{"id":842946,"date":"2025-04-28T17:06:56","date_gmt":"2025-04-28T21:06:56","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/camp4-to-present-new-preclinical-data-from-its-urea-cycle-disorders-and-syngap1-related-disorders-programs-at-the-28th-american-society-of-gene-and-cell-therapy-annual-meeting\/"},"modified":"2025-04-28T17:06:56","modified_gmt":"2025-04-28T21:06:56","slug":"camp4-to-present-new-preclinical-data-from-its-urea-cycle-disorders-and-syngap1-related-disorders-programs-at-the-28th-american-society-of-gene-and-cell-therapy-annual-meeting","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/camp4-to-present-new-preclinical-data-from-its-urea-cycle-disorders-and-syngap1-related-disorders-programs-at-the-28th-american-society-of-gene-and-cell-therapy-annual-meeting\/","title":{"rendered":"CAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting"},"content":{"rendered":"
\n

\n Preclinical data to showcase the potential of regRNA-targeting to increase protein levels in a clinically meaningful way by upregulating gene expression<\/em>\n <\/p>\n

\n Interim safety data including patient demographic data from all SAD cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers to be presented<\/em>\n <\/p>\n

CAMBRIDGE, Mass., April 28, 2025 (GLOBE NEWSWIRE) — CAMP4 Therapeutics Corporation (\u201cCAMP4\u201d) (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 three oral presentations at the upcoming 28th<\/sup> Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, LA, May 13 \u2013 17, 2025.<\/p>\n

\n Details for the oral presentations are as follows:<\/strong>\n <\/p>\n

Title: Targeting Regulatory RNAs with Antisense Oligonucleotides for the Potential Treatment of Urea Cycle Disorders
<\/em>Presenter: Dan Tardiff, Ph.D.
Session title: Oligonucleotide Therapeutics II
Session date and time: Friday May 16, 2025, 3:45 PM – 5:40 PM CT<\/p>\n

Title: Targeting Regulatory RNAs with Antisense Oligonucleotides for the Potential Treatment of SYNGAP1-Related Disorders
<\/em>Presenter: Dan Tardiff, Ph.D.
Session title: Oligonucleotide Therapeutics II
Session date and time: May 16, 2025, 3:45 PM – 5:40 PM CT<\/p>\n

Title: A First-in-Human Double-Blind, Placebo-Controlled Single and Multiple Ascending Dose Study (SAD, MAD) in Healthy Volunteers to Evaluate the Safety and Tolerability of an Investigational Antisense Oligonucleotide Therapy (CMP-CPS-001) for the Treatment of Urea\u00a0Cycle Diseases (UCDs): Interim Safety Readout
<\/em>Presenter: Yuri Maricich, M.D.
Session title: Oligonucleotide Therapeutics II
Session date and time: May 16, 2025, 3:45 PM – 5:40 PM CT<\/p>\n

The presentations will be made available on the CAMP4 website at https:\/\/investors.camp4tx.com\/news-events\/presentations<\/a> simultaneously with the session.<\/p>\n

\n About CAMP4 Therapeutics <\/strong>\n <\/p>\n

CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic ASO drug candidates target regulatory RNAs (regRNAs), which act locally on transcription factors and are the master regulators of gene expression.\u00a0CAMP4\u2019s proprietary RAP Platform\u2122 enables the mapping of regRNAs and generation of therapeutic candidates designed to target the regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. For more information, visit camp4tx.com<\/a>.<\/p>\n

\n Forward-Looking Statements<\/strong>\n <\/p>\n

This press release contains forward-looking statements which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as \u201cmay,\u201d \u201cwill,\u201d \u201cshould,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201canticipate,\u201d \u201ccould,\u201d \u201cintend,\u201d \u201ctarget,\u201d \u201cproject,\u201d \u201ccontemplate,\u201d \u201cbelieve,\u201d \u201cestimate,\u201d \u201cpredict,\u201d \u201cpotential\u201d or \u201ccontinue\u201d or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning CAMP4\u2019s plans and expectations regarding its ongoing Phase 1 clinical trial of CMP-CPS-001 and its intention to initiate the expansion into a Phase 1b clinical trial of CMP-CPS-001; the anticipated timing and results of the company\u2019s ongoing and future clinical trials, including expectations regarding the timing of reporting data from the CMP-CPS-001 clinical trials; the expected timing for the company\u2019s initiation of GLP toxicity studies relating to CAMP4\u2019s SYNGAP1 program; the therapeutic potential of CAMP4\u2019s product candidates; estimates regarding the size of patient populations; and cash runway guidance. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company\u2019s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company\u2019s limited operating history, incurrence of substantial losses since the Company\u2019s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company\u2019s need for substantial additional financing to achieve the Company\u2019s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company\u2019s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company\u2019s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company\u2019s ability to realize the benefits of the Company\u2019s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company\u2019s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company\u2019s dependence on the services of the Company\u2019s senior management and other clinical and scientific personnel, and the Company\u2019s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company\u2019s ability to grow the Company\u2019s organization, and manage the Company\u2019s growth and expansion of the Company\u2019s operations; risks related to the manufacturing of the Company\u2019s product candidates, which is complex, and the risk that the Company\u2019s third-party manufacturers may encounter difficulties in production; the Company\u2019s ability to obtain and maintain sufficient intellectual property protection for the Company\u2019s product candidates or any future product candidates the Company may develop; the Company\u2019s reliance on third parties to conduct the Company\u2019s preclinical studies and clinical trials; the Company\u2019s compliance with the Company\u2019s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company\u2019s product candidates; risks related to the operations of the Company\u2019s suppliers; and other risks and uncertainties described in the section \u201cRisk Factors\u201d in the Company\u2019s Annual Report on Form 10-K for the year ended December 31, 2024, as well as other information the Company files with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company\u2019s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.<\/p>\n

\n Contacts<\/strong>\n <\/p>\n

\n Investor Relations:<\/strong>\n <\/p>\n

Dan Ferry (US)
LifeSci Advisors
daniel@lifesciadvisors.com<\/a><\/p>\n

Sandya von der Weid (Europe)
LifeSci Advisors
svonderweid@lifesciadvisors.com<\/a><\/p>\n

\n Media:<\/strong>\n <\/p>\n

Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com<\/a><\/p>\n

\"\"
\n
\n \"\"\n <\/div>\n

<\/div>\n","protected":false},"excerpt":{"rendered":"

Preclinical data to showcase the potential of regRNA-targeting to increase protein levels in a clinically meaningful way by upregulating gene expression Interim safety data including patient demographic data from all SAD cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers to be presented CAMBRIDGE, Mass., April 28, 2025 (GLOBE NEWSWIRE) — CAMP4 Therapeutics Corporation (\u201cCAMP4\u201d) (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 three oral presentations at the upcoming 28th Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, LA, May … <\/p>\n

Continue reading “CAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting”<\/span><\/a><\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[],"tags":[],"class_list":["post-842946","post","type-post","status-publish","format-standard","hentry"],"yoast_head":"\nCAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting - Market Newsdesk<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.marketnewsdesk.com\/index.php\/camp4-to-present-new-preclinical-data-from-its-urea-cycle-disorders-and-syngap1-related-disorders-programs-at-the-28th-american-society-of-gene-and-cell-therapy-annual-meeting\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"CAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting - 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