We are a clinical-stage biopharmaceutical company pioneering the discovery and development of regulatory RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Regulatory RNAs, or regRNAs, play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA, or mRNA, expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our proprietary RNA Actuating Platform, or RAP Platform, allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide, or ASO, candidates, which we also refer to as RNA Actuators. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way. We are initially focused on metabolic and central nervous system, or CNS, diseases with validated disease biology, and we believe our RAP Platform allows us to address a broad range of genetic diseases in which a modest increase in protein expression can be clinically meaningful. Based on our preclinical studies, we believe our lead product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of the most prevalent urea cycle disorders, or UCDs. UCDs are a group of severe, inherited metabolic diseases caused by mutations in the genes that encode one or more of the eight enzymes and transporters necessary to convert ammonia into urea. The inability of the body to properly metabolize ammonia leads to the accumulation of toxic levels in circulation, ultimately resulting in severe health outcomes, such as neurologic disability, seizure and death. CMP-CPS-001 is designed to improve urea cycle activity by amplifying expression of carbamoyl phosphate synthetase 1, or CPS1, an enzyme that catalyzes the first step of the urea cycle, by binding to a CPS1-specific regRNA. Our preclinical studies have demonstrated that modulating the activity of the target regRNA increases expression of the CPS1 gene, resulting in increased CPS1 enzyme levels, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges. These preclinical studies also demonstrated that CMP-CPS-001 can increase the level of, or upregulate, the production of multiple enzymes responsible for converting ammonia into urea, potentially allowing us to address more than 85% of patients with UCDs, which we refer to as our pan-UCD approach. We are in the early stages of development and are evaluating CMP-CPS-001 in an ongoing Phase 1 clinical trial in healthy volunteers and expect to report data from all four cohorts of the single ascending dose, or SAD, portion of the trial in the first quarter of 2025 and from the multiple ascending dose, or MAD, portion of the trial in the second half of 2025. We are also leveraging our RAP Platform to advance our first preclinical program for the treatment of synaptic Ras GTPase activating protein 1, or SYNGAP1,-related disorders. We expect to initiate final Good Laboratory Practice, or GLP, toxicology studies in our SYNGAP1 program in 2025 to enable the filing of clinical trial applications. The transcription of DNA into mRNA, the molecular template that is then translated into protein, is a complex yet carefully coordinated cellular process involving numerous components. Only a small portion of the DNA in the human genome is transcribed into RNA that codes for proteins. The vast majority of the transcriptome originates from non-coding regions of DNA, a portion of which, referred to as enhancers and promoters, perform a crucial role in determining the specificity, timing and level at which a particular gene is expressed. RegRNAs are non-coding RNAs that are transcribed by these enhancer and promoter DNA regions that form localized complexes with transcription factors to control the expression of protein-coding genes, either increasing or decreasing their expression within natural physiological ranges. The approximately 20,000 genes that code for mRNA in the human genome are controlled by hundreds of thousands of DNA enhancers and their associated regRNAs. Deficient protein levels characterize over a thousand diseases. Haploinsufficient diseases are dominantly inherited conditions in which inadequate gene expression is driven by a mutation in a single allele, or gene copy, and results in reductions of protein levels by as much as 50%. Numerous other genetic conditions are caused by recessive mutations that result in diminished gene activity. Data from our preclinical studies and research reports published by third parties demonstrate that increasing expression of disease-associated genes by modest amounts can restore healthy protein levels and provide therapeutic benefit in these disorders. Therefore, modest increases in protein expression have the potential to be clinically meaningful in both haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200. Our RAP Platform has the potential to identify the regRNA associated with all of these diseases, which we believe enables us to design RNA Actuators to address the underlying biology of these diseases. We aim to leverage our RAP Platform to develop product candidates designed to regulate transcription in a gene-specific manner to restore healthy protein levels and remedy these diseases. However, our approach is unproven and may not lead to successful efforts to develop and commercialize our product candidates and to identify and discover additional potential product candidates. Our RAP Platform We believe our RAP Platform can unlock the potential of the human genome and have broad applications across a range of diseases caused by sub-optimal levels of protein expression. Our technology is based upon the pioneering work in transcription regulation conducted by our co-founders, Richard