SCGE
@scge.bsky.social
The Somatic Cell Genome Editing (SCGE) Consortium is an NIH Common Fund program that aims to develop safe and effective methods to perform gene editing to treat genetic diseases in somatic cells. Reposts/likes do not equal endorsements.
scge.mcw.edu
scge.mcw.edu
Kiran Musunuru and Sonia Vallabh, panelists on "Concluding Panel: The Future of Targeted In Vivo Gene Editing" (3/3)
November 21, 2025 at 3:56 PM
Kiran Musunuru and Sonia Vallabh, panelists on "Concluding Panel: The Future of Targeted In Vivo Gene Editing" (3/3)
Rebecca Ahrens-Nicklas, "Personalized liver-directed corrective gene editing" (2/3)
November 21, 2025 at 3:56 PM
Rebecca Ahrens-Nicklas, "Personalized liver-directed corrective gene editing" (2/3)
Are you at the @asgct.bsky.social Breakthroughs in Targeted In Vivo Gene Editing event? Hear from these SCGE researchers on Friday:
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
November 21, 2025 at 3:56 PM
Are you at the @asgct.bsky.social Breakthroughs in Targeted In Vivo Gene Editing event? Hear from these SCGE researchers on Friday:
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Sonia Vallabh, "AAV-mediated epigenetic editing for prion disease" (1/3)
Krishanu Saha, "Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency" (Poster)
@sahakris.bsky.social (4/4)
@sahakris.bsky.social (4/4)
November 20, 2025 at 1:51 PM
Krishanu Saha, "Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency" (Poster)
@sahakris.bsky.social (4/4)
@sahakris.bsky.social (4/4)
Yong-hui Jiang, "Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome" (3/4)
November 20, 2025 at 1:51 PM
Yong-hui Jiang, "Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome" (3/4)
Kiran Musunuru, "LNP-mediated editing for treatment of cardiometabolic disease"
@kiranmusunuru.bsky.social (2/4)
@kiranmusunuru.bsky.social (2/4)
November 20, 2025 at 1:51 PM
Kiran Musunuru, "LNP-mediated editing for treatment of cardiometabolic disease"
@kiranmusunuru.bsky.social (2/4)
@kiranmusunuru.bsky.social (2/4)
Are you attending American Society of Gene & Cell Therapy's Breakthroughs in Targeted In Vivo Gene Editing event? If so, check out presentations from these SCGE researchers on Thursday:
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
November 20, 2025 at 1:51 PM
Are you attending American Society of Gene & Cell Therapy's Breakthroughs in Targeted In Vivo Gene Editing event? If so, check out presentations from these SCGE researchers on Thursday:
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
Bill Peranteau, "In utero editing of muscles with LNPs" (1/4)
@asgct.bsky.social
Reposted by SCGE
A team at the Broad Institute co-led by Sarah Pierce, Steven Erwood, and David Liu has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple unrelated genetic diseases.
Single prime editing system could potentially treat multiple genetic diseases
Researchers have developed a genome-editing strategy that targets a common cause of roughly 30 percent of rare diseases and could vastly improve access to gene-editing treatments for patients.
www.broadinstitute.org
November 19, 2025 at 6:40 PM
A team at the Broad Institute co-led by Sarah Pierce, Steven Erwood, and David Liu has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple unrelated genetic diseases.
Reposted by SCGE
New online! Harnessing artificial intelligence to advance CRISPR-based genome editing technologies
Harnessing artificial intelligence to advance CRISPR-based genome editing technologies
Nature Reviews Genetics, Published online: 18 November 2025; doi:10.1038/s41576-025-00907-1CRISPR-based genome editing has revolutionized biotechnology, enabling precise DNA modifications for research and therapy. The authors review how artificial intelligence, including deep learning, is advancing genome editing by improving guide RNA design, editor protein engineering, novel effector discovery and predicting editing outcomes.
www.nature.com
November 18, 2025 at 8:07 PM
New online! Harnessing artificial intelligence to advance CRISPR-based genome editing technologies
Reposted by SCGE
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
November 13, 2025 at 4:07 PM
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
Reposted by SCGE
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
November 12, 2025 at 7:06 PM
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
Richard Lewis, PhD is the Principal Regulatory Device & Biologics Expert at Hyman, Phelps & McNamara, P.C.
