Our Pipeline

Pipeline

A platform approach advancing multiple programs across neurological diseases, beginning with Huntington’s disease.

Development pipeline
Disease Area Target (Asset) Platform Technology
Progress (stages: Discovery, IND Enabling, Phase 1)
Ownership
Huntington's Disease HTT (ATL-101) di-siRNA
Stage: end of IND Enabling Phase, beginning of Phase 1
MSH3 di-siRNA
Stage: IND Enabling Phase
HTT + MSH3 di-siRNA
Stage: Discovery
Tauopathies MAPT di-siRNA
Stage: Discovery
Synuclein­opathies SNCA di-siRNA
Stage: Discovery
CNS Disorders Multiple undisclosed internal targets Undisclosed
Stage: Discovery
Next-Generation Platform Undisclosed bi-siRNA Brain Shuttle
Stage: Discovery
Partnered Programs
CNS Disorders Multiple undisclosed targets Undisclosed
Stage: Discovery

Huntington's Disease

HTT (ATL-101) di-siRNA
Stage: end of IND Enabling Phase, beginning of Phase 1
MSH3 di-siRNA
Stage: IND Enabling Phase
HTT + MSH3 di-siRNA
Stage: Discovery

Tauopathies

MAPT di-siRNA
Stage: Discovery

Synuclein­opathies

SNCA di-siRNA
Stage: Discovery

CNS Disorders

Multiple undisclosed internal targets Undisclosed
Stage: Discovery

Next-Generation Platform

Undisclosed bi-siRNA Brain Shuttle
Stage: Discovery

Partnered Programs

CNS Disorders

Multiple undisclosed targets Undisclosed
Stage: Discovery
Learn more about our approach

Huntington’s disease

Programs targeting complementary drivers of Huntington’s disease

Arexera is advancing programs directed at HTT, the primary cause of Huntington’s disease, and MSH3, a modifier associated with somatic CAG repeat expansion and disease progression.

ATL-101

Targeting the underlying cause of Huntington’s disease

ATL-101 is an investigational di-siRNA therapeutic designed to reduce HTT expression across the CNS regions affected by Huntington’s disease.

Arexera is moving ahead with regulatory filing for ATL-101 and intends to initiate first-in-human clinical development by the end of 2026, with initial clinical data anticipated in 2027.

ARX-MSH3

Targeting a key driver of disease progression

Arexera is advancing a second Huntington’s disease program targeting MSH3, a modifier associated with the continued expansion of the disease-causing CAG repeat.

The program is designed to complement HTT lowering by addressing an additional mechanism involved in Huntington’s disease progression.

Broader pipeline

Arexera is applying its platforms to additional targets implicated in neurological disease, including MAPT in tauopathies and SNCA in synucleinopathies.

ARX-MAPT

Target: MAPT

Indication / disease area: Tauopathies
Stage: Discovery

A discovery-stage program targeting MAPT, a central driver of tau-associated neurodegenerative disease.

ARX-SNCA

Target: SNCA
Indication / disease area: Synucleinopathies
Stage: Discovery

A discovery-stage program targeting SNCA, which is implicated in synucleinopathies.