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
In this video of the SCGE Meet the Expert series, Andy Holt and Jacob Smith discuss AAV manufacturing and considerations for IND-enabling studies. (1/3)
Watch the full webinar at www.youtube.com/watch?v=fyCm...
Watch the full webinar at www.youtube.com/watch?v=fyCm...
Meet the Expert: Andy Holt and Jacob Smith
YouTube video by SCGE Outreach
www.youtube.com
January 21, 2026 at 4:27 PM
In this video of the SCGE Meet the Expert series, Andy Holt and Jacob Smith discuss AAV manufacturing and considerations for IND-enabling studies. (1/3)
Watch the full webinar at www.youtube.com/watch?v=fyCm...
Watch the full webinar at www.youtube.com/watch?v=fyCm...
The interleukin (IL)-1 pathway is a key mediator of inflammation and innate immune responses. Its dysregulation contributes to rheumatoid arthritis and autoinflammatory diseases. (1/4)
January 20, 2026 at 6:13 PM
The interleukin (IL)-1 pathway is a key mediator of inflammation and innate immune responses. Its dysregulation contributes to rheumatoid arthritis and autoinflammatory diseases. (1/4)
Reposted by SCGE
⏱️ It's time to finalize your #ASGCT2026 abstract. Hit that submit button today! https://annualmeeting.asgct.org/abstracts/submission-information
January 20, 2026 at 6:10 PM
⏱️ It's time to finalize your #ASGCT2026 abstract. Hit that submit button today! https://annualmeeting.asgct.org/abstracts/submission-information
Reposted by SCGE
A new review from the Doudna Lab lab on tissue-targeted in vivo delivery of gene editors: https://www.nature.com/articles/s41587-025-02945-w
#CRISPR #delivery
#CRISPR #delivery
January 13, 2026 at 8:20 PM
A new review from the Doudna Lab lab on tissue-targeted in vivo delivery of gene editors: https://www.nature.com/articles/s41587-025-02945-w
#CRISPR #delivery
#CRISPR #delivery
Gene Therapy News: Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia
investors.solidbio.com/news-release...
investors.solidbio.com/news-release...
Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia | Solid Biosciences Inc.
The Investor Relations website contains information about Solid Biosciences Inc.'s business for stockholders, potential investors, and financial analysts.
investors.solidbio.com
January 15, 2026 at 2:33 PM
Gene Therapy News: Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia
investors.solidbio.com/news-release...
investors.solidbio.com/news-release...
For Phase 2, the goal of the SCGE program is to accelerate the development of genome editing therapies into clinic.
There are many rare diseases being studied in the current phase of the program. Learn more about the different diseases being studied here: scge.mcw.edu/rare-diseases/
#RareDisease
There are many rare diseases being studied in the current phase of the program. Learn more about the different diseases being studied here: scge.mcw.edu/rare-diseases/
#RareDisease
January 14, 2026 at 3:10 PM
For Phase 2, the goal of the SCGE program is to accelerate the development of genome editing therapies into clinic.
There are many rare diseases being studied in the current phase of the program. Learn more about the different diseases being studied here: scge.mcw.edu/rare-diseases/
#RareDisease
There are many rare diseases being studied in the current phase of the program. Learn more about the different diseases being studied here: scge.mcw.edu/rare-diseases/
#RareDisease
Researchers from the SCGE Phase 1 Biological Effects Initiative published this review on monitoring biological effects of somatic cell genome editing. Read the publication at www.nature.com/articles/s41...
Thanks to everyone in the group who contributed!
#BiologicalEffects #GeneTherapy
Thanks to everyone in the group who contributed!
#BiologicalEffects #GeneTherapy
Client Challenge
www.nature.com
January 13, 2026 at 6:43 PM
Researchers from the SCGE Phase 1 Biological Effects Initiative published this review on monitoring biological effects of somatic cell genome editing. Read the publication at www.nature.com/articles/s41...
Thanks to everyone in the group who contributed!
#BiologicalEffects #GeneTherapy
Thanks to everyone in the group who contributed!
#BiologicalEffects #GeneTherapy
Cas9 is a programmable nuclease that has furnished transformative technologies, including base editors and transcription modulators, but several applications of these technologies, including therapeutics, mandatorily require precision control of their half-life. (1/5)
January 13, 2026 at 2:41 PM
Cas9 is a programmable nuclease that has furnished transformative technologies, including base editors and transcription modulators, but several applications of these technologies, including therapeutics, mandatorily require precision control of their half-life. (1/5)
Reposted by SCGE
Happy to report that our paper on "Imaging CRISPR-edited CAR-T cell therapies with optical and PET reporters" has been published in Theranostics (IF 13.3) t.co/qTOWjGqssf. Huge thanks to co-first author Rafael Sánchez-Pupo, our mentor John Ronald and the rest of the team to get this over the line!
