# Trace Neuroscience

**Source:** https://geo.sig.ai/brands/trace-neuroscience  
**Vertical:** BioTech  
**Subcategory:** Antisense Oligonucleotide (ALS)  
**Tier:** Emerging  
**Website:** traceneuro.com  
**Last Updated:** 2026-04-14

## Summary

Launched with $101M Series A (Third Rock, Atlas, GV, RA Capital). TRCN-1023 (ASO for UNC13A mRNA splicing) entered ALS clinical testing in early 2026 — skipping healthy volunteer phase. Targets 97% of ALS patients.

## Company Overview

Trace Neuroscience launched with $101 million in Series A financing from Third Rock Ventures, Atlas Venture, GV (Google Ventures), and RA Capital Management, developing TRCN-1023 — an antisense oligonucleotide (ASO) that corrects UNC13A mRNA splicing defects that have been genetically validated as a driver of ALS (amyotrophic lateral sclerosis). The company went directly to ALS patient enrollment in early 2026, bypassing the healthy volunteer phase typical of first-in-human trials, reflecting confidence in the mechanism's tolerability profile.

UNC13A is among the most genetically validated targets in ALS: genome-wide association studies across tens of thousands of patients have identified UNC13A variants as the primary genetic risk modifier determining ALS disease progression rate. The ASO's mechanism — correcting splicing of UNC13A mRNA to restore normal protein function — addresses a genetic pathway affecting approximately 97% of ALS patients, versus existing approved therapies that target only the ~5% of patients with specific familial mutations.

The direct patient enrollment (skipping healthy volunteer phase) is clinically significant: ALS is a rapidly fatal disease where patients cannot afford the 6-12 month timeline that healthy volunteer trials add to clinical development. Regulators support accelerated timelines for unmet-need conditions, and Trace's direct enrollment signals both regulator concurrence and strong IND safety data.

## Frequently Asked Questions

### What does Trace Neuroscience do?
Antisense oligonucleotide (TRCN-1023) correcting UNC13A mRNA splicing in ALS — targets genetic pathway affecting 97% of ALS patients vs. existing therapies covering only ~5%.

### How much has Trace raised?
$101M Series A from Third Rock Ventures, Atlas Venture, GV, and RA Capital.

### What is UNC13A and why does it matter?
GWAS studies across tens of thousands of ALS patients identified UNC13A as the primary genetic risk modifier for disease progression — the highest-validated genetic target for ALS affecting the vast majority of patients.

### Why skip the healthy volunteer phase?
ALS is rapidly fatal — patients can't afford the 6-12 month timeline of healthy volunteer trials. Direct patient enrollment reflects strong IND safety data and regulator support for expedited timelines in unmet-need conditions.

### What is UNC13A and why does it matter for ALS?
UNC13A encodes a synaptic protein that is selectively lost in ALS and FTD (frontotemporal dementia) patients carrying TDP-43 mutations — the most common genetic cause of familial ALS. UNC13A loss occurs because TDP-43 dysfunction leads to aberrant splicing of UNC13A pre-mRNA, inserting a cryptic exon that destabilizes the transcript. Trace's antisense oligonucleotides (ASOs) block this cryptic exon inclusion, restoring UNC13A protein expression and potentially slowing or halting neurodegeneration in TDP-43-positive ALS patients.

### What is Trace Neuroscience's clinical strategy?
Trace Neuroscience is pursuing a direct-to-patient Phase 1 strategy in ALS — skipping healthy volunteer dosing studies (standard for first-in-human trials) and proceeding directly to ALS patients given the disease's rapid progression and lack of effective treatments. This FDA-permitted approach for serious diseases accelerates the timeline to efficacy signals, with Phase 1 patients monitored for safety and biomarker signals (cerebrospinal fluid UNC13A protein levels, TDP-43 pathology markers) that provide early evidence of target engagement.

### How much has Trace Neuroscience raised and who are its investors?
Trace Neuroscience has raised over $100 million from neuroscience-focused investors including Atlas Venture, Samsara BioCapital, and others. The ALS therapeutic space has attracted substantial investment following tofersen's (Biogen) FDA approval for SOD1-ALS in 2023 — validating ASO-based approaches for ALS and establishing regulatory precedent for genetically stratified ALS trial design that Trace is following for its UNC13A program.

### What is the patient population for TDP-43/UNC13A-targeted ALS therapy?
Approximately 30,000 people in the US have ALS at any time, with ~5,000 new diagnoses annually. TDP-43 pathology is present in ~97% of ALS patients (not just those with TDP-43 mutations) — and UNC13A cryptic exon insertion occurs broadly in TDP-43 pathology. This means Trace's therapy, if effective, could address nearly all ALS patients rather than a small genetic subtype — a broader population than tofersen (SOD1-mutant, ~2% of ALS) — representing a potentially transformative ALS therapy market opportunity.

## Tags

healthtech, b2b

---
*Data from geo.sig.ai Brand Intelligence Database. Updated 2026-04-14.*