Business
CAMP4 Therapeutics Initiates GLP Toxicology Studies for CMP-SYNGAP-01
CAMBRIDGE, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4” or “the Company”) (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today announced the initiation of toxicology studies conducted under Good Laboratory Practice (GLP) standards for its lead product cand
About this update from Camp4 Therapeutics Corporation
[{"type":"image","alt":"CAMP4 Therapeutics","displaySize":"","headline":null,"caption":"CAMP4 Therapeutics","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":94,"url":"https://media.zenfs.com/en/globenewswire.com/21702dc1d4660b70780cbc865b3cc20b"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/FOgCfQCojUOWCCToPToW_A--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTEzMjtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/21702dc1d4660b70780cbc865b3cc20b","width":300,"height":94}},"lazy":false},{"type":"text","content":"CAMBRIDGE, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4” or “the Company”) (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today announced the initiation of toxicology studies conducted under Good Laboratory Practice (GLP) standards for its lead product candidate, CMP-SYNGAP-01. These studies will support the Company’s planned submission of a clinical trial application, which could enable the initiation of a first-in-human Phase 1/2 clinical trial in SYNGAP1-related disorders as early as the second half of 2026.","length":764,"tagName":"p"},{"type":"text","content":"“CMP-SYNGAP-01 has demonstrated robust preclinical activity, both restoring SYNGAP1 protein levels to improve phenotypes in a mouse model and increasing SYNGAP1 protein in brain regions of non-human primates that are affected in human disease,” said Daniel Tardiff, Ph.D., Chief Scientific Officer of CAMP4. “Our preclinical data gives us confidence in the potential of CMP-SYNGAP-01 to translate into meaningful clinical benefit for patients living with SYNGAP1-related disorders, and advancing our candidate through GLP toxicology studies marks a critical step towards the clinic.”","length":583,"tagName":"p"},{"type":"text","content":"SYNGAP1-related disorders are a group of neurodevelopmental conditions caused by pathogenic variants in the SYNGAP1 gene that result in a haploinsufficient state, reducing SYNGAP protein levels by up to 50%. SYNGAP plays a critical role in cognitive development and synaptic function. SYNGAP-related disorders are reported to represent 0.5% to 1.0% of all intellectual disability cases...