APRTX-001 / Aperture Therapeutics - LARVOL DELTA

Aperture Therapeutics…announced the nomination of APRTX-001 as its development candidate (PRNewswire) - "APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of Frontotemporal Dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS), with strong potential for indication expansion into Alzheimer's disease (AD). The program is now advancing through investigational new drug (IND)-enabling studies, marking a significant milestone on its path toward clinical development....The discovery and optimization of potent CD33 modulators was supported by funding from the National Institute of Neurological Disorders and Stroke (NINDS/NIH)."

New molecule • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • Dementia

Aperture Therapeutics Announces Development Candidate Targeting CD33 to Restore Microglial Homeostasis in FTD and ALS (Le Lézard) - "Aperture Therapeutics...announced the nomination of APRTX-001 as its development candidate. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of Frontotemporal Dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS), with strong potential for indication expansion into Alzheimer's disease (AD). The program is now advancing through investigational new drug (IND)-enabling studies, marking a significant milestone on its path toward clinical development."

Pipeline update • Alzheimer's Disease • Amyotrophic Lateral Sclerosis