Such EV-based delivery strategy, with a scalable EV loading approach, enhanced the in vivo PROTAC drug stability and bioavailability and improved tissue penetration and targeting, filling an important gap in the clinical translation of PROTAC-based cancer therapy. (3/3)
To enhance the in vivo therapeutic efficacy of PROTACs, SCGE researchers introduced extracellular vesicles (EVs) for in vivo PROTAC delivery, which is leveraged by a novel microfluidic droplet-based EV electro-transfection system (μDES). (2/3)
Proteolysis targeting chimeras (PROTACs) represent an emerging targeted cancer therapy approach. However, their poor cell penetration and instability in vivo pose daunting challenges for wide-spread clinical usage. (1/3)
isevjournals.onlinelibrary.wiley.com/doi/10.1002/...
#Biotech #GeneTherapy
That's a wrap on the SCGE spring 2026 meeting! Thank you to everyone who attended the meeting virtually and in-person.
Find a photo recap of the meeting here: scge.mcw.edu/2026/04/15/s...
Closing remarks from Dr. Joni Rutter to end our SCGE spring 2026 meeting.
Trainee talks from Ms. Elena Kahn and Mr. Hanbing Cao at our SCGE spring meeting.
A couple of presentations from our technology and assay project talks at our SCGE spring meeting.
Presentation about the progress of the SCGE TCDC by Drs. Karen Clark (pictured) and Mindy Dwinell.
See exciting updates to the SCGE platform at scge.mcw.edu/platform/home
A thoughtful discussion this morning on regulatory guidance, data capture and clinical trial infrastructure led by Drs. Sarah Dunsmore and Rebecca Ahrens-Nicklas at our SCGE spring meeting.
Photos from our poster session today at the SCGE spring meeting.
Excellent trainee talks today from Dr. Gregory Mohl and Dr. Tim Barry.
An engaging group discussion led by Dr. Sonia Vallabh.
SCGE Spring 2026 Meeting Group Photo!
The SCGE spring 2026 meeting is off to a strong start with some great presentations this morning!
Congrats to SCGE researchers Kiran Musunuru and Rebecca Ahrens-Nicklas for being named to Time's "100 Most Influential People in 2026" list!
Read the blurb at: time.com/collection/1...
We are starting off our SCGE Spring 2026 meeting by celebrating researcher wins!
Looking forward to great presentations and discussions by consortium researchers at the meeting.
"KJ’s story is a miracle. But it should not remain a miracle. It should become a model."
This article highlights the work of SCGE researchers who created a personalized gene therapy for KJ and are now trying to expand on this work to treat other patients.
www.nytimes.com/2026/04/09/o...
Description of genetic disease
Description of heritable disease
Learn more about rare and genetic diseases in our patient and caregiver guide: scge.mcw.edu/patient-care...
#RareDisease #GeneticDisease #PatientEducation
In this commentary, SCGE researchers explore αβ T cell-derived engineering strategies applicable to γδ T cells, while also highlighting genome-editing innovations poised to advance next-generation γδ CAR-T development.
jitc.bmj.com/content/13/1...
#RareDisease #GeneTherapy #Engineering
Primary care physicians (PCPs) can be a great resource for rare disease patients as they manage their condition. Below are a few ways that PCPs can help their rare disease patients and families.
Find more information and resources for PCPs at scge.mcw.edu/pcp-guide/
#RareDisease #PrimaryCare
🚨 New today!!
📄Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform
🧑🤝🧑 @kiranmusunuru.bsky.social @ahrensnicklas.bsky.social & co
You can find the researchers' Pre-IND briefing book and the FDA’s written responses available on the SCGE website at scge.mcw.edu/platform/pub...
Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform: The American Journal of Human Genetics www.cell.com/ajhg/fulltex...
Find their Pre-IND briefing book and the FDA’s written responses available on the SCGE website at scge.mcw.edu/platform/pub...
Read the full publication (open-access) here: www.cell.com/ajhg/fulltex... (4/4)
#GeneTherapy #PersonalizedMedicine #RareDisease
They anticipate that their findings will be of interest to academic investigators and industry sponsors who wish to pursue expeditious FDA approvals of therapies for ultra-rare diseases using the plausible mechanism framework. (3/4)
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. (2/4)
In this new publication, SCGE researchers report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders (UCDs) and other liver-centered disorders... (1/4)
Consistently, expression of IL-15 resulted in blockade of tumor progression in the TKP CCA model.
These findings highlight the importance of oncogenic Kras in CCA tumor maintenance and underscore KRAS inhibition as a potential therapeutic approach for CCA. (3/3)
#RareDisease #BiomedicalResearch