Pioneering precision medicines for high-need CNS disorders

  • Two lead programs: an ASO for sporadic and genetic ALS patients targeting lipid biology, and an oral small molecule for schizophrenia targeting glutamate biology
  • Lead programs on track for INDs this year, with clinical data in 2025
  • Additional pipeline programs in Alzheimer’s disease, epilepsy, and depression

Pipeline

Program

Target

Indication

Modality

Leads

Preclinical

DC

IND-Enabling

Clinical

LTX-002

SPTLC1 /

Lipid metabolism

ALS, Peripheral Neuropathy, Alzheimer’s Disease
ASO

LTX-001

GLS1 /

Glutamate presynaptic

Schizophrenia, MDD, ALS

Small molecule

LTX-003

Complement

Alzheimer’s Disease, FTD, AMD

ASO

LTX-004

Lipid trafficking
Alzheimer’s Disease, Dementia with Lewy bodies
ASO

LTX-005

GRIN2A /

Glutamate NMDAR

Refractory Epilepsy, MDD/TRD

ASO

Pipeline

Program: LTX-002

Modality:  ASO

Target: SPTLC1 / Lipid metabolism

Indication: ALS, Peripheral Neuropathy, Alzheimer’s Disease

Leads

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-001

Modality:  Small molecule

Target:  GLS1 / Glutamate presynaptic

Indication: Schizophrenia, MDD, 

ALS

Leads

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-003

Modality:  ASO

Target: Complement

Indication: Alzheimer’s Disease, FTD, AMD

Leads

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-004

Modality:  ASO

Target:  Lipid trafficking

Indication: Alzheimer’s Disease, Dementia with Lewy bodies

Leads

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-005

Modality:  ASO

Target:  GRIN2A / Glutamate NMDAR

Indication: Refractory Epilepsy, MDD/TRD

Leads

Pre-clinical

DC

IND-enabling

Clinical

Developing novel precision CNS therapeutics that target key pathogenic pathways