The molecule was created by chemist Corey Hopkins, PhD, at the University of Nebraska Medical Center College of Pharmacy. UNeMed, UNMC's technology transfer office, helped establish Goldenrod in 2023 and put the licence in place. In June 2026 the company closed a $6.5M seed round led by Ataxia Ventures and an affiliate of Fannin Partners to complete the studies needed for clinical trials planned for 2027.
Sunday, October 11, 2026
Goldenrod Therapeutics: brain-penetrant PDE4 inhibitor from UNMC
Goldenrod Therapeutics is developing 11h, a brain-penetrant inhibitor of the inflammatory enzyme PDE4, for neurological diseases driven by brain inflammation, with Friedreich's ataxia as an initial target. Existing PDE4 drugs are limited by nausea and poor uptake into the central nervous system, which 11h was designed to overcome.
Saturday, October 10, 2026
Compound Heterozygote Friedreich Ataxia Patients With Covert Proximal FXN Gene Deletions
M. P. Lazaropoulos, M. C. Devore, C. Lam, C. Park, S. Bidichandani, and D. R. Lynch, “ Compound Heterozygote Friedreich Ataxia Patients With Covert Proximal FXN Gene Deletions,” Annals of Clinical and Translational Neurology 13, no. 10 (2026): 2134–2137, doi:10.1002/acn3.70408.
We present Friedreich ataxia patients with frataxin gene deletions. Data and records were collected at the Children's Hospital of Philadelphia from patients enrolled in the FACOMS natural history study.
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