ by Spark Therapeutics under the leadership of Dr Katherine High Penn Medicine.
She also built Luxturna - the first approved AAV medicine.
Dr High is a hero and inspiration to all working in our field.
#WomenInSTEM

Original Article: Exagamglogene Autotemcel for Severe Sickle Cell Disease (CLIMB SCD-121) nej.md/3UvzPDT

Editorial: Extending Gene Medicines to All in Need nej.md/3QjCLAS

#Hematology

Dr Booth is leading the way for all genetic medicines in “rare disease” with her approach to have her hospital gain approval for the gene therapy she trialed.
👏🏻👏🏻👏🏻

I admire Regeneron for its commitment to gene-edited therapies. Earlier, bringing in 270 and Philip Gregory, a superstar, to run edited CAR-T programs - now, bringing in Team Mammoth Biosciences - quite a constellation of shining stars themselves!
1+2=7 synergy there.

Super excited to see the exa-cel (Casgevy) #SickleCellDisease results out today in NEJM. These are the data that led to FDA approval Dec23 (during #ash2023 ). Terrific study steering committee. More data to come in younger pts. Now to work on access! Vertex Pharmaceuticals Cancer Center @ CHOP

The first approved gene editing therapy - the clinical trial data!

Bravo, clinical team, bravo Vertex CMC, thank you patients on the trial!

$CoI: I am a paid advisor to Vertex on the exa-cel program.

Friends!
Romans (and those who wish to visit Rome)!
Countrymen (and folks from every other country)!
I come to bring CRISPR to life, not to praise it (it needs no praise)!
Enough Shakespeare - this meeting is PHENOMENAL, come join us.

The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. trib.al/j7ub6yf

This is essential analysis for design of editing strategies to treat Mendelian disease.
Grateful to the authors and the journal for providing it.

An essential step on CRISPR’s journey to becoming a platform for addressing the public health challenge of genetic disease.
👏🏻 Penn Medicine team.
🙏 Megan Molteni for reporting on bona fide impactful advances in our field.

Two important firsts in genome editing
1. Fetal non-human primate base editing delivers to multiple organs
statnews.com/2024/04/22/cri… by Megan Molteni STAT William Peranteau Kiran Musunuru
2. #AI -generated gene editors success in human cells
nytimes.com/2024/04/22/tec… Cade Metz…

What actually happened: AI produced CRISPR-inspired genome editors that have modest on-target activity in human cancer cells.
Impact on actual clinical editing in next 3 years: none.
Long term - I sure hope so.
I think X needs better AI to write these 'Explore' blurbs. :-)

AI's latest: start with natural #CRISPR and engineer new editors.
Clinical efforts have plenty of editors already (issues are cost + regulatory).
Looking ahead I hope this bring us closer to 'perfect editor for a patient's mutation in weeks.'
Cade Metz
nytimes.com/2024/04/22/tec…

SCOTUS will determine whether jailing folks experiencing homelessness is a good idea.
The justices should reread this timeless classic by O'Henry, supreme master of narrating tragedy - and compassion for it - with a Chekhovian light touch.
americanenglish.state.gov/files/ae/resou…

I've been teaching the immortal Sturtevant paper since 1997 (it's on Classical Papers in Genetics) and the first thing I say to my students is the point his grandson makes below.
#yesyoucan - but, I say, you don't HAVE to. Just experience joy from the work.
H/t Keith Robison