January 12, 2026 at 11:28 PM
Happy to report that our paper on "Imaging CRISPR-edited CAR-T cell therapies with optical and PET reporters" has been published in Theranostics (IF 13.3) t.co/qTOWjGqssf. Huge thanks to co-first author Rafael Sánchez-Pupo, our mentor John Ronald and the rest of the team to get this over the line!
Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for Alpha-1 Antitrypsin Deficiency (AATD)
checkorphan.org/news/tessera...
checkorphan.org/news/tessera...
Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for Alpha-1 Antitrypsin Deficiency (AATD) – CheckOrphan
checkorphan.org
January 12, 2026 at 3:31 PM
Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for Alpha-1 Antitrypsin Deficiency (AATD)
checkorphan.org/news/tessera...
checkorphan.org/news/tessera...
ICYMI: FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation
www.fda.gov/news-events/...
www.fda.gov/news-events/...
FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation
The U.S. Food and Drug Administration today announced it is sharing information about the agency’s flexible approach to overseeing chemistry, manufacturing and control (CMC) requirements for cell and ...
www.fda.gov
January 12, 2026 at 2:20 PM
ICYMI: FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation
www.fda.gov/news-events/...
www.fda.gov/news-events/...
Reposted by SCGE
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases. www.wired.com/story/crispr...
Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases.
www.wired.com
January 9, 2026 at 11:06 AM
Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases. www.wired.com/story/crispr...
Meet the Expert is a webinar series presented by the Somatic Cell Genome Editing (SCGE) Consortium. This webinar series aims to connect researchers within the consortium and the greater scientific community with experts in the gene therapy space. (1/2)
#Webinar #GeneTherapy #RareDisease #Research
#Webinar #GeneTherapy #RareDisease #Research
January 7, 2026 at 3:47 PM
Meet the Expert is a webinar series presented by the Somatic Cell Genome Editing (SCGE) Consortium. This webinar series aims to connect researchers within the consortium and the greater scientific community with experts in the gene therapy space. (1/2)
#Webinar #GeneTherapy #RareDisease #Research
#Webinar #GeneTherapy #RareDisease #Research
Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is sequestered in hepatocytes, causing lung emphysema due to insufficient AAT protein to inhibit neutrophil elastase in the lung. (1/3)
A compact base editor rescues AATD-associated liver and lung disease in mouse models
Xue and colleagues developed a novel eNme2 base editor that significantly improves
both alpha-1 antitrypsin deficiency associated liver and lung disease in clinically
relevant mouse models.
www.cell.com
January 6, 2026 at 5:07 PM
Alpha-1 antitrypsin deficiency (AATD) is commonly caused by a G-to-A mutation in the SERPINA1 gene (the PiZ mutation). The mutant PiZ AAT protein is sequestered in hepatocytes, causing lung emphysema due to insufficient AAT protein to inhibit neutrophil elastase in the lung. (1/3)
Reposted by SCGE
CHANGE-seq-BE finds off-target changes in the genome from base editors #NBTintheNews via @stjuderesearch.bsky.social www.stjude.org/media-resour...
CHANGE-seq-BE finds off-target changes in the genome from base editors
Find how Circularization for High-throughput Analysis of Nuclease Genome-wide Effects by Sequencing Base Editors (CHANGE-seq-BE) improves finding off-targets.
www.stjude.org
January 5, 2026 at 7:56 PM
CHANGE-seq-BE finds off-target changes in the genome from base editors #NBTintheNews via @stjuderesearch.bsky.social www.stjude.org/media-resour...
Reposted by SCGE
Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE - @stjuderesearch.bsky.social go.nature.com/4qB6qp8
Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE - Nature Biotechnology
Base editor off-target effects are profiled with high precision in a tailored approach.
go.nature.com
January 2, 2026 at 7:12 PM
Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE - @stjuderesearch.bsky.social go.nature.com/4qB6qp8
SCGE researchers developed a mouse model called tdTomato in vivo genome-editing reporter (TIGER) to serve as a reporter with single-cell resolution to evaluate and compare genome editing delivery methods across the mouse body. (1/2)🧬🧪
www.pnas.org/doi/10.1073/...
www.pnas.org/doi/10.1073/...
