An international consortium led by Dr. Stephan Grupp published final results of a key clinical trial on gene therapy CASGEVY for sickle cell disease treatment. The findings, published in NEJM, led to FDA approval of CASGEVY™ in December 2023.

More: ms.spr.ly/6011YJEX3.

A Welcome to the Era of Genome Editing today NEJM
Overview George Q Daley
nejm.org/doi/full/10.10…
Casgevy for Sickle-Cell Disease
nejm.org/doi/full/10.10…
Casgevy for Beta-Thalassemia
nejm.org/doi/full/10.10…

The treatment is called Casgevy and is targeted to help people with sickle cell 12+
casgevy.com/sickle-cell-di…

$verv Despite Casgevy approval, application left many questions unanswered. EMA advises to focus on unmet medical needs.

Regulatory panel at #crisprmed24

Recently FDA-approved gene therapy for Sickle Cell shows really promising results. Over 90% of clinical trial participants reported having felt no pain crises for at least a year. But a single round of treatment can cost around 2 million dollars…
goodrx.com/conditions/sic…

#Casgevy why is the amount of fetal hemoglobin drastically lower in sickle cell vs beta-thalassemia population?

Are gamma-globin/sickle cell globin heterodimers not formed/measured?

Editing levels are very similar. $CRSP $VRTX

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

Last week on the @misciwriters blog, Madison (@fitz_gerM) talks about the first FDA approved CRISPR-based gene therapy. Read along as she talks about how the drug, Casgevy, works and what it means for the for the future of gene therapies. buff.ly/3Qn1iFb

Hi, there! Science talk.

Here are some recent breakthroughs in medical research:

1. CRISPR Gene-Editing Therapy: The U.S. FDA approved the world’s first CRISPR/Cas9 gene-editing therapy, Casgevy, which targets sickle cell disease by helping patients produce healthy hemoglobin.

But CRISPR gene therapy already did that😏

In case any AS human being is thinking of getting married to another AS because of this tweet,

Just know that, Casgevy therapy costs over 2.2 million dollars, and it's not done in Nigeria 🫣🫣

An understated milestone is that Casgevy is not only the first CRISPR therapeutic, but also the first enhancer-targeting therapeutic. The remaining ~500,000+ predicted enhancers in the human genome await our exploration of their therapeutic potential.

Jasbinder (Jessie) Chimni (My Opinions only) When they are laughing about making money off CASGEVY, I could only think of $alny they got 5 drugs approved and they lose money hand over fist.

.Children's Hospital researchers demonstrated improved quality of life for #sicklecelldisease patients with recurrent vaso-occlusive crises. The findings provide the complete details of the trial leading to U.S. FDA approval of #Casgevy in Dec. 2023. bit.ly/3Uw3SLA

Joe JoJo Somerville Incorrect. Zero chemo is needed!

For SCD Casgevy from $CRSP needs chemo but none of these in vivo therapies need chemo.

Stories by Gwendolyn Wu and me on Vertex and CRISPR Therapeutics' sickle cell treatment Casgevy were recognized for Best Series:

biopharmadive.com/news/crispr-bi…

biopharmadive.com/news/sickle-ce…

biopharmadive.com/news/crispr-ca…

LaborIsMoney Casgevy became “easy” once they learned about fetal hemoglobin’s relationship with SCD patients.

HAE and ATTR are just gene knockouts which help reduce harmful proteins that were found to be the main reason for those diseases.

Where things get REALLY exciting is with $BEAM-302

LaborIsMoney Casgevy by $CRSP (also $VRTX now owns 60% of it through deals with CRSP).

It is essentially curative for SCD.

$NTLA also has a cure for HAE going to phase 3 in Q3 and another ATTR program already in phase 3. And both of these are in vivo. Casgevy is ex vivo.

Did you know that approximately 100,000 people in the U.S. suffer from sickle cell disease (SCD)? This genetic disorder primarily affects African Americans and Hispanic Americans, causing severe pain and organ damage... Read The Full Article 👇👇
thesciencegang.com/casgevy-first-…

Casgevy is the first CRISPR-based medicine to be approved for use in the United States #CheckRare #BetaThalassemia #Casgevy
checkrare.com/fda-approves-c…

Fyodor Urnov Prime Medicine How big is the edit they are making here? Prime editing offers a lot of possibilities but from what I've seen only the real small edits can be done with a bit of precision??
And ignorant question maybe, but does this treatment also require conditioning like Casgevy?