SCGE

Non-viral nanomedicines, including nanoparticles (NPs) composed of lipids and polymers, represent a transformative approach to drug and gene therapy. (1/5) www.sciencedirect.com/science/arti...

Animal models and new approach methodologies are also addressed for NP assessment and screening, including the physiological distinctions when comparing rodent models to larger species that can impact NP delivery. (4/5)

Non-viral nanomedicines, including nanoparticles (NPs) composed of lipids and polymers, represent a transformative approach to drug and gene therapy. (1/5) www.sciencedirect.com/science/arti...

However, clinical translation of these technologies is limited by two key barriers: the scale-up of NP formulations and the challenge of conducting predictive preclinical studies in relevant animal models. (2/5)

Kidney organoids derived from human pluripotent stem cells have emerged as promising models for studying kidney disease and therapeutic development. However, the lack of a scalable production system has limited their industrial applications in regenerative medicine. (1/3)

Here, a team including SCGE researchers have developed a cost-effective mass-production method for manufacturing vascularized kidney organoids, which has improved production efficiency by more than 50 times compared to conventional culture systems. (2/3) pmc.ncbi.nlm.nih.gov/articles/PMC...

In February 2026, the US Food and Drug Administration (FDA) published a draft guidance on a new plausible mechanism framework for the development and approval of individualized therapies for genetic conditions.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

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...

In February 2026, the US Food and Drug Administration (FDA) published a draft guidance on a new plausible mechanism framework for the development and approval of individualized therapies for genetic conditions.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

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)

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...

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)

Translational development of somatic cell genome editing requires monitoring the extent of editing in the body at a given time, the specificity of editing, durability, and the potential for adverse events. (1/4)

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)

Translational development of somatic cell genome editing requires monitoring the extent of editing in the body at a given time, the specificity of editing, durability, and the potential for adverse events. (1/4)

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)

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

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)

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)

The clinical translation of gene therapy has been challenging in part because of the limitations of current delivery approaches. (1/3)

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/...

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...