SCGE

The SCGE platform has added a section that provides information on the forms and documents needed for an initial IND application for a gene therapy. This section includes relevant guidance, templates, examples, and resources to assist researchers in their applications.

scge.mcw.edu/platform/dat...

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 about this exciting resource at buffaloinitiative.org

#RareDisease

By studying DNA repair in clinically relevant cells, they reveal unforeseen challenges and opportunities for precise therapeutic editing. (4/4)

Here, SCGE researchers address this barrier by using induced pluripotent stem cells (iPSCs) and iPSC-derived neurons to examine how postmitotic human neurons repair Cas9-induced DNA damage. (3/4)
www.nature.com/articles/s41...

DNA repair is especially understudied in nondividing cells like neurons, limiting the efficiency and precision of genome editing in many clinically relevant tissues. (2/4)

Genome editing is poised to revolutionize treatment of genetic diseases, but poor understanding and control of DNA repair outcomes hinders its therapeutic potential. (1/4)

Find more tips and resources for PCPs at scge.mcw.edu/pcp-guide/ (2/2)

#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. (1/2)

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.
@kiranmusunuru.bsky.social @urnov.bsky.social

Cell and Gene Day 2026 Cell and gene therapy has some wind beneath its sails. New regulatory frameworks from the Food and Drug Administration could help streamline development and manufacturing. Large pharma companies are i...

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

Installation of dominant-negative mutations in FAS and TGFβR2 via base editing in primary T cells Adoptive cell transfer (ACT) of engineered T cells is effective against B cell malignancies but has faltered against solid tumors due to the immunosuppressive tumor microenvironment (TME). FAS ligand (FASL) and transforming growth factor β (TGF-β) ...

Considering the efficiency of BE and its amenability for multiplex editing, this approach lends itself to engineering strategies necessary to overcome T cell dysfunction in solid tumors. (2/2)

#GeneTherapy #Biotech

Installation of dominant-negative mutations in FAS and TGFβR2 via base editing in primary T cells Adoptive cell transfer (ACT) of engineered T cells is effective against B cell malignancies but has faltered against solid tumors due to the immunosuppressive tumor microenvironment (TME). FAS ligand (FASL) and transforming growth factor β (TGF-β) ...

Here, SCGE researchers employ base editing (BE) in primary human T cells to install naturally occurring dominant negative FAS and TGFβR2 mutations. (1/2)

pmc.ncbi.nlm.nih.gov/articles/PMC...

What is a rare disease?

Find more information in our new guides:

Providing Better Care for Rare Disease Patients: Primary Care Physician Guide - scge.mcw.edu/pcp-guide/

Information Guide for Rare Disease Patients and Caregivers - scge.mcw.edu/patient-care...

How to create personalized gene editing platforms: Next steps toward interventional genetics How do we go from a single individual receiving a personalized gene-editing therapy to a future of “interventional genetics” in which such therapies are the standard of care? First and foremost: regul...

In this article, SCGE researchers discuss their ideas for a future of “interventional genetics”, in which personalized gene-editing therapies are the standard of care.

www.cell.com/ajhg/fulltex...

#RareDisease #PersonalizedMedicine #RegulatoryInnovation #GeneTherapy

Meet the winners of the 2026 ASGCT Honorific Awards! This year, we are thrilled to recognize 17 distinguished leaders who are making a profound impact to advance the field of cell and gene therapy.

Read the full announcement and learn more about our winners: https://bit.ly/4aQZi1C

FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety whe...

Rare Disease News: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

www.fda.gov/news-events/...

Adeno-associated Virus-mediated Gene Therapy: Advances, Immune Challen Adeno-associated Virus-mediated Gene Therapy: Advances, Immune Challenges, and Research Innovations

For those interested:

FDA Grand Rounds – Adeno-associated Virus-mediated Gene Therapy: Advances, Immune Challenges, and Research Innovations

Thursday, February 19th, 2026 at 12:00pm ET

www.fda.gov/science-rese...

Looking forward to it!

excited to be giving a Rare Story at 10:50am at #RDDNIH! I don’t get to publicly speak about what it’s like to be a #RareDisease patient working on genetic therapies very often so I’m quite excited!

