Somatic Cell Genome Editing (SCGE) Outreach

SCGE researchers speculate that the use of humanized mice will allow for engraftment of tumor and alleviate cytokine armored CAR-NK toxicity, thereby allowing for effective assessment of CAR-NK efficacy in the absence of toxicity. #PublicationFriday frontiersin.org/journals/immun…

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/data/… #GeneTherapy #Regulatory #FreeResource

Atamyo Therapeutics Reports Promising Early Results from the First Patients Treated with ATA-200 Gene Therapy in its LGMD-R5 Clinical Trial - For More Information Visit shorturl.at/fKQqN @AtamyoTX #LGMD #LGMD_R5 #Limb_Girdle #Muscular_Dystrophy #Rare_diseases

One-Time Gene Therapy Shows Potential to Improve Bone Health in Adults With Gaucher Disease - For More Information Visit shorturl.at/bWtVd @SpurTherapeutx #Gaucher_Disease #Rare_Diseases #Gaucher

SCGE researchers recently published an article on peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC), a new approach for ex vivo genome editing of primary human cells. #PublicationFriday nature.com/articles/s4159…

Don’t miss today’s George Stamatoyannopoulos Memorial Symposium featuring three visionary keynote speakers: 🔹Paula Cannon, PhD: Designing what nature can’t: Cell and gene therapy for HIV 🔹Erik Sontheimer, PhD: CRISPR, from bacterial immunity to genome editing therapeutics 🔹Beverly Davidson, PhD: Outstanding Achievement Award Recipient 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. See you there! Session details: pag.virtual-meeting.org/US/asgct2026/e…

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)

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)

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)

Today in @ScienceTM, we report the use of in vivo adenine base editing to correct a variant causing Dravet syndrome, a severe childhood epilepsy and neurodevelopmental disorder, substantially ameliorating disease symptoms and extending lifespan in an animal model. 1/13 drive.google.com/file/d/12rcxew…

Wednesday May 13th: - David Spencer, "From a research staple to a clinical approval for the versatile chemical inducer of dimerization rimiducid" (5/9)

Wednesday May 13th: - Lindsey George, "AAV and systemic toxicities in clinic trials and clinical practice" - Karen Clark, "The Gene Therapy Clinical Trial Browser: Development of a public data resource for monitoring the gene therapy clinical trial landscape" (4/9)

Wednesday May 13th: - Allyson Berent, "Patient and nonprofit engagement driving therapeutic advancement" - Somatic cell genome editing as a therapeutic platform: From clinic to coverage (Scientific Symposia) (3/9)

Wednesday May 13th: - Elizabeth Berry-Kravis, "Adaptive and innovative study design in rare diseases" - Lindsey George, "Evolving understanding of AAV toxicities" - Fiona Serack, "Progress towards a one-time epigenetic editing therapy for prion disease" (2/9)

There's still time to register! ⬇️

There are a lot of SCGE researchers presenting at #ASGCT2026 this week! Check out this thread to learn more. Tuesday May 12th: - Bill Peranteau, "Germline editing: Technical background perinatal" - Becca Ahrens-Nicklas and Kiran Musunuru are accepting the Catalyst Award (1/9)

We are proud to see our trainees Apoorva Ramamurthy and Anna Tommasi presenting their research at #ASGCT2026 in Boston. Apoorva presents her oral abstract on May 12 at 11:30 a.m., followed by Anna in the Hot Topics session on May 13 at 4 p.m. Learn more: bit.ly/4w9Ooxy

You won't want to miss our first general session of #ASGCT2026! Hear from: 🔹Founders Award recipient R. Jude Samulski, PhD 🔹Mendell Award Recipient @timyu 🔹Catalyst Award co-recipients @AhrensNicklas, @kiranmusunuru, @UrnovFyodor, and @DanaherCorp Plus, meet this year's Career Development Award and Underrepresented Populations Fellowship recipients. Join us at 1:30 p.m. ET in Hall B1 or stream it live! Session details: pag.virtual-meeting.org/US/asgct2026/e…

Myrtelle to Present New Clinical Trial Data for Canavan Disease Gene Therapy at ASGCT 2026, Highlighting Early Signs of Therapeutic Benefit - For More Information Visit shorturl.at/2ylkH @MyrtelleInc #ASGCT #Canavan_Disease #Orphan_Drugs #Rare_Diseases

🧬 See you in Boston for the ASGCT 2026 Annual Meeting! We’re excited to see strong representation from Gene Therapy Institute investigators, trainees, and collaborators throughout this year’s meeting. Check out posters featuring our researchers below!   #ASGCT2026 #GeneTherapy