Goldenrod Therapeutics Raises $6.5M in Initial Close of Series Seed Funding

The company plans to use the funding to expand manufacturing, improve its drug formulation, complete studies required for regulatory approval, and support a Phase I clinical trial for Friedreich’s Ataxia (FA), a rare and progressive neurodegenerative disease.

PDE4 is a well-known target involved in controlling inflammation in the brain and supporting nerve cell function. However, earlier PDE4 inhibitors have often been limited by side effects particularly gastrointestinal issues or by their inability to reach the brain effectively.

Goldenrod’s lead candidate, 11h, was developed by researchers at UNMC to overcome these challenges. In preclinical studies, the drug showed strong penetration into the brain, and was well tolerated, with no significant signs of nausea related side effects. Because it targets a key pathway linked to neuroinflammation and neuronal signaling, Goldenrod believes 11h could have potential across multiple neurological conditions.

Friedreich’s Ataxia (FA) is a rare inherited neurological disease that causes worsening coordination, balance problems, speech difficulties and sensory impairment over time. Although the US FDA approved the first treatment for FA in 2023 many patients continue to experience disease progression, highlighting the need for additional treatment options with different approaches.

Goldenrod sees 11h as an important first program that supports its broader efforts to develop therapies for neurological diseases. Beyond FA, the company believes the technology could have potential applications in conditions such as Alzheimer's disease, Multiple Sclerosis, ALS, Substance Use Disorders, Batten Disease, pain, and traumatic brain injury. Goldenrod expects to begin clinical development in 2027.

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“Our 11h program is a next-generation, orally bioavailable, brain-penetrant PDE4 inhibitor, where researchers overcame longstanding limitations associated with earlier PDE4 inhibitors. We believe this creates the potential for a best-in-class therapy for Friedreich’s Ataxia and a potential foundation for development across multiple neurodegenerative and neuroinflammatory disorders,” said Dev Chatterjee, MD, PhD, Chief Executive Officer of Goldenrod. The company in-licensed the technology from the University of Nebraska Medical Center (UNMC).

”We believe 11h is an important advancement in CNS-targeted PDE4 inhibition, and we’re excited to help drive this science towards a therapeutic candidate for FA,” said Tom Hamilton of Ataxia Ventures.

About Goldenrod

Founded in 2020, Goldenrod Therapeutics is a Fannin founded biotechnology company developing new treatments for neurological and neurodegenerative diseases. The company focuses on brain-penetrant therapies that target inflammation and signaling pathways in the central nervous system. Its lead drug candidate, 11h, is a small molecule PDE4 inhibitor designed to address significant unmet medical needs through innovative treatments for disorders affecting the brain and nervous system.

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