“Never So Jesus” — A May Update
There are moments in GG’s precision medicine journey that feel technical, and there are moments that feel deeply human. This past month gave us both.
On the scientific side, we are continuing to advance our ASO program in a meaningful way.
Our lead candidates—originally identified through high-throughput screening—have now demonstrated reproducible increases in NAA15 expression at the mRNA level, along with emerging evidence of corresponding protein-level changes under the care of talented scientists at La Jolla Labs.
This has been a critical step for us, moving the work beyond isolated transcript signal into early evidence of biologically meaningful translation. Importantly, this process has yielded a broad funnel of active candidates rather than a single isolated hit, giving us multiple lead ASOs to evaluate as we move into disease-relevant systems.
Unlike traditional antisense oligonucleotides (ASOs), which are typically designed to reduce or silence gene expression, this program is attempting something far less established: increasing expression of an underactive gene. Achieving reliable upregulation at both the RNA and protein level in human neuronal systems has historically been much more difficult and less predictable than traditional gene-silencing approaches. That is part of why these early translational signals have been so encouraging to our team.
We are now taking these candidates into an NAA15 mutation system: a CRISPR-engineered cell line carrying GG’s specific mutation. This allows us to evaluate whether the same upregulation holds in a model that more closely reflects her biology.
This step matters. It’s where early promise starts to meet real-world complexity.
May 2026
In parallel, we are making important progress on GG’s patient-derived induced pluripotent stem cell (iPSC) line.
In simple terms, this means we can now take GG’s cells and turn them into the kinds of brain cells affected by her condition—growing versions of GG’s own brain cells in a lab.
These neurons carry her exact genetic makeup, including the NAA15 mutation. This gives us a way to study her biology—and test our potential treatments—in a system that is uniquely hers.
We are now guiding these cells to become two key types of neurons:
Glutamatergic neurons (which help activate brain signaling)
GABAergic neurons (which help regulate and balance that activity)
As these models develop, they will allow us to:
See whether our ASO candidates increase NAA15 in GG’s own cells
Determine whether the protein trends observed in earlier systems are reproducible in GG’s own cells
Begin to understand how this may impact real neuronal function in NAA15-related conditions
Evaluate potential neuronal rescue associated with NAA15 upregulation
Advancing GG’s iPSC Model
What Comes Next
As these systems come online, we will be integrating data across:
LJL screening (RNA + protein)
CRISPR disease models
GG’s iPSC-derived neurons
This combined dataset will inform selection of 2–3 lead candidates for advancement into early in vivo safety studies and IND-enabling development.
Naming the ASO
And then there was the name.
While drafting an email to Dr. Olivia Kim-McManus—GG’s neurologist and one of our key advisors—I found myself writing a name for the therapy: Georgiasen.
It wasn’t something we had formally decided. It just… came.
So I asked GG what she thought—if she liked it, if it felt right to her.
GG is nonspeaking and communicates by spelling, so we brought out the letterboard.
She spelled:
“Never So Jesus.”
What she meant was clear to me.
It’s a divine name, mom—and it may just be the miracle I’ve been asking for.
A Moment of Resolve
And after encouraging early protein readouts in the lab, I sat with GG again.
We brought out the letterboard.
She spelled:
“I’m not stopping until something is made.”
“Get it made for me.”
There is a clarity in that kind of communication that’s hard to explain.
It doesn’t replace the science. It doesn’t change the work that has to be done.
But it reminds me—very clearly—why we are doing it.
We don’t take moments like that lightly.
This work is grounded in science, rigor, and an extraordinary amount of effort from people who are dedicating their lives to making something like this possible. But every so often, we’re reminded that it’s also something else.
Something deeply personal—while also carrying GG’s belief that what we are building could help others, too.
We are incredibly excited to be advancing this program into its next phase.
And we are deeply grateful—for the science, for the people, and for the community that continues to support GG and this mission.
Thank you for being part of this with us.
Acknowledgments
With gratitude to the individuals and teams helping move this work forward:
Scientific & Clinical Advisors
Dr. Michelle Mattson-Hoss
Dr. Olivia Kim-McManus
Dr. Gholson Lyon
Curt Mazur
Development & Research Partners
La Jolla Labs — Geramie Shaver, Melissa Keenan, Tamar Grossman, Jeff Milton
iXcells - Joan Schein, Danny Medina, Bing Yan, Xinyu Kong, Christy Holt
Dewing Bio and BioQual - our Toxicology screening partners
ProPharma - Steve Jensen, Kosha Shah, Gregory Kufner, Elizabeth, Soames,
Dr. Kristen Wigby and the Precision Medicine Team at Rady Children’s Hospital
Advarra - for their support on our IRB
EditCo - Ashleigh Teator
ChemGenes - Jeremy Little & Anuj Mohan
Her clinical care team at Rady Children’s Hospital in San Diego including:
Olivia Kim-McManus, MD; Amber Contillo, NP; Anita Sharma, MD; Anuja Jindal, MD; Ashley Sheng, PA-C; Brittany Passiak, MD; Cathleen Collins, MD; Elizabeth Patty, NP; Emily Ramirez, NP; Hari Narayan, MD; Henri Justino, MD; Corrine Blucher, Kristen Wigby, MD; Jennifer Friedman, MD; Matthew Bock, MD; Morgan Bliss, MD; Paul Grossfeld, MD; Robert Gray, PhD; Wen Jiang, MD
Dr. Richard Frye for recommending whole genome sequencing in 2025, and Lanier Rossignol, RN who insisted on it.
And to the families and children who continue to show us what is possible.