PNAS
Proceedings of the National Academy of Sciences (PNAS), a peer reviewed journal of the National Academy of Sciences (NAS) - an authoritative source of high-impact, original research that broadly spans...
www.pnas.org
December 30, 2025 at 5:21 PM
SCGE researchers developed a mouse model called tdTomato in vivo genome-editing reporter (TIGER) to serve as a reporter with single-cell resolution to evaluate and compare genome editing delivery methods across the mouse body. (1/2)🧬🧪
www.pnas.org/doi/10.1073/...
www.pnas.org/doi/10.1073/...
Wishing everyone a happy and safe holiday season!
December 22, 2025 at 3:27 PM
Wishing everyone a happy and safe holiday season!
The SCGE Consortium has a goal to disseminate documents generated through regulatory interactions between the U.S. FDA and consortium projects to help rare disease communities, providers, and researchers.
These documents are free and available to the public at scge.mcw.edu/platform/pub...
These documents are free and available to the public at scge.mcw.edu/platform/pub...
December 17, 2025 at 2:58 PM
The SCGE Consortium has a goal to disseminate documents generated through regulatory interactions between the U.S. FDA and consortium projects to help rare disease communities, providers, and researchers.
These documents are free and available to the public at scge.mcw.edu/platform/pub...
These documents are free and available to the public at scge.mcw.edu/platform/pub...
ICYMI: SCGE researcher Dr. Sonia Vallabh recently presented at the NIH Director's Wednesday Afternoon Lecture Series (WALS). The recording is now available at videocast.nih.gov/watch=57108
#PatientScientist #RareDisease 🧪🧬
#PatientScientist #RareDisease 🧪🧬
December 17, 2025 at 2:47 PM
ICYMI: SCGE researcher Dr. Sonia Vallabh recently presented at the NIH Director's Wednesday Afternoon Lecture Series (WALS). The recording is now available at videocast.nih.gov/watch=57108
#PatientScientist #RareDisease 🧪🧬
#PatientScientist #RareDisease 🧪🧬
To meet the brain’s moment-to-moment energy demand, neural activation rapidly increases local blood flow. This process, known as neurovascular coupling, involves rapid, coordinated vasodilation of the brain’s arterial network. (1/4)🧵🧬🧪
December 16, 2025 at 3:59 PM
To meet the brain’s moment-to-moment energy demand, neural activation rapidly increases local blood flow. This process, known as neurovascular coupling, involves rapid, coordinated vasodilation of the brain’s arterial network. (1/4)🧵🧬🧪
Reposted by SCGE
Start the new year strong with our new Foundations of CGT online course – self-paced, interactive, and free! Check out the ASGCT Learning Center: https://www.asgct.org/education-career-resources/asgct-learning-center
December 15, 2025 at 5:56 PM
Start the new year strong with our new Foundations of CGT online course – self-paced, interactive, and free! Check out the ASGCT Learning Center: https://www.asgct.org/education-career-resources/asgct-learning-center
Reposted by SCGE
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity.
www.statnews.com/2025/12/10/n...
www.statnews.com/2025/12/10/n...
In a first, a nonprofit wins FDA approval for rare disease gene therapy
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity.
www.statnews.com
December 10, 2025 at 6:23 PM
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug company, but an Italian charity.
www.statnews.com/2025/12/10/n...
www.statnews.com/2025/12/10/n...
Reposted by SCGE
A fired public-health official, a mosquito breeder and a baby with a smile seen around the world. These are just a few of the remarkable people chosen for Nature’s 10 2025
Check out the full list: go.nature.com/4rIQH8H
Check out the full list: go.nature.com/4rIQH8H
December 8, 2025 at 5:37 PM
A fired public-health official, a mosquito breeder and a baby with a smile seen around the world. These are just a few of the remarkable people chosen for Nature’s 10 2025
Check out the full list: go.nature.com/4rIQH8H
Check out the full list: go.nature.com/4rIQH8H
Do you want to learn more about current gene therapy clinical trials? Our SCGE Gene Therapy Clinical Trial Browser is a free tool available to the public.
Get started exploring today at scge.mcw.edu/platform/dat...
#RareDisease #ClinicalTrials #GeneTherapy 🧪🧬💻
Get started exploring today at scge.mcw.edu/platform/dat...
#RareDisease #ClinicalTrials #GeneTherapy 🧪🧬💻
December 10, 2025 at 7:25 PM
Do you want to learn more about current gene therapy clinical trials? Our SCGE Gene Therapy Clinical Trial Browser is a free tool available to the public.
Get started exploring today at scge.mcw.edu/platform/dat...
#RareDisease #ClinicalTrials #GeneTherapy 🧪🧬💻
Get started exploring today at scge.mcw.edu/platform/dat...
#RareDisease #ClinicalTrials #GeneTherapy 🧪🧬💻