Young, PhD and Leonard Zon, MD. We have built our RAP Platform to identify and characterize every regRNA that controls protein-coding genes and to develop novel ASO-based therapeutics to modulate regRNA activity to increase the expression of protein-coding genes of interest and thereby address the underlying cause of genetic diseases. Based on our proprietary mapping of regRNAs and screening and optimizing of ASOs, we have established a leadership position in regRNA-targeting therapies. Our goal is to be the preeminent company focused on discovering, developing and delivering regRNA-targeting therapeutics to patients. We believe that the ability to upregulate genes selectively through targeting regRNA could provide a new way to treat a wide range of human diseases and has the potential to become a class of new medicines. At present, very few regRNAs are described in public genomic databases, as they are often expressed at low levels and their importance was not fully understood. Our RAP Platform utilizes next-generation sequencing technologies and custom sequence analyses to map the active regulatory elements controlling every expressed gene. These data empower our proprietary machine learning algorithm, known as EPIC, to identify the specific control elements that regulate any gene of interest in the most specific manner, including elements that may restrict gene expression to a particular cell type. This enables us to identify the exact sites of regRNA synthesis and ultimately map the complete sequence of every candidate regRNA to target for therapeutic gene control. To date, we have mapped multiple cell types in as little as three months, comprising a number of potentially addressable diseases in the liver, CNS, heart, skeletal muscle and immune system. Our in-house development and application of this technology has enabled us to identify tens of thousands of enhancer and promoter regRNA sequences and their key biological properties, resulting in what we believe to be the most robust regRNA dataset available. We combine our RAP Platform with ASO chemistry that has been utilized and validated in U.S. Food and Drug Administration, or FDA,-approved products to develop programmable RNA Actuators that are designed to precisely upregulate gene expression at the transcriptional level. Once a target gene is nominated, our RAP Platform rapidly identifies the controlling regRNA sequence, and we perform ASO screens to identify regions where ASO binding results in optimal upregulation of that target gene. Further rational design is applied to the ASOs identified in the screen. Our proprietary technology enables us to design RNA Actuators that optimize for specificity by avoiding binding to regRNAs that act on more than one gene and any other similar sequences found elsewhere in the transcriptome. As a result, our sequence-specific approach enables us to precisely target regRNA transcripts to increase gene expression. Our approach is designed to enable the efficient and systematic creation of RNA Actuators to target regRNAs of interest. Building upon the power of this technology, our RNA Actuators can be programmed to engage regRNA targets, producing tunable increases in protein expression. While other ASOs have received regulatory approval, no regulatory authorities to date have approved ASOs that are directed towards regRNAs and, as a result, there is uncertainty as to the safety and efficacy profile of our product candidates compared to currently approved ASOs. --- We design RNA Actuators to leverage existing oligonucleotide delivery approaches to enable drug delivery to specific types of tissues throughout the body. We believe our RAP Platform can address any disease where a modest increase in protein expression has the potential to be clinically meaningful, including haploinsufficient diseases or recessive loss-of-function diseases. Furthermore, as we continue to map regRNAs and conduct ASO screens in more cell types, the data generated will improve the algorithms we use to identify the candidate regRNAs to specifically control gene expression. We believe the knowledge and learnings from our initial programs will significantly expedite selection of lead candidates and position us to rapidly expand our pipeline. We were originally incorporated under the laws of the State of Delaware in 2015 under the name Marauder Therapeutics, Inc. and began operations in 2016. We changed our name to CAMP4 Therapeutics Corporation in March 2018. Our principal executive offices are located at One Kendall Square, Building 1400 West, 3rd Floor, Cambridge, Massachusetts.
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- Like Alnylam Pharmaceuticals, but Camp4 uses RNA to both activate and silence genes, rather than just silencing them.
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- Genetic Epilepsy Program (CAMP-A101): A clinical-stage therapeutic program developing an RNA-modulating drug candidate for the treatment of genetic epilepsies, such as Dravet syndrome.
- Huntington's Disease Program (CAMP-A001): A preclinical-stage therapeutic program developing an RNA-modulating drug candidate for the treatment of Huntington's disease.
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Camp4 Therapeutics (symbol: CAMP) is a clinical-stage biotechnology company focused on developing RNA-modulating therapeutics. As a company in the research and development phase, it does not currently have any commercial products approved for sale on the market.
