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SCGE @scge.bsky.social · 06/10/2026

They anticipate their findings will be of interest to academic investigators and industry sponsors who wish to pursue expeditious FDA approvals of therapies for ultra-rare diseases using the plausible mechanism framework. (4/4)

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SCGE @scge.bsky.social · 06/10/2026

as well as the outcome of a formal meeting with the FDA to discuss the use of the platform in an "umbrella-of-umbrellas" clinical trial including subjects with any of the 7 UCDs. (3/4) www.sciencedirect.com/science/arti...

sciencedirect.com

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SCGE @scge.bsky.social · 06/10/2026

Here, SCGE researchers report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders (UCDs) and other liver-centered disorders, (2/4)

sciencedirect.com

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SCGE @scge.bsky.social · 06/10/2026

In February 2026, the US Food and Drug Administration (FDA) published a draft guidance on a new plausible mechanism framework for the development and approval of individualized therapies for genetic conditions. (1/4)

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SCGE @scge.bsky.social · 30/09/2026

Group photo from our Fall 2026 Consortium meeting!

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SCGE @scge.bsky.social · 30/09/2026

Our steering committee update from Dr. Rebecca Ahrens-Nicklas highlighted some exciting wins from our consortium researchers.

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SCGE @scge.bsky.social · 30/09/2026

Starting our Fall 2026 SCGE Consortium meeting with opening remarks from Dr. Amy Adams.

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SCGE @scge.bsky.social · 29/09/2026

SCGE Workshop Final Panel Panelists: PJ Brooks, Anna Kwilas, Rebecca Ahrens-Nicklas, Janet Woodcock, Jack Tyndall, Winston Yan, Jayson Slotnik

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SCGE @scge.bsky.social · 29/09/2026

SCGE Workshop Session 4 Moderator: Shyam Patel Speakers: Winston Yan, David Tischler, Jayson Slotnik Panelists: Shyam Patel, Winston Yan, David Tischler, Jayson Slotnik

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SCGE @scge.bsky.social · 29/09/2026

SCGE Workshop Session 3 Moderator: Deanna Portero Speakers: Jessica Galens, Jack Tyndall, Mitchell Zack, Caroline Larson Panelists: Deanna Portero, Jessica Galens, Jack Tyndall, Mitchell Zack, Caroline Larson

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SCGE @scge.bsky.social · 29/09/2026

SCGE Workshop Session 2 Moderator: Brian Stultz Speakers: Jacob Bitterman, Aimee Magnarelli, Dan O'Connor Panelists: Brian Stultz, Jacob Bitterman, Aimee Magnarelli, Dan O'Connor

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SCGE @scge.bsky.social · 29/09/2026

Session 1 of the SCGE workshop

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SCGE @scge.bsky.social · 29/09/2026

Excited for our SCGE workshop, which is starting soon!

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SCGE @scge.bsky.social · 23/09/2026

In our September SCGE Spotlight, learn more about: - OptiPrime, a machine learning model of prime editing efficiency based on current understanding of prime editing mechanisms - A genome-wide CRISPR screening platform - The SCGE public workshop www.linkedin.com/pulse/scge-s...

linkedin.com

SCGE Spotlight #6

In this edition of the SCGE Spotlight, we’ve got two recent publications, an SCGE public workshop (there’s still time to register!), and a few wins from consortium researchers. Let’s get started.

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Reposted by SCGE

American Society of Gene + Cell Therapy @asgct.bsky.social · 22/09/2026

🔬 Don't wait for the Annual Meeting to share your latest science! Submit your late-breaking abstract for Breakthroughs in Cell + Gene Therapy by Monday, October 12, 2026. Learn more and submit now ➡️ bit.ly/4wHH10l

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SCGE @scge.bsky.social · 22/09/2026

Collectively, these findings suggest that liver base editing over a range of ages may benefit individuals with ZSD and provides a foundation for developing precision gene correction treatments that address the root cause of a wide range of peroxisomal disorders. (4/4)

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SCGE @scge.bsky.social · 22/09/2026

Here, SCGE researchers report the development and application of an adenine base editing strategy to correct an established homozygous Pex1-p.G844D ZSD mouse model that manifests liver pathologies and metabolic dysfunction found in patients. (3/4) www.nature.com/articles/s41...

nature.com

In vivo base editing rescues liver pathophysiology and peroxisome dysfunction in a mouse model of Zellweger spectrum disorder - Nature Biomedical Engineering

In vivo adenine base editing corrects a common Pex1 pathogenic allele in mouse models of Zellweger spectrum disorder, rescuing liver pathology, fatty acid levels, body weight and peroxisome function.

