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Aperture Therapeutics, based in San Carlos, California, has announced the nomination of a new development candidate, known as APRTX-001, designed to target CD33 for the treatment of neurodegenerative diseases, specifically Frontotemporal Dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS). This announcement, made on November 19, 2025, represents a noteworthy advancement in the company’s efforts to develop precision medicines for conditions that currently lack effective treatment options.
APRTX-001 is an antisense oligonucleotide (ASO) that aims to mimic the protective effects of natural variants of CD33 loss-of-function. These variants have been linked to increased resilience against neurodegenerative diseases, reduced neuroinflammation, and lower levels of biomarkers associated with neural aging. The drug is entering the investigational new drug (IND)-enabling studies phase, marking a critical step toward clinical trials.
Aperture’s approach focuses on the role of CD33 in regulating neuroinflammatory signaling. Elevated levels of CD33 have been observed in tissues from patients with ALS, FTD, Alzheimer’s Disease (AD), and Parkinson’s Disease (PD). By inhibiting CD33, APRTX-001 seeks to restore microglial homeostasis and reduce chronic neuroinflammation, which is crucial for maintaining brain health.
Dr. Martin Jacko, Founder and CEO of Aperture Therapeutics, emphasized the challenges associated with previous attempts to target CD33 using small molecules or antibodies. “Prior attempts have failed due to modality-associated challenges and poor translation in preclinical models,” he stated. “APRTX-001 overcomes these historical barriers with a precision approach grounded in human genetics.”
To facilitate the development of APRTX-001, Aperture Therapeutics has created a proprietary humanized CD33 knock-in mouse model. This model enables more relevant target validation and therapeutic testing, ensuring that results are applicable to human conditions. The development of potent CD33 modulators has also been supported by funding from the National Institute of Neurological Disorders and Stroke (NINDS/NIH).
In addition to APRTX-001, Aperture Therapeutics is pursuing other programs targeting key mechanisms involved in neuroinflammation and neurodegeneration. The company is also working on a microglia-specific drug delivery technology, aimed at enhancing the precision of its therapeutics in treating neuroinflammatory conditions.
Founded in 2022, Aperture Therapeutics is dedicated to developing innovative oligonucleotide therapies inspired by human genetic resilience. The company utilizes a proprietary platform that integrates multi-omics analyses and machine-learning-guided ASO design to identify and validate clinically relevant targets. Aperture has gained financial backing from various investors, including Mission BioCapital, Age1, and Tensor Ventures, among others.
For more information about Aperture Therapeutics and its ongoing research, visit their official website or follow them on LinkedIn.