Consequently, Camp4 Therapeutics does not have "major customers" in the traditional sense of companies or individuals purchasing its products or services. Its operations are primarily funded through:
- Equity Financing: Raising capital through the issuance of stock to investors.
- Potential Research Collaborations/Grants: While such collaborations could provide funding, Camp4 Therapeutics has not publicly disclosed any significant, active partnerships or licensing agreements that would constitute a major customer generating substantial revenue from product sales or technology licensing at this stage.
Therefore, Camp4 Therapeutics does not primarily sell to other companies or individuals, but rather operates as a development-stage company reliant on investment to fund its scientific programs.
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Josh Mandel-Brehm, President & Chief Executive Officer
Mr. Mandel-Brehm is a co-founder and has served as the President and Chief Executive Officer of CAMP4 Therapeutics since 2017. He was previously an entrepreneur partner with Polaris Partners. He has held key business development and operations leadership roles at various biotech companies, including serving in the Business Development group at Biogen, where he led multiple strategic activities and transactions, such as expanding Biogen's non-malignant hematology franchise and overseeing investments into ophthalmology. Prior to Biogen, he worked in business development for Genzyme's rare disease business unit. Mr. Mandel-Brehm also co-founded VICO Therapeutics, a biotech company based in the Netherlands.
Kelly Gold, Chief Financial Officer
Ms. Gold is the Chief Financial Officer at CAMP4 Therapeutics, having joined the company in 2017 and progressing through roles of increasing responsibility, including Chief Business Officer and SVP, Finance. She oversees corporate finance, accounting, investor relations, and strategic financial planning. Before joining CAMP4, Ms. Gold held various corporate finance and business planning roles at Biogen, where she provided financial leadership for late-stage and marketed rare disease programs. Her experience also includes working in the healthcare investment banking group at Deutsche Bank, where she advised on M&A, public and private equity offerings, leveraged finance transactions, and joint ventures, as well as executing dual-listed IPOs in Deutsche Bank's Latin American Investment Banking group. CAMP4 is a venture-backed biotech company.
Dr. Yuri Maricich, M.D., Chief Medical Officer
Dr. Maricich serves as Chief Medical Officer at CAMP4. He is a licensed physician-scientist, clinical/therapeutic developer, investor, and strategist. His background includes clinical practice as an internal medicine physician and leading teams and developing therapeutics at companies such as Xdynia (acquired Cavion), Cavion (acquired Jazz Pharmaceuticals), and Corixa (acquired GlaxoSmithKline). He also contributed to Pear Therapeutics, which became a public company with FDA authorized products like Somryst. He is a Venture Partner & Advisor at Angelini Ventures.
Dr. David Bumcrot, PhD, Chief Scientific Officer
Dr. Bumcrot is the Chief Scientific Officer at CAMP4 Therapeutics, where he oversees the Biology, Data Science, and Discovery teams. Prior to his role at CAMP4, he led groups at innovative biotech companies focused on advancing novel technologies toward clinical development. Notably, he was one of the first employees at Alnylam Pharmaceuticals, spending ten years working on RNA interference-based drugs for various indications and making key contributions to a first-in-class systemically administered siRNA therapeutic.
Caleb Moore, Chief Business Operations Officer
Mr. Moore is the Chief Business Operations Officer at CAMP4 Therapeutics. In this role, he is responsible for guiding the company's disease selection and program strategy based on biological rationale, as well as downstream development and commercial considerations. He leads CAMP4's program leadership, alliance management, and portfolio strategy functions, and also oversees key operational activities including laboratory operations, IT, and space planning.
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Addressable Markets for Camp4 Therapeutics' Main Products/Services:
- CMP-SYNGAP-01 (SYNGAP1-related disorders): The market size for SYNGAP1-related disorders is difficult to quantify in monetary terms from the available data. As of Q3 2024, there are 1,497 known patients worldwide. Over 1,530 patients have been identified to date. The incidence is reported as 1–4 per 10,000 individuals, or approximately 0.5–1.0% of all intellectual disability cases globally.
