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)

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 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.