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

Rhea-AI Summary

CAMP4 Therapeutics (Nasdaq: CAMP) has announced three oral presentations at the upcoming 28th Annual Meeting of the American Society of Gene and Cell Therapy in New Orleans, May 13-17, 2025.

The presentations will showcase:

• Preclinical data on regulatory RNA-targeting to increase protein levels for gene expression
• Interim safety data from Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers
• Research on potential treatments for Urea Cycle Disorders and SYNGAP1-Related Disorders

Three key presentations by the company include:

• Two presentations by Dr. Dan Tardiff on targeting regulatory RNAs for treating Urea Cycle Disorders and SYNGAP1-Related Disorders
• One presentation by Dr. Yuri Maricich on the first-in-human clinical trial results of CMP-CPS-001

All presentations will take place on May 16, 2025, during the Oligonucleotide Therapeutics II session and will be available on the company's investor website.

Positive

• Three oral presentations scheduled at major scientific conference (ASGCT) demonstrating company's progress in regulatory RNA therapeutics
• Presentation of interim safety data from Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers
• Development of potential treatments for multiple diseases: Urea Cycle Disorders and SYNGAP1-Related Disorders
• Company has advanced to clinical stage with first-in-human trials

Negative

• Only preclinical data available for most programs
• Safety data is interim, not final results
• Limited to early-stage clinical trials (Phase 1) with no efficacy data presented yet

Insights

Biotechnology/Pharmaceutical Analyst

CAMP4's conference presentations will reveal interim Phase 1 safety data and preclinical results, showing progress in their regulatory RNA platform development.

CAMP4's upcoming presentations at the ASGCT conference represent meaningful progress in their clinical and preclinical programs. The company will present interim safety data from their first-in-human Phase 1 trial of CMP-CPS-001 in healthy volunteers, which marks an important milestone in the development of their lead program targeting Urea Cycle Disorders (UCDs). This data will include patient demographics from all Single Ascending Dose cohorts, providing a first look at the safety profile of their regulatory RNA-targeting approach.

Additionally, two preclinical presentations will showcase their technology platform's potential applications in both Urea Cycle Disorders and SYNGAP1-Related Disorders, demonstrating the versatility of their approach across different genetic conditions. Their technology aims to upregulate gene expression by targeting regulatory RNAs, potentially restoring healthy protein levels in diseases where protein production is insufficient.

These oral presentations—which typically undergo competitive selection—will provide valuable scientific validation of their approach. For investors, this represents continued execution on CAMP4's development roadmap, though the actual impact will depend on the data revealed during the presentations in May. The selection of multiple presentations at this prominent gene therapy conference suggests scientific interest in their novel regulatory RNA-targeting platform.

Clinical Research Expert

Interim Phase 1 safety data from CAMP4's regulatory RNA therapy represents an important de-risking milestone for their novel therapeutic approach.

The presentation of interim safety data from CAMP4's first-in-human Phase 1 trial of CMP-CPS-001 represents a significant step in validating their regulatory RNA-targeting platform. This data from the Single Ascending Dose (SAD) cohorts will provide the first clinical insights into the safety and tolerability profile of their approach, which is designed to increase protein levels by upregulating gene expression.

For novel therapeutic modalities like antisense oligonucleotides targeting regulatory RNAs, establishing safety in healthy volunteers is a crucial de-risking step before advancing to patient populations. The preclinical data being presented alongside the clinical results will help contextualize the mechanism of action, demonstrating how targeting regulatory RNA can increase protein levels in a clinically meaningful way.

The focus on Urea Cycle Disorders and SYNGAP1-Related Disorders highlights CAMP4's strategy of addressing genetic diseases where insufficient protein production is the underlying cause. These conditions have limited treatment options, making new therapeutic approaches particularly valuable. While this data represents early clinical development, it provides important validation of their platform technology and development approach as they progress toward potential proof-of-concept in patients.

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 (“CAMP4”) (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 28^th Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, LA, May 13 – 17, 2025.

Details for the oral presentations are as follows:

Title: Targeting Regulatory RNAs with Antisense Oligonucleotides for the Potential Treatment of Urea Cycle Disorders
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

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

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 Cycle Diseases (UCDs): Interim Safety Readout
Presenter: Yuri Maricich, M.D.
Session title: Oligonucleotide Therapeutics II
Session date and time: May 16, 2025, 3:45 PM - 5:40 PM CT

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

About CAMP4 Therapeutics

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. CAMP4’s proprietary RAP Platform™ 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.

Forward-Looking Statements

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 “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” 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’s 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’s 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’s initiation of GLP toxicity studies relating to CAMP4’s SYNGAP1 program; the therapeutic potential of CAMP4’s 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’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s 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’s 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’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s 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’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers; and other risks and uncertainties described in the section “Risk Factors” in the Company’s 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’s 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.

Contacts

Investor Relations:

Dan Ferry (US)
LifeSci Advisors
daniel@lifesciadvisors.com

Sandya von der Weid (Europe)
LifeSci Advisors
svonderweid@lifesciadvisors.com

Media:

Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com

FAQ

What will CAMP4 (CAMP) present at the 2025 American Society of Gene and Cell Therapy Meeting?

CAMP4 will present three oral presentations on May 16, 2025, including preclinical data on treating Urea Cycle Disorders and SYNGAP1-Related Disorders, plus interim safety data from their Phase 1 trial of CMP-CPS-001 in healthy volunteers.

When and where is CAMP4's presentation at the 2025 ASGCT Meeting?

CAMP4's presentations will take place on Friday, May 16, 2025, from 3:45 PM to 5:40 PM CT in New Orleans, LA, during the Oligonucleotide Therapeutics II session.

What diseases is CAMP4 (CAMP) targeting with their regulatory RNA therapy?

CAMP4 is targeting Urea Cycle Disorders (UCDs) with their CMP-CPS-001 therapy and SYNGAP1-Related Disorders through their regulatory RNA-targeting therapeutics program.

How can investors access CAMP4's ASGCT 2025 presentation materials?

Investors can access CAMP4's presentation materials on their website at https://investors.camp4tx.com/news-events/presentations simultaneously with the session on May 16, 2025.

What clinical trial data will CAMP4 (CAMP) present at ASGCT 2025?

CAMP4 will present interim safety data and patient demographics from all Single Ascending Dose (SAD) cohorts of their first-in-human Phase 1 trial of CMP-CPS-001 in healthy volunteers.