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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 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 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

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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Session 1 of the SCGE workshop

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SCGE @scge.bsky.social · 23/09/2026

Excited for our SCGE workshop, which is starting soon!

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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

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/

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/

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

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 · 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...

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

SCGE @scge.bsky.social · 05/08/2026

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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...

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

SCGE @scge.bsky.social · 04/08/2026

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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

Interactions between genes or cis-regulatory elements (CREs) underlie many biological processes. High-throughput CRISPR screens have allowed researchers to assess the impact of activation or repression of gene and regulatory elements on many phenotypes. (1/3) www.nature.com/articles/s41...

nature.com

dHyperCas12a enables multiplexed CRISPRi screens - Nature Communications

CRISPR/Cas9 screens have identified genetic contributions to many phenotypes. However, studying combinations of genes or regulatory elements remains challenging. Here, the authors use CRISPR/Cas12a to...

SCGE @scge.bsky.social · 22/07/2026

In this interview, Marsha Michie, PhD and Megan Allyse, PhD, discuss a range of ethics topics related to gene therapy, from adapting patient educational materials to the ethical considerations around somatic cell in utero gene editing therapies. Watch the interview: www.youtube.com/watch?v=r_vt...

Meet the Researcher: Marsha Michie and Megan Allyse

SCGE @scge.bsky.social · 21/07/2026

Extracellular vesicles (EVs) have emerged as promising nanocarriers for cancer therapy given their high biocompatibility and low immunogenicity. However, their clinical utility remains limited by challenges such as off-target, premature drug release, and rapid clearance. (1/5)

pmc.ncbi.nlm.nih.gov

Engineering stimuli-responsive extracellular vesicles for enhanced anticancer therapeutics

Extracellular vesicles (EVs), so called nano-sized vesicles shedding out from cells, have emerged as promising nanocarriers for cancer therapy given their high biocompatibility and low immunogenicity....

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SCGE @scge.bsky.social · 15/07/2026

In this "Meet the Researcher” interview, hear from Christopher Baehr, PhD, as he discusses innovative approaches for improving patient access to gene therapies and shares his ideas and hopes for the gene therapy field in the next decade. @chrisbaehr.bsky.social www.youtube.com/watch?v=yS6l...

Meet the Researcher: Chris Baehr

SCGE @scge.bsky.social · 14/07/2026

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Synthetic biology offers control over cellular and tissue functions. As it moves beyond microbes into humans, synthetic biology enables precise control over gene expression, cell fate, and tissue organization across heart, lung, blood, and sleep systems. (1/4)

sciencedirect.com

Integrating synthetic biology to understand and engineer the heart, lung, blood, and sleep systems

Synthetic biology offers control over cellular and tissue functions. As it moves beyond microbes into humans, synthetic biology enables precise contro…

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SCGE @scge.bsky.social · 08/07/2026

In this interview, Greg Mohl, PhD highlights the challenges of delivering CRISPR therapies to the brain and discusses how him and the research team try to balance designing the most effective therapy with the time pressure to get therapies to patients. www.youtube.com/watch?v=QGQd...

Meet the Researcher: Greg Mohl

SCGE @scge.bsky.social · 07/07/2026

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The U.S. Food and Drug Administration (FDA)'s platform technology designation program aims to streamline the development and approval process for advanced therapy medicinal products and is anticipated to be beneficial for the development of gene therapies for rare and ultra-rare diseases. (1/3)

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SCGE @scge.bsky.social · 01/07/2026

In this interview, Jennifer Panagoulias provides her perspective on some current regulatory frameworks, and shares practical advice for academic labs preparing their first IND application for a gene therapy. Watch the full interview at: www.youtube.com/watch?v=53WP...

Meet the Researcher: Jennifer Panagoulias

SCGE @scge.bsky.social · 23/06/2026

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In this study, SCGE researchers isolated a library of natural capsids from human tissues and identified two variants that conferred strong transduction of retinal tissues following intravitreal injections into mice and non-human primates. (1/3)

sciencedirect.com

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SCGE @scge.bsky.social · 19/06/2026

Industry News: Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration investors.beamtx.com/news-release...

investors.beamtx.com

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SCGE @scge.bsky.social · 17/06/2026

In this "Meet the Researcher Interview", hear from Karen Clark, PhD, as she highlights the free resources available on the SCGE platform. www.youtube.com/watch?v=O2gj...

