AcuraStem

AcuraStem is looking forward to joining the European Network to Cure ALS community in Madrid from June 24 to 26 for ENCALS 2026.

The ENCALS congress provides an essential venue to exchange the latest scientific discoveries and explore new avenues for collaboration in ALS research.

Our Co-founder and CEO Sam Alworth and Chief Scientific Officer Marcel van der Brug will be attending, and are excited to connect and discuss our recent breakthroughs and strategic priorities:

➡️ Advancing A*O programs designed to restore TDP-43 function
➡️ Translating unbiased screens of patient-derived motor neurons into viable therapeutic targets
➡️ Evaluating the impact of partial SYF2 suppression in TDP-43 driven ALS
➡️ Building partnerships that support the translation of early discovery into clinical progress

In April, the California Institute for Regenerative Medicine awarded AcuraStem $7.5 million to advance our lead candidate AS-241 toward a first-in-human trial, a decision that follows one of the most rigorous scientific and translational reviews in the field.

Sam and Marcel are in Madrid for those three days only…If you are attending ENCALS 2026, please reach out to connect and to learn more about our science head to acurastem.com

https://encals-congress.com/ENCALS2026

A great few days at Target ALS 2026 in Boston.

Our CSO Dr. Marcel van der Brug presented "Dose-Response of SYF2-Targeted A*Os in Diverse ALS Patient-Derived Models" the first of three talks from our Target ALS-funded consortium, "Advancing SYF2 Antisense Oligonucleotides to Restore TDP-43 Function."

Huge thanks to our consortium collaborators Dr. Philip Wong (Johns Hopkins University) and Dr. Wilfried Rossoll (Mayo Clinic), whose biomarker and interactome work complements our patient-derived dose-response studies, and to Target ALS for the funding and for convening the field.

To everyone who stopped by and engaged with the work: thank you. Those conversations sharpen the path forward.

Follow our progress at acurastem.com

AcuraStem is heading to the 2026 Target ALS Annual Meeting in Boston, May 5–7.

We're thrilled to be funded by Target ALS through the New Drug Discovery Consortia program for our SYF2 A*O project, "Advancing SYF2 Antisense Oligonucleotides to Restore TDP-43 Function," and to be presenting alongside our consortium collaborators Dr. Philip Wong (Johns Hopkins University) and Dr. Wilfried Rossoll (Mayo Clinic).

On May 5, our CSO Dr. Marcel van der Brug will give the first of the three consortium talks: "Dose-Response of SYF2-Targeted A*Os in Diverse ALS Patient-Derived Models." Dr. Wong will follow on multimodal biomarker profiling, and Dr. Rossoll will close on the SYF2-dependent TDP-43 interactome and proteostasis networks.

SYF2 emerged from an unbiased screen of patient-derived motor neurons as a top modifier of survival specifically in TDP-43 driven ALS. Together, the three projects test whether partial SYF2 suppression with our A*O can restore TDP-43 function.

Grateful to Target ALS for the support and for convening the field.

More on the science at acurastem.com

The NIH, DOD, and CIRM receive thousands of grant applications each year and fund the ones that demonstrate the strongest science. AcuraStem has received peer-reviewed grant awards from each institution across multiple funding cycles.

Those awards reflect the strength of the iNeuroRx® platform and the rigor of the science behind our UNC13A and SYF2 programs, both developed in patient-derived neurons and designed to address the biological mechanisms that drive disease in the vast majority of ALS and FTD patients, regardless of genetic background.

Learn more about our work at acurastem.com.

ALS has resisted treatment for a long time partly because the biology is not simple. When TDP-43 breaks down in a neuron, it triggers a cascade: toxic aggregates accumulate, a gene called UNC13A loses the ability to support synaptic function, and splicing errors spread across hundreds of other genes. That cascade plays out in nearly 97% of patients, familial and sporadic alike.

AcuraStem's UNC13A program targets the splicing error that cuts off a protein the synapse depends on. The SYF2 program addresses the wider splicing disruption across the hundreds of other genes that TDP-43 loss affects. Both were developed in patient-derived neurons, targeting mechanisms that animal models were never going to show us.

Read about the science behind each program at acurastem.com/therapeutics.

Why is the scientific community so focused on UNC13A?

In nearly 97% of ALS cases and roughly half of FTD cases, the loss of nuclear TDP-43 triggers a critical error in the UNC13A gene. A cryptic exon gets inserted into the messenger RNA, carrying a premature stop signal that halts production of the proteins essential for healthy synaptic communication. When those proteins disappear, neurons lose the ability to function properly and eventually degenerate.

Our team has identified A*O candidates, using the iNeuroRx® platform, designed to block the inclusion of that cryptic exon and restore the neuron's natural function. The work happens in human patient cells, which means it reflects what is actually happening in the 90% of patients whose disease has no known genetic cause.

Explore the science behind our UNC13A program at acurastem.com/therapeutics.

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