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About Stitch

Stitch is a next-generation therapeutics company pioneering antisense oligonucleotide (ASO) medicines that restore normal RNA splicing disrupted by TDP-43 proteinopathy,  a central mechanism underlying ALS, frontotemporal dementia (FTD), Alzheimer’s Disease (AD), and related neurodegenerative diseases.

Our mission is to repair the transcriptome—to “stitch” the genome’s broken messages back together—by correcting cryptic exon (CE) inclusion events that arise when TDP-43 function is lost. Through precision molecular design, we create ASOs that block CE insertions, restore normal protein expression, and slow or reverse neuronal dysfunction.

Our Therapeutic Focus

Platform Approach

Modular ASO chemistry with optimized modifications, enabling high CNS distribution, potency, and durability.

Lead Program: STIX-01

an antisense oligonucleotide targeting a key cryptic exon inclusion event in ALS and FTD, designed to restore normal neuronal splicing and preserve synaptic integrity and function.

Translational Biomarkers

Our therapeutic program are powered by quantitative biomarker science that defines molecular signatures of TDP-43 dysfunction in human brain tissues, CSF, and plasma.

Our biomarker platform enables:

  • Identification of cryptic exon splicing signatures that reflect TDP-43 loss-of-function.
  • CSF and plasma proteomic markers that mirror TDP-43 loss of function and transcript-level rescue following ASO treatment.
  • Pharmacodynamic assays that guide dose optimization and demonstrate target engagement in clinical trials.

A Precision Medicine Engine for ALS

By integrating ASO chemistry, RNA biology, and proteomic biomarkers, Stitch is redefining how neurodegenerative diseases are treated.

Our precision-medicine framework links:

  1. Mechanism — TDP-43-dependent RNA splicing dysregulation
  2. Molecule — Cryptic-exon-correcting ASOs
  3. Measurement — Biomarkers that track functional rescue in the brain and periphery

This end-to-end approach enables rational target selection, rapid preclinical translation, and biomarker-anchored clinical development.

The Vision

Stitch is building a future in which the underlying molecular pathology of ALS and other TDP-43 disorders can be corrected at its source — restoring the fidelity of gene expression and the health of neurons.

Stitching the transcriptome back together. Restoring function. Transforming lives.