This section highlights FDA-related milestones and regulatory updates for drugs developed by CAMP4 Therapeutics (CAMP).
Over the past two years, CAMP4 Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
CMP-SYNGAP-01 and CPS-001. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
CMP-SYNGAP-01 - FDA Regulatory Timeline and Events
CMP-SYNGAP-01 is a drug developed by CAMP4 Therapeutics for the following indication: Treatment rescued multiple SYNGAP1-dependent behavioral phenotypes.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CMP-SYNGAP-01
- Announced Date:
- May 16, 2025
- Indication:
- Treatment rescued multiple SYNGAP1-dependent behavioral phenotypes
Announcement
CAMP4 Therapeutics Corporation , today delivered three oral presentations on its SYNGAP1-related disorders and Urea Cycle Disorders (UCDs) programs and shared favorable safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001 in healthy volunteers at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, May 13 – 17, 2025.
AI Summary
CAMP4 Therapeutics Corporation recently presented three oral talks at the 28th Annual Meeting of the American Society of Gene and Cell Therapy in New Orleans (May 13–17, 2025). The presentations focused on the company’s programs targeting SYNGAP1-related disorders and Urea Cycle Disorders, highlighting innovative approaches to genetic diseases with limited treatment options.
CAMP4 shared promising results from its Phase 1 trial of CMP-CPS-001 in healthy volunteers, where the drug showed favorable safety and pharmacokinetics. In addition, data from the SYNGAP1 program demonstrated that CMP-SYNGAP-01 effectively increased SYNGAP1 protein levels in both haploinsufficient mice and non-human primates, improving disease-related behaviors. These findings underline CAMP4’s potential in using regulatory RNA-targeting strategies to upregulate gene expression, aiming to deliver much-needed disease-modifying therapies for patients with genetic disorders.
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CPS-001 - FDA Regulatory Timeline and Events
CPS-001 is a drug developed by CAMP4 Therapeutics for the following indication: For the treatment of urea cycle disorders.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CPS-001
- Announced Date:
- January 7, 2025
- Indication:
- For the treatment of urea cycle disorders
Announcement
CAMP4 Therapeutics provided corporate updates and key objectives for 2025.
AI Summary
CAMP4 Therapeutics has outlined key corporate updates and objectives for 2025. The company, which had a transformative 2024 following its successful IPO, is moving forward with strong momentum into the new year. CAMP4 is encouraged by the safety results from the Single Ascending Dose segment of its Phase 1 study for CMP-CPS-001, a treatment for urea cycle disorders. The company plans to report Multiple Ascending Dose safety, pharmacokinetic, and key pharmacodynamic biomarker data in the second half of 2025. These data are expected to help move the CMP-CPS-001 program into a registrational Phase 2/3 trial in 2026.
CAMP4 is also starting a new discovery program targeting a GBA1 regRNA aimed at increasing gene expression for the treatment of Parkinson’s disease. The company will continue expanding its strategic partnerships and capitalizing on its RAP platform to drive future development.
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