Startup aims for clinical trials of microglia-targeting FTD treatment that ‘reprograms’ the brain’s immune system into a protective state.
Neuro-focused biotech startup Modulo Bio has secured a strategic investment of $4.8 million in funding from the Alzheimer’s Drug Discovery Foundation (ADDF) to advance the development of its drug candidate for the treatment of frontotemporal dementia (FTD).
San Diego-based Modulo is on a mission to harness the neuroimmune system to develop treatments for neurodegenerative diseases. Its lead drug candidate, a small molecule therapeutic called MOD 001, is being evaluated in FTD and other conditions, including and amyotrophic lateral sclerosis (ALS). The company says it will use the new funding to fund the development of the drug through Investigational New Drug (IND) enabling studies, prior to conducting first-in-human clinical trials.
Modulo was founded to leverage emerging understanding of the neuroimmune system’s role in brain health. The neuroimmune system, composed of specialized immune cells in the brain called microglia, plays a critical role in protecting and repairing neurons. Microglia can switch between beneficial and harmful states, either supporting neural repair or contributing to further degeneration. Modulo’s platform is designed to harness and reprogram microglia to shift them toward a protective state, potentially halting or reversing disease progression.
MOD 001 specifically targets a protein called CSF1R, which plays a key role in the development, maintenance and activation of microglia. Modulo says MOD 001 shows “exceptional” blood-brain barrier penetration and claims its preclinical studies have demonstrated the drug’s potential to reduce neuroinflammation and slow disease progression.
“The ADDF’s endorsement strongly validates our focus on combating neuroinflammation in degenerative brain disorders,” said Modulo CEO Michael Horowitz. “Their scientific team has conducted thorough due diligence on MOD 001 as a potential treatment for FTD, a devastating early-onset form of dementia that currently lacks any disease-modifying therapies. Based on their comprehensive review, the ADDF shares our interest in MOD 001’s potential to make a meaningful difference for FTD patients.”
The ADDF aims to accelerate the development of treatments designed to prevent, treat and cure Alzheimer’s disease and related dementias, including FTD.
“The ADDF has a long history of supporting and investing in novel and innovative approaches in the fight against neurodegenerative diseases,” said Dr Howard Fillit, Chief Science Officer at the ADDF. “Modulo Bio’s novel approach to targeting CSF1R and modulating microglial function is one of the many exciting new approaches in therapeutic development for FTD and ALS.”
Modulo’s approach begins with patient-derived cells. Using advanced stem cell technology, Modulo generates brain cells from patients with different neurodegenerative diseases. These cells are integrated into experimental models of the neuroimmune system, including a triculture model that combines microglia, astrocytes and neurons. This allows researchers to observe microglial behavior in a controlled environment and test various genetic and chemical interventions.
Central to the platform is the ability to generate an AI-derived neuroimmune fingerprint of disease. By combining experimental results with machine learning, Modulo identifies patterns in microglial behavior and correlates them with disease progression and neuroprotection. The platform’s machine learning capabilities help analyze complex datasets, revealing which biological targets influence microglia and how different microglial states impact brain health.
Modulo’s drug discovery process focuses on identifying compounds that enhance microglial neuroprotection. By understanding how microglia respond to different interventions, the company aims to develop drugs that shift microglia into a protective state.
Photograph: digitalstormcinema/Envato