More info about the SCGE Meet the Expert series can be found at scge.mcw.edu/meet-the-exp...
#RegulatoryGuidance #RareDisease #Preclinical #Biotech #BiomedicalResearch
More info about the SCGE Meet the Expert series can be found at scge.mcw.edu/meet-the-exp...
#RegulatoryGuidance #RareDisease #Preclinical #Biotech #BiomedicalResearch
November 12, 2025 at 2:36 PM
Richard Lewis, PhD is the Principal Regulatory Device & Biologics Expert at Hyman, Phelps & McNamara, P.C.
More info about the SCGE Meet the Expert series can be found at scge.mcw.edu/meet-the-exp...
#RegulatoryGuidance #RareDisease #Preclinical #Biotech #BiomedicalResearch
More info about the SCGE Meet the Expert series can be found at scge.mcw.edu/meet-the-exp...
#RegulatoryGuidance #RareDisease #Preclinical #Biotech #BiomedicalResearch
James Valentine, JD, MHS is a Director at Hyman, Phelps & McNamara, P.C. where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters relating to new drug and biologic development and approval.
November 12, 2025 at 2:36 PM
James Valentine, JD, MHS is a Director at Hyman, Phelps & McNamara, P.C. where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters relating to new drug and biologic development and approval.
In this video of the SCGE Meet the Expert series, panelists use their legal and U.S. Food and Drug Administration (FDA) expertise to provide practical guidance to rare disease researchers in preclinical stages of development.
Watch the full video at www.youtube.com/watch?v=0U1x...
Watch the full video at www.youtube.com/watch?v=0U1x...
Meet the Expert: James Valentine and Richard Lewis
YouTube video by SCGE Outreach
www.youtube.com
November 12, 2025 at 2:36 PM
In this video of the SCGE Meet the Expert series, panelists use their legal and U.S. Food and Drug Administration (FDA) expertise to provide practical guidance to rare disease researchers in preclinical stages of development.
Watch the full video at www.youtube.com/watch?v=0U1x...
Watch the full video at www.youtube.com/watch?v=0U1x...
With enhanced targeting versatility, Flex-Cas12a unlocks access to previously inaccessible genomic loci, providing new opportunities for both therapeutic and agricultural genome engineering. (3/3)
#Research #CRISPR #GeneEditing
#Research #CRISPR #GeneEditing
November 11, 2025 at 3:20 PM
With enhanced targeting versatility, Flex-Cas12a unlocks access to previously inaccessible genomic loci, providing new opportunities for both therapeutic and agricultural genome engineering. (3/3)
#Research #CRISPR #GeneEditing
#Research #CRISPR #GeneEditing
To mitigate this constraint, SCGE researchers used a bacterial-based directed evolution assay combined with rational engineering to identify variants of Lachnospiraceae bacterium Cas12a with expanded PAM recognition. (2/3)
November 11, 2025 at 3:20 PM
To mitigate this constraint, SCGE researchers used a bacterial-based directed evolution assay combined with rational engineering to identify variants of Lachnospiraceae bacterium Cas12a with expanded PAM recognition. (2/3)
CRISPR-Cas12a enzymes' dependence on a 5'-TTTV-3' protospacer adjacent motif (PAM) next to DNA target sequences restricts Cas12a's gene targeting capability to only ∼1% of a typical genome. (1/3)
academic.oup.com/nar/article/...
academic.oup.com/nar/article/...
November 11, 2025 at 3:20 PM
CRISPR-Cas12a enzymes' dependence on a 5'-TTTV-3' protospacer adjacent motif (PAM) next to DNA target sequences restricts Cas12a's gene targeting capability to only ∼1% of a typical genome. (1/3)
academic.oup.com/nar/article/...
academic.oup.com/nar/article/...