Find a few events for #RareDiseaseDay listed below:

🦓 FDA Rare Disease Day 2026 (February 23rd): www.fda.gov/news-events/...

🦓 Rare Disease Day at NIH (February 27th): ncats.nih.gov/news-events/...

🦓 RareDiseaseDay.Org Global Events Calendar: www.rarediseaseday.org/category/eve...

Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs - Nature Biomedical Engineering Hybrid gRNAs significantly increase targeted editing in the liver while simultaneously reducing unwanted bystander editing in humanized mouse models.

These studies highlight the use of hybrid gRNAs to improve the safety and efficiency of adenine base-editing therapies. (3/3)

www.nature.com/articles/s41...

#GeneTherapy #RareDisease #Biotech

Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs - Nature Biomedical Engineering Hybrid gRNAs significantly increase targeted editing in the liver while simultaneously reducing unwanted bystander editing in humanized mouse models.

Here SCGE researchers evaluate the off-target editing profiles of clinical lead gRNAs that, when combined with adenine base editors, correct the recurrent PAH P281L variant, PAH R408W variant or ABCC6 R1164X variant, or disrupt either of two sites in the HPD gene in human hepatocytes. (2/3)

Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs - Nature Biomedical Engineering Hybrid gRNAs significantly increase targeted editing in the liver while simultaneously reducing unwanted bystander editing in humanized mouse models.

Phenylketonuria (PKU), pseudoxanthoma elasticum (PXE) and hereditary tyrosinemia type 1 (HT1) are autosomal recessive disorders linked to the PAH, ABCC6, and FAH and HPD genes, respectively. (1/3)

Text on blue background welcoming the National Academy of Engineering Class of 2026 with the NAE logo below.

This week, four members of the UC Berkeley community, including IGI Founder Jennifer Doudna, were elected to the National Academy of Engineering — one of the highest professional distinctions for engineers (and #bioengineers). Learn more: https://ow.ly/Zivp50YcAV6

TIME100 Health: Kiran Musunuru and Rebecca Ahrens-Nicklas Find out why they're on the TIME100 Health 2026 list.

Congrats to SCGE researchers Kiran Musunuru and Rebecca Ahrens-Nicklas for being named to the Time100 Most Influential People in Health list for 2026!

time.com/collections/...

Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice Pathogenic missense mutations in the alpha actin isotype 2 gene (ACTA2) cause multisystemic smooth muscle dysfunction syndrome (MSMDS), a genetic vasculopathy that is associated with stroke, aortic dissection, and death in childhood. Here, we ...

Here, SCGE researchers perform mutation-specific protein engineering to develop a bespoke CRISPR-Cas9 enzyme with enhanced on-target activity against the most common MSMDS-causative mutation ACTA2 R179H.
pmc.ncbi.nlm.nih.gov/articles/PMC...

FDA Rare Disease Innovation Hub FDA created the Rare Disease Innovation Hub (the Hub) to serve as a point of collaboration and connectivity between CBER and CDER.

Gene Therapy News: FDA Rare Disease Innovation Hub 2026 Strategic Agenda is now available at www.fda.gov/industry/med...

Microfluidic Device Type Improves Heart mRNA Delivery In Vivo To improve lipid nanoparticle (LNP)-mediated delivery to nonliver tissues, scientists modify LNP chemistry or add targeting ligands. One underexplored alternative is to change the formulation process ...

Here, SCGE researchers report that an LNP formulated with a herringbone mixer led to 2-fold more heart delivery than the same LNP formulated with a bifurcating mixer. These data suggest that it is possible to increase heart delivery via nanoparticle processing. (2/2)
pubs.acs.org/doi/10.1021/...

Microfluidic Device Type Improves Heart mRNA Delivery In Vivo To improve lipid nanoparticle (LNP)-mediated delivery to nonliver tissues, scientists modify LNP chemistry or add targeting ligands. One underexplored alternative is to change the formulation process ...

To improve lipid nanoparticle (LNP)-mediated delivery to nonliver tissues, scientists modify LNP chemistry or add targeting ligands. One underexplored alternative is to change the formulation process that creates the LNP. (1/2)

3)And more!

View the platform website at scge.mcw.edu/platform/home

#RareDisease #Regulatory #GeneTherapy #FreeResource (3/3)