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SCGE @scge.bsky.social · 22/09/2026

The PEX1-p.G843D (c.2528G>A) allele, present in approximately 30% of individuals with ZSD, frequently results in chronic liver disease that can progress to cirrhosis, hepatocellular carcinoma and degraded neurological health. (2/4)

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SCGE @scge.bsky.social · 22/09/2026

Zellweger spectrum disorder (ZSD) is caused by biallelic loss-of-function variants in PEX genes required for peroxisome biogenesis, which is critical for normal cellular metabolism and signalling. (1/4)

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SCGE @scge.bsky.social · 16/09/2026

The SCGE workshop, “Rethinking Clinical Delivery, Regulation, and Payment Models for Individualized Platform-Based Genome Editing Therapies”, is happening in a few short weeks! There are some great speakers and sessions planned for this workshop. Register to attend at scge.mcw.edu/scge-workshop/

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SCGE @scge.bsky.social · 15/09/2026

PET imaging provided evidence of editing in the fetal liver, which was sustained. Appropriate insertion of the promoterless HSV-sr39TK reporter in frame with the PCSK9 gene was confirmed in the fetal liver near term using RNA sequencing, and correctly targeted insertions were observed. (3/4)

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SCGE @scge.bsky.social · 15/09/2026

A noninvasive approach that can identify edited cells in vivo is beneficial for addressing these and related questions. These studies used total-body positron emission tomography (PET) to identify somatic cell gene editing in vivo in fetal rhesus macaques. (2/4)

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SCGE @scge.bsky.social · 09/09/2026

Are you interested in learning more about current gene therapy clinical trials? Check out the SCGE Trial Browser! This great resource is free and available to the public. Get started with the browser at scge.mcw.edu/platform/dat...

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SCGE @scge.bsky.social · 02/09/2026

The SCGE is hosting a public workshop, “Rethinking Clinical Delivery, Regulation, and Payment Models for Individualized Platform-Based Genome Editing Therapies”. Learn more about the workshop and register to attend virtually at scge.mcw.edu/scge-workshop/

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SCGE @scge.bsky.social · 26/08/2026

In this edition of the SCGE Spotlight, we're covering: - A public SCGE workshop - Two publications from SCGE researchers - Some recent news in the gene therapy space www.linkedin.com/pulse/scge-s...

linkedin.com

SCGE Spotlight #5

In this edition of the SCGE Spotlight, we’ve got a public SCGE workshop, new publications, and some gene therapy news you may have missed over the busy summer months. Let’s get started.

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SCGE @scge.bsky.social · 25/08/2026

Calcium shock improves expression of plasmid, ribonucleoprotein, or adeno‐associated viral vectors with minimal toxicity in intact organoids representing diverse lineages. (4/5)

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SCGE @scge.bsky.social · 25/08/2026

Calcium shock works by increasing endocytotic uptake while simultaneously disarming cell‐cell junctions. CaSh‐Pro further incorporates specific molecular targeting agents and amphiphilic peptides for preferential editing of different cell types. (3/5)

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SCGE @scge.bsky.social · 25/08/2026

SCGE researchers demonstrate that an innovative methodology called calcium shock (CaSh) dramatically improves particle delivery into single cells, colonies, and organoids, and enables programmable delivery (CaSh‐Pro) into specific cell types within heterocellular populations. (2/5)

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SCGE @scge.bsky.social · 25/08/2026

Classical intracellular delivery methods such as transfection and transduction are inefficient, particularly with confluent cells and organoids, and lack cell type‐specific programmability. (1/5)

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SCGE @scge.bsky.social · 18/08/2026

This study highlights the potential of μDES-produced RNP-EVs for gene editing as a treatment for progressive nonsyndromic hearing loss in patients. (3/3)

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SCGE @scge.bsky.social · 18/08/2026

Here, SCGE researchers report an efficient nonviral genome editor delivery approach using extracellular vesicles (EVs) carrying single-guide RNA (sgRNA): CRISPR-Cas9 ribonucleoprotein (RNP) complexes for in vivo gene therapy. (2/3) www.science.org/doi/10.1126/...

science.org

Extracellular vesicle–mediated gene editing for the treatment of nonsyndromic progressive hearing loss in adult mice

High-throughput loading of sgRNA:Cas9 RNPs targeting Myo7aSh1 into extracellular vesicles prevents progressive hearing loss in an adult mouse model.

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SCGE @scge.bsky.social · 12/08/2026

Shyam Patel is the Associate VP of Patient Access at California Institute for Regenerative Medicine (CIRM). Kevin A. Strauss, MD is the Founder and Chief Medical Officer of Plowshare Therapies LLC. Manar Zaghlula, Ph.D. is the Public Impact Director for the Innovative Genomics Institute (IGI).

