Findings from SCGE researchers suggest that liver base editing 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. nature.com/articles/s41551-0…

FDA has cleared the IND for PM647, our investigational in vivo Prime Editor for alpha-1 antitrypsin deficiency (AATD), designed to correct the most prevalent disease-causing mutation. Initial data expected in 2027. tinyurl.com/mum7reba #PrimeEditing #AATD #GeneEditing

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 linkedin.com/pulse/scge-spot…

These studies used total-body PET to identify somatic cell gene editing in vivo in fetal rhesus macaques and showed that total-body PET can provide insights into gene-edited somatic cells in utero and without evidence of adverse effects. pmc.ncbi.nlm.nih.gov/article…

Today we published our first organoid model of diabetic kidney disease! #DKD is a major health issue. High glucose causes human #minikidneys to lose structure via cytokines. Press release @ISSCR : lnkd.in/ek_SWr3k Full paper @stemcellreports : lnkd.in/e4iuCQfq

Today, we approved a one-time gene therapy for pediatric patients with Sanfilippo syndrome type A (MPS IIIA), a rare, inherited disorder that progressively damages the brain and central nervous system. This is the first approved treatment for this condition. fda.gov/news-events/press-an…

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 sessions planned for this workshop. Register to attend at scge.mcw.edu/scge-workshop/

Today we announced the final design of the Expedited Investigational New Drug (IND) Pilot, a key Trump Administration priority to accelerate American medical innovation and keep early clinical research in the U.S. The program seeks to accelerate the time from a drug being identified to a first-in-human clinical trial. FDA will be partnering drug companies with qualified research institutions that have scientific and regulatory expertise to support efficient IND applications. The agency will accept applications to participate in the pilot until October 30, 2026. fda.gov/news-events/press-an…

Here, SCGE researchers share 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 7 urea cycle disorders (UCDs). #PublicationFriday cell.com/ajhg/fulltext/S0002…

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/data/s…

Today, Beam announced updated clinical data from the Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency. Beam will host a conference call & webcast today at 7:00 a.m. ET to review these updates. Read the press release here: investors.beamtx.com/news-re… #DaretoBeam

Here, SCGE researchers identify base editing strategies for in vivo correction of the second and third most common pathogenic variants of Phenylketonuria (PKU). #PublicationFriday sciencedirect.com/science/ar…

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/

Today we announced the submission of a BLA to the U.S. FDA for the accelerated approval of AMT-130, an investigational #genetherapy for the treatment of #HuntingtonsDisease. We also announced that an MAA for AMT-130 has been submitted to the U.K.’s MHRA. uniqure.gcs-web.com/news-rel…

Researchers demonstrate that an innovative methodology called calcium shock dramatically improves particle delivery into single cells, colonies, and organoids, and enables programmable delivery into specific cell types within heterocellular populations. pmc.ncbi.nlm.nih.gov/article…

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 linkedin.com/pulse/scge-spot…

Precision BioSciences Doses First Patient in Phase 1/2 Trial of PBGENE-DMD Gene Editing Therapy for Duchenne Muscular Dystrophy - For More Information Visit shorturl.at/sMq8H #Duchenne #Muscular_Dystrophy #DMD @PrecisionBioSci #Rare_Diseases #Orphan_Drugs #FDA

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. #PublicationFriday science.org/doi/10.1126/scit…

FDA Grants Accelerated Approval to Ultragenyx’s GENGLYCOS, First Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) - For More Information Visit shorturl.at/4vEPN @ultragenyx #Rare_Diseases #GSDIa #FDA #Glycogen_Storage_Disease #Orphan_Drugs #Ultragenyx

Big news!!! news.med.miami.edu/kidney-or…