{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T03:08:06Z","timestamp":1779073686251,"version":"3.51.4"},"reference-count":14,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,4,25]],"date-time":"2026-04-25T00:00:00Z","timestamp":1777075200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,4,25]],"date-time":"2026-04-25T00:00:00Z","timestamp":1777075200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Translational Sci"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Nomlabofusp is a recombinant, cell\u2010penetrating human frataxin (hFXN) fusion protein in development for the treatment of Friedreich's ataxia (FRDA). This study evaluated whether nomlabofusp\u2010derived hFXN concentrations covary across accessible peripheral matrices and FRDA\u2010relevant tissues, supporting the feasibility of surrogate tissue sampling to monitor drug\u2010derived hFXN exposure. Following subcutaneous administration in mice, rats, non\u2010human primates, and patients with FRDA, we quantified nomlabofusp\u2010derived hFXN and assessed cross\u2010tissue correlations across target tissues (brain, heart, skeletal muscle, dorsal root ganglia) and accessible peripheral matrices (skin, buccal cells, platelets). We observed significant and consistent cross\u2010tissue correlations, with concordant relationships among heart, skeletal muscle, dorsal root ganglia, skin, buccal cells, liver, and mitochondrial fractions, indicating coordinated distribution and\/or retention of nomlabofusp\u2010derived hFXN across these matrices. Correlation patterns were maintained across species, supporting the robustness and translational relevance of the observed relationships. Collectively, these data support the use of peripheral tissues\u2014particularly skin and buccal cells\u2014for cross\u2010sectional and longitudinal monitoring of hFXN supplementation in FRDA.<\/jats:p>","DOI":"10.1111\/cts.70565","type":"journal-article","created":{"date-parts":[[2026,4,25]],"date-time":"2026-04-25T07:40:53Z","timestamp":1777102853000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Nomlabofusp Treatment Produces Frataxin Levels That Correlate Across Peripheral Tissues: Preclinical and Clinical Support for Surrogate Tissue Sampling"],"prefix":"10.1111","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6701-7710","authenticated-orcid":false,"given":"Flavia","family":"De Toni","sequence":"first","affiliation":[{"name":"Larimar Therapeutics Inc.  Bala Cynwyd Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-5899-294X","authenticated-orcid":false,"given":"Mohamed","family":"Hamdani","sequence":"additional","affiliation":[{"name":"Larimar Therapeutics Inc.  Bala Cynwyd Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-3588-0293","authenticated-orcid":false,"given":"Chintan","family":"Patadia","sequence":"additional","affiliation":[{"name":"Larimar Therapeutics Inc.  Bala Cynwyd Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-7224-1201","authenticated-orcid":false,"given":"Gopi","family":"Shankar","sequence":"additional","affiliation":[{"name":"Larimar Therapeutics Inc.  Bala Cynwyd Pennsylvania USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2026,4,25]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41419-023-06320-y"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-019-09989-y"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2019.00075"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1080\/21688370.2025.2462357"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2010.07.001"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.03.019"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/6.11.1771"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0017627"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0063958"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1208\/s12248-025-01054-5"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51971"},{"key":"e_1_2_9_13_1","article-title":"Effect of Nomlabofusp Administration on Tissue Frataxin Levels, Plasma Lipid Profiles, and Gene Expression in Patients With Friedreich's Ataxia","author":"Scherer N.","year":"2024","journal-title":"International Congress for Ataxia Research"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1208\/s12248-025-01093-y"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/nprot.2006.478"}],"container-title":["Clinical and Translational Science"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/cts.70565","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/cts.70565","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/cts.70565","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T02:54:09Z","timestamp":1779072849000},"score":1,"resource":{"primary":{"URL":"https:\/\/ascpt.onlinelibrary.wiley.com\/doi\/10.1111\/cts.70565"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,25]]},"references-count":14,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026,5]]}},"alternative-id":["10.1111\/cts.70565"],"URL":"https:\/\/doi.org\/10.1111\/cts.70565","archive":["Portico"],"relation":{},"ISSN":["1752-8054","1752-8062"],"issn-type":[{"value":"1752-8054","type":"print"},{"value":"1752-8062","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,25]]},"assertion":[{"value":"2025-12-18","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-27","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-25","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70565"}}