Meet the Researcher: Karen Clark

SCGE @scge.bsky.social · 10/06/2026

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In this SCGE "Meet the Researcher" interview, hear from Dr. Xiaoshu Pan as she highlights the benefits of extracellular vesicles (EVs) as a delivery method for gene therapies, and discusses the next milestones in her SCGE project. Watch the interview: www.youtube.com/watch?v=0HTR...

Meet the Researcher: Xiaoshu Pan

SCGE @scge.bsky.social · 09/06/2026

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CRISPR-based genome editing therapeutics are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess the safety and efficacy of these new therapies and accelerate progress. (1/3) www.nature.com/articles/s41...

nature.com

Monitoring biological effects of somatic cell genome editing - Nature Reviews Genetics

CRISPR-based genome editing therapeutics are entering the clinic, but in vitro and in vivo tools are needed to assess their safety and efficacy. The authors review complementary technologies to monito...

SCGE @scge.bsky.social · 03/06/2026

In this SCGE researcher interview, Dr. Mei He highlights how her teams' high-throughput extracellular vesicle loading platform improves the delivery of genome editors and discusses the different kinds of expertise needed to build her research project. www.youtube.com/watch?v=cmor...

Meet the Researcher: Mei He

SCGE @scge.bsky.social · 02/06/2026

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Autosomal recessive polycystic kidney disease (ARPKD) leads to severe renal cysts and progressive kidney dysfunction, with no approved treatments. The absence of such cystic phenotypes in Pkhd1-/- mice underscores the need for novel models that better recapitulate the human disease. (1/5)

advanced.onlinelibrary.wiley.com

Deciphering the Impact of RAC1‐SPTAN1 in ARPKD Cystogenesis Using Multifaceted Models

Distal/connecting tubules expressing SLC8A1 have been suggested as a potential origin of ARPKD cysts. SPTAN1 has been identified as a key molecule in ARPKD cyst formation. Restoring SPTAN1 in PKHD1−/...

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SCGE @scge.bsky.social · 26/05/2026

Emerging therapies in sickle cell disease (SCD) aim to restore healthy red blood cell (RBC) function, but they often yield heterogeneous cellular responses. There are no proven techniques to evaluate restored rheological functionality and heterogeneity in these RBCs. (1/3)

pubs.rsc.org

Microfluidic capillary transit velocity as a functional measure for sickle cell disease and in vitro-derived red blood cells

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SCGE @scge.bsky.social · 20/05/2026

Are you part of a research team that is submitting (or planning to submit) an Investigational New Drug (IND) application for a novel gene therapy? View our free IND application resources at scge.mcw.edu/platform/dat... #GeneTherapy #Regulatory #FreeResource

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SCGE @scge.bsky.social · 19/05/2026

Natural killer (NK) cells have been engineered to express chimeric antigen receptors (CARs) to enhance their cytotoxic capabilities through CAR-mediated activation, a strategy that has yielded promising advancements in cancer treatment in recent pre-clinical and clinical trials. (1/3)

frontiersin.org

Frontiers | Perspective: IL-15 cytokine-armored NK cells as ready-to-use immunotherapy for diverse malignancies: therapeutic potential and toxicity risks

Natural killer (NK) cells have been engineered to express chimeric antigen receptors (CARs) to enhance their cytotoxic capabilities through CAR-mediated acti...

SCGE @scge.bsky.social · 11/05/2026

Reposted by SCGE

Thursday May 14th: - Beverly Davidson, Outstanding Achievement Award Winner - Samantha Maragh, "NIST Genome Editing Consortium expanded and extended to develop genome editing control strategies, tools, samples, and standards" (8/9)

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Reposted by SCGE

SCGE @scge.bsky.social · 11/05/2026

Thursday May 14th: - Beno Freedman, "Using human iPS cell-derived organoids and CRISPR gene editing in kidney diseases" - Alan Flake, "Artificial womb to support fetal development and manipulations" (7/9)

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Reposted by SCGE

SCGE @scge.bsky.social · 11/05/2026

Thursday May 14th: - Danilo Pellin, "Metagenomic integration analysis of hematopoietic stem cell following lentiviral gene transfer" - Chris Baehr, "Chemically modified CRISPR enzymes for multi-organ genome editing in vivo" (6/9)

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Reposted by SCGE

American Society of Gene + Cell Therapy @asgct.bsky.social · 14/05/2026

Don’t miss today’s general session featuring three visionary keynote speakers! We'll also be celebrating the recipients of the George Stamatoyannopoulos Mentorship Award, the Excellence in Research Award, and the Mavis Agbandje-McKenna Scholarship and Fund. Details: bit.ly/3P3UiQl