Editas Medicine David R. Liu 11/The most advanced Base editing research in IRDs is Beam Therapeutics first ever use made with In Vivo Base editing technology to correct a gene mutation which causes Stargardt disease - an inherited retinal disease. $BEAM has presented its promising pre-clinical data in #ASGCT2023. $XBI

Prime Medicine 4/Prime Medicine has already presented an impressive Preclinical data - during #ASGCT2023, for its leading Ex-Vivo program – PM359 for the treatment of patients with chronic granulomatous disease CGD. Here’s a 🧵which I have written shortly after $PRME presentation👇

Get our update, directly from #ASGCT2023 on how gene and cell therapies continue to advance. Learn how innovative approaches promise targeted delivery of genes and editing-cargo in vivo. Then, take our GenQuiz! genscript.com/crispr-news/ev… #genetherapy #celltherapy #geneengineering

1/A new & promising platform was presented at #ASGCT2023 by Penn Medicine’s Nils_Wellhausen - #EpitopeEditing. By using #BaseEditing #EpitopeEditing aims to shield hematopoietic cells from CD45-directed #immunetherapy without the loss of CD45 protein function. #BioTech #CRISPR $BEAM

Our Research and Manufacturing teams are striving to optimize and expand our novel AAV genetic medicine capabilities for the potential treatment of #heartdisease. Thanks to all who recently represented our work at #ASGCT2023!

1/SparingVision has recently presented promising preclinical data of its second clinical program - SPVN20 - #GeneTherapy aimed to treat late stage #RetinitisPigmentosa patients by restoring the function of dormant #Cone #Photoreceptors #cells. #BioTech #Genomics #ASGCT2023 $NTLA

13/Prime Medicine has presented an impressive updated Preclinical data for its proprietary platform - #PASSIGE - which applies #PrimeEditing to insert gene sized sequences precisely in the genome. Here’s a 🧵that I wrote after $PRME #ASGCT2023 presentation👇

4/Prime Medicine has presented an impressive updated Preclinical data - during #ASGCT2023, for its leading Ex-Vivo #PrimeEditing program – PM359 for the treatment of patients with chronic granulomatous disease #CGD. Here’s a 🧵which I wrote shortly after $PRME presentation👇

An important milestone in #ocular #GeneEditing was Beam Therapeutics’s recent #ASGCT2023 presentation of its preclinical data regarding the first ever use made with In Vivo #BaseEditing #technology aimed to correct a #gene #mutation which causes #Stargardt - an inherited retinal diseases

1/BeamTx has recently presented an interesting preclinical data regarding the first ever use made with In Vivo #BaseEditing #technology to correct a #gene #mutation which causes #Stargardt - an inherited retinal disease. #BioTech #CRISPR #Genomics #GeneEditing #ASGCT2023 $BEAM

1/Beam Therapeutics has recently presented an interesting preclinical data regarding the first ever use made with In Vivo #BaseEditing #technology to correct a #gene #mutation which causes #Stargardt - an inherited retinal disease. #BioTech #CRISPR #Genomics #GeneEditing #ASGCT2023 $BEAM

Thank you ASGCT for the award and for a wonderful conference! 🙏🏾 Can’t wait to be back next year. Check out our abstract (and recently published study) if you are designing any prime editing experiments! #ASGCT2023

We spoke with Carol Miao, PhD, a principal investigator at Seattle Children's Research Institute, about her preclinical research on nonviral delivery methods for gene editing tools in the context of treating #HemophiliaA at #ASGCT2023. Check it out: ow.ly/cSZF50OzY52

We know the burden of #adrenomyeloneuropathy is under-recognized. The goal of our recent #HEOR research has been to shine more light on life with #AMN. Read our findings: bit.ly/3oy7Vdp #realworlddata #raredisease #ALD #AANAM #ispor2023 #ASGCT2023

Missed our oral presentation at the #ASGCT2023? Watch CSO Francesca Barone, MD, PhD, discuss the highlights from our phase 1 clinical trial of CAN-3110 in recurrent high-grade glioma in an interview with CGTLive® from the #ASGCT23 here, bit.ly/3qgGAfZ.

CBE-Ts, ABE8.20, PE5max, HDR 2.0... woundering what would be next? CRISPR-Cas9 Correction via Coding Sequence Replacement to Preserve Endogenous Gene Regulation and Locus Structure - HDR 2.0 researchsquare.com/article/rs-256… #CRISPR #asgct2023

Fyodor Urnov ASGCT I too in the beginning was stunned by the size of #ASGCT2023. But then, I saw the poster from Florence Bourgeois and Magdi Elsallab, MD, PhD where they quantified the probability of success in cell and gene therapy development (considered as FDA approval)…