Reposted by SCGE
Coming up *tomorrow* November 6th — join us to hear about the latest in #CRISPR for human health from IGI's Brad Ringeisen, Fyodor Urnov, and Carlotta Ronda as part of @ucberkeleyofficial.bsky.social's Basic Science Lights the Way lecture series! Register here: https://basicscience.berkeley.edu/
November 5, 2025 at 5:00 PM
Coming up *tomorrow* November 6th — join us to hear about the latest in #CRISPR for human health from IGI's Brad Ringeisen, Fyodor Urnov, and Carlotta Ronda as part of @ucberkeleyofficial.bsky.social's Basic Science Lights the Way lecture series! Register here: https://basicscience.berkeley.edu/
Our hope is that these documents can be useful to investigators that are developing gene editing therapies for rare diseases.
#Regulatory #GeneTherapy #RareDisease #BiotechResearch #BiomedicalResearch
#Regulatory #GeneTherapy #RareDisease #BiotechResearch #BiomedicalResearch
November 5, 2025 at 2:36 PM
Our hope is that these documents can be useful to investigators that are developing gene editing therapies for rare diseases.
#Regulatory #GeneTherapy #RareDisease #BiotechResearch #BiomedicalResearch
#Regulatory #GeneTherapy #RareDisease #BiotechResearch #BiomedicalResearch
The goal of the SCGE program is to accelerate the translation of genome editing therapies into the clinic. One of the ways we are working to achieve this goal is by publicly sharing successful strategies for starting clinical trials.
November 5, 2025 at 2:36 PM
The goal of the SCGE program is to accelerate the translation of genome editing therapies into the clinic. One of the ways we are working to achieve this goal is by publicly sharing successful strategies for starting clinical trials.
The SCGE platform now has the first sets of researchers' regulatory documents available to the public. These documents are from regulatory interactions that SCGE researchers have had with the U.S. Food and Drug Administration (FDA).
Access these documents at: scge.mcw.edu/platform/pub...
Access these documents at: scge.mcw.edu/platform/pub...
November 5, 2025 at 2:36 PM
The SCGE platform now has the first sets of researchers' regulatory documents available to the public. These documents are from regulatory interactions that SCGE researchers have had with the U.S. Food and Drug Administration (FDA).
Access these documents at: scge.mcw.edu/platform/pub...
Access these documents at: scge.mcw.edu/platform/pub...
Reposted by SCGE
📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts!
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
Breakthroughs in Targeted In Vivo Gene Editing
Dedicated to cutting-edge solutions for targeted delivery, this event is your chance to engage with pioneering research, overcome tissue-specific…
www.asgct.org
November 3, 2025 at 11:15 PM
📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts!
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv
The SCGE site is undergoing scheduled maintenance on 11/3 from approximately 11:00am-2:00pm CT. We are sorry for any inconvenience.
November 3, 2025 at 5:10 PM
The SCGE site is undergoing scheduled maintenance on 11/3 from approximately 11:00am-2:00pm CT. We are sorry for any inconvenience.
Reposted by SCGE
Read more about today's paper (tinyurl.com/23c5vzd9) from @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social that discusses regulatory innovation in the pathway toward interventional genetics!
Personalized gene editing helped one baby: can it be rolled out widely?
In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the approach.
www.nature.com
October 31, 2025 at 6:47 PM
Read more about today's paper (tinyurl.com/23c5vzd9) from @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social that discusses regulatory innovation in the pathway toward interventional genetics!
Reposted by SCGE
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
How to create personalized gene editing platforms: Next steps toward interventional genetics
How do we go from a single individual receiving a personalized gene-editing therapy
to a future of “interventional genetics” in which such therapies are the standard
of care? First and foremost: regul...
www.cell.com
October 31, 2025 at 1:13 PM
In this article, SCGE researchers Rebecca Ahrens-Nicklas and Kiran Musunuru discuss the next steps towards creating personalized gene editing platforms.
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social
Read the full paper at: www.cell.com/ajhg/fulltex...
@ahrensnicklas.bsky.social @kiranmusunuru.bsky.social