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SCGE @scge.bsky.social · 12/08/2026

In this video of the SCGE Meet the Expert Webinar Series, panelists discuss different economic and reimbursement perspectives of gene editing therapies. Watch the full video at: www.youtube.com/watch?v=Oo41...

youtube.com

Meet the Expert: Shyam Patel, Kevin Strauss, Manar Zaghlula

YouTube video by SCGE Outreach

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SCGE @scge.bsky.social · 11/08/2026

This modular and naturally derived, unmodified collagen microbead technology unites tunability, throughput, and functional scalability, establishing a versatile platform for tissue modeling, drug testing, and therapeutic EV biomanufacturing. (4/4)

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SCGE @scge.bsky.social · 11/08/2026

SCGE researchers developed a pressure-tunable microfluidic platform that enables the rapid, high-throughput generation of collagen microbeads with controlled size, fibrillar architecture, and mechanical properties for versatile downstream biofabrication. (3/4) pmc.ncbi.nlm.nih.gov/articles/PMC...

pmc.ncbi.nlm.nih.gov

High-throughput Generation of Collagen Microbeads for Extracellular Vesicle Production and Therapeutic Delivery

Collagen type I, a key component of the extracellular matrix, underpins cellular organization and regeneration but remains challenging to process into scalable, reproducible, and biologically faithful...

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SCGE @scge.bsky.social · 11/08/2026

Although several recent platforms have achieved high-throughput collagen microgel fabrication, most rely on chemical modification or rigid process parameters that compromise native collagen structure or limit biological versatility. A scalable yet biologically unmodified system remains lacking.(2/4)

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SCGE @scge.bsky.social · 05/08/2026

Jessica Chaikof and Rachel Chaikof provide patient perspectives, while Stacey Ashlund offers the caregiver perspective for this webinar.

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SCGE @scge.bsky.social · 05/08/2026

In our latest video in the SCGE "Meet the Expert" series, panelists share their experiences with rare disease and provide patient and caregiver perspectives on gene therapy for hearing and vision loss. Watch the full video at www.youtube.com/watch?v=z_WQ...

youtube.com

Meet the Expert: Jessica Chaikof, Rachel Chaikof, and Stacey Ashlund

YouTube video by SCGE Outreach

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SCGE @scge.bsky.social · 04/08/2026

These findings highlight elements critical for regulating TnpB endonuclease activity and demonstrate latent activity accessible through mutation. (4/4) www.nature.com/articles/s41...

nature.com

Engineered TnpB genome editors for plants and human cells identified by ribonucleoprotein mutational scanning - Nature Biotechnology

TnpB endonucleases are engineered for improved genome editing.

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SCGE @scge.bsky.social · 04/08/2026

Leveraging the protein’s mutational landscape, they constructed a combinatorial library of activating mutations, from which they identified two enhanced TnpB variants. These variants increased editing in human cells, Nicotania benthamiana, pepper and rice. (3/4)

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SCGE @scge.bsky.social · 04/08/2026

Here, researchers mapped comprehensive sequence–function landscapes of a TnpB ribonucleoprotein using deep mutational scanning and they discovered activating mutations in both the RNA and the protein. (2/4)

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SCGE @scge.bsky.social · 04/08/2026

TnpB is a diverse family of RNA-guided endonucleases associated with prokaryotic transposons. Because of their small size and putative evolutionary relationship to CRISPR–Cas12, TnpB enzymes hold great potential for genome editing. However, most TnpBs lack robust gene-editing activity. (1/4)

nature.com

Engineered TnpB genome editors for plants and human cells identified by ribonucleoprotein mutational scanning - Nature Biotechnology

TnpB endonucleases are engineered for improved genome editing.

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SCGE @scge.bsky.social · 29/07/2026

In this edition of the SCGE Spotlight, we're covering: - Prime editing using LNPs - Gene editing in human kidney organoids - A toolkit for IND applications - Interviews with SCGE researchers www.linkedin.com/pulse/scge-s...

linkedin.com

SCGE Spotlight #4

In this edition of the SCGE Spotlight, we’re sharing new publications, a collection of resources to help fill out an IND submission, and more SCGE researcher interviews. Let’s get started.

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SCGE @scge.bsky.social · 28/07/2026

They demonstrate this system with several activation and repression domains, in cultured primary immune cells, and to differentiate induced pluripotent stem cells. These tools create possibilities for highly multiplexed control of gene expression in many biological systems. (3/3)

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SCGE @scge.bsky.social · 28/07/2026

However, assessment of interactions between those genes or elements remains limited. To enable efficient highly-multiplexed control of regulatory element activity, researchers combine a dHyperLbCas12a with RNA Polymerase II expression of long CRISPR RNA arrays. (2/3)

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