Somatic Cell Genome Editing (SCGE) Outreach

SCGE researchers provide a practical framework to evaluate and assess off-target safety based on the tools available today and ones that will be developed in the future, aiming to streamline the transition of CRISPR therapeutics from bench to bedside. nature.com/articles/s4158…

Ultragenyx Reports Positive Phase 3 Results for DTX301 Gene Therapy in OTC Deficiency, Showing Significant Ammonia Reduction - For More Information Visit shorturl.at/fJ2Jd @ultragenyx #OTC_Deficiency #Rare_Diseases #Orphan_Drugs #Ornithine_Transcarbamylase_deficiency

Here, SCGE researchers use induced pluripotent stem cells (iPSCs) and iPSC-derived neurons to examine how postmitotic human neurons repair Cas9-induced DNA damage. #PublicationFriday nature.com/articles/s4146…

Explore the complex interplay between advanced nanotechnology and biomedical innovation, focusing on the transformative potential of LNPs in developing precise and effective therapeutic interventions in this free webinar. Join us March 25 at 10 a.m. CT: bit.ly/3MJ6RiV

Benitec Reports Promising Interim Results with 100% Response Rate in BB-301 Gene Therapy Trial for Oculopharyngeal Muscular Dystrophy - For More Information Visit shorturl.at/tlFXR @BenitecBio #Rare_Diseases #Oculopharyngeal_Muscular_Dystrophy #Orphan_Drugs

The SCGE platform has added a new section that provides information on the forms and documents needed for an initial IND application for a gene therapy. Learn more at scge.mcw.edu/platform/data/… #Regulatory #IND #GeneTherapy

Sangamo Therapeutics Advances Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease - For More Information Visit shorturl.at/bQOSX @SangamoTx #Sangamo #Fabry_Disease #Rare_Diseases #FDA

The Buffalo Initiative is a mission-driven effort to accelerate treatments for ultra-rare diseases by empowering patient-led, nonprofit, and mission-driven innovators with funding, infrastructure, and collaboration. Learn more at buffaloinitiative.org #RareDisease

Atamyo Therapeutics Reports Positive Results for ATA-200 Gene Therapy in Limb-Girdle Muscular Dystrophy Type 2C/R5 - For More Information Visit shorturl.at/eyH6s @AtamyoTX #LGMD_2C_R5 #Rare_Diseases #Muscular_Dystrophy #Limb_Girdle #Orphan_Drugs #LGMD #Atamyo

Precision BioSciences’ PBGENE-DMD Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Treatment - For More Information Visit shorturl.at/kUlFo @PrecisionBioSci #Duchenne #FDA #Rare_Diseases #DMD #Muscular_Dystrophy #Orphan_Drugs #Pediatric_Disorder

We’re thrilled to welcome Rebecca Ahrens-Nicklas, MD, PhD as a keynote speaker for the 2nd Annual Gene Therapy Symposium. Her research develops gene therapies for rare metabolic diseases 🧬 #GeneTherapy #RareDisease #GenomicMedicine

Congratulations to Beverly L. Davidson, Ph.D., recipient of the 2026 ASGCT Outstanding Achievement Award! As the Society’s highest honor, this award recognizes Dr. Davidson’s pioneering research into the molecular mechanisms of inherited brain diseases and her leadership in developing and testing gene-based therapeutic strategies. Join us in celebrating her incredible contributions to the field at #ASGCT2026! Meet all of our 2026 award winners: asgct.org/news-publicati…

“I don’t think it’s exaggerating to say that this is the future of medicine.” – Dr. Kiran Musunuru We are thrilled to welcome @TIME 100 Health honoree @kiranmusunuru, MD, Ph.D., MPH, ML, MRA (@Penn), to the Empowering Patients Summit this week! Fresh off his groundbreaking work developing a world-first customized CRISPR therapy for an infant with a rare genetic mutation, Dr. Musunuru joins Olivia Kim-McManus, MD (@radychildrens), to explore The Realities of N of 1 and Implications for the Patient Community. Don’t miss this deep dive into the promise and complexities of individualized gene therapies and what they mean for the future of ultra-rare disease advocacy. Join us for this free, virtual event (March 11+12): asgct.org/empowering

Here, SCGE researchers employ base editing in primary human T cells to install naturally occurring dominant negative FAS and TGFβR2 mutations. #PublicationFriday pmc.ncbi.nlm.nih.gov/articles/PMC12…

Professor @davidrliu of @HarvardCCB is the recipient of the 2026 @AmerChemSociety Award for Creative Invention! See his #ACSSpring2026 talk at 3:25 PM in Convention Center room B206 on Tuesday, March 24.

Find more tips and resources for PCPs at scge.mcw.edu/pcp-guide/ #RareDisease #PrimaryCare #ClinicalTrials

Many rare diseases do not have approved treatments, making clinical trials the best (or only) option for patients. Sometimes patients or caregivers may come to a PCP to ask for guidance about a clinical trial. The following tips may help with navigating these discussions:

SCGE researchers Kiran Musunuru and Fyodor Urnov will be speaking at the event during the segment titled "'Plausible mechanism' and regulating personalized therapies" at 12:55pm ET.

This Thursday (March 5th) is Endpoints News Cell & Gene Day 2026. This virtual event will examine the economics, regulatory shifts, and global competition that are reshaping cell and gene therapies. Find more information at events.endpoints.news/cgday26/home #GeneTherapy #CellTherapy

🎬Missed FDA's virtual #RareDiseaseDay? The full recording is now available on the FDA’s YouTube page! youtube.com/live/B6bGjpQVq… Explore our discussions on key rare disease topics and FDA's continued commitment to this community. fda.gov/RareDiseaseDay