- CMP-CPS-001 (Urea Cycle Disorders):
- Global market: The urea cycle disorders treatment market was valued at $1.33 billion in 2024 and is projected to reach $1.37 billion in 2025. It is expected to grow to $1.62 billion by 2029. Another estimate values the global market at $2.5 billion in 2023, with a projection to reach $3.5 billion by 2030.
- U.S. market: The U.S. urea cycle disorders treatment market is estimated at $547.0 million in 2025 and is expected to reach $698.8 million by 2032.
- GBA1 regRNA program (Parkinson's Disease with GBA1 mutations):
- Global Parkinson's disease market: The Parkinson's Disease market size was approximately $3.218 billion in 2023. Approximately 5% of Parkinson's disease patients carry a GBA1 mutation.
- U.S. Parkinson's disease market: The U.S. accounted for 45% of the 2.7 million diagnosed prevalent cases of Parkinson's disease in 2023.
- Other pipeline indications (as mentioned in general company descriptions):
- Huntington's Disease:
- Global market: The global Huntington's disease treatment market was estimated at $500 million in 2024 and is projected to reach $1,871.2 million by 2030. Other estimates place the global market at $1.05 billion in 2024, projected to reach $2.60 billion by 2032.
- North America market: Held the largest revenue share of 40.85% in 2024 and was valued at $1.05 billion in 2024. The U.S. market is expected to hold $1.11 billion in 2025.
- Amyotrophic Lateral Sclerosis (ALS):
- Global market: The global amyotrophic lateral sclerosis therapeutics market was valued at $1.01 billion in 2023 and is projected to grow from $0.77 billion in 2024 to $1.80 billion by 2032.
- North America market: Dominated the market with a 71.29% share in 2023, valued at $0.72 billion. It is expected to reach $515.6 million by the end of 2032.
- Alpha-1 Antitrypsin Deficiency (AATD):
- Global market: The Alpha-1 Antitrypsin Deficiency market was valued at $3.57 billion in 2025 and is expected to reach $9.64 billion by 2032. Another source estimates the global market at $3.8 billion in 2025, projected to reach $12.7 billion by 2035.
- U.S. market: The U.S. Alpha-1 Antitrypsin Deficiency market was valued at $0.98 billion in 2025 and is expected to reach $2.61 billion by 2032.
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Camp4 Therapeutics (CAMP) is a clinical-stage biopharmaceutical company, and as such, its future revenue growth over the next 2-3 years will primarily be driven by progress in its pipeline and strategic collaborations, rather than immediate product sales. Based on available information, the key expected drivers include:
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Advancement of Lead Clinical Programs: A significant driver is the progression of its lead programs, particularly CMP-SYNGAP-01 for SYNGAP1-related disorders. The company initiated GLP toxicology studies for CMP-SYNGAP-01 (now referred to as CMP-002) in the third quarter of 2025, with a planned Phase 1/2 clinical trial anticipated to start as early as the second half of 2026. This program is positioned as a potential first-in-class treatment, and successful progression through clinical stages could unlock significant value and potential future revenues.
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Strategic Partnerships and Collaborations: Camp4 Therapeutics actively pursues partnerships for the development of its pipeline assets. The company has explicitly stated its intention to seek partnerships for the further development of CMP-CPS-001, its program for Urea Cycle Disorders (UCDs). Such collaborations can provide non-dilutive funding, upfront payments, milestone payments, and future royalties if products are successfully commercialized, thereby contributing to revenue growth. Its Q2 2025 revenue exceeding estimates also suggests growing collaboration and research activity.
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Growth in Research and Collaboration Revenue: Beyond specific pipeline assets, Camp4's business model includes generating revenue from research and collaboration agreements. The company's reported GAAP revenue has shown growth, indicating an increase in such activities. Continuing to leverage its regulatory RNA-targeting platform through new and expanded research partnerships could contribute to consistent, albeit early-stage, revenue streams.
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Share Issuance
- Camp4 Therapeutics completed an Initial Public Offering (IPO) around October 10, 2024, offering 6,820,000 shares of common stock at $11.00 per share, raising approximately $75.0 million in gross proceeds.
- In September 2025, the company secured an initial $50 million in gross proceeds from an oversubscribed private placement through the issuance of 26,681,053 shares of common stock at $1.53 per share, alongside additional shares to directors, employees, and consultants at $1.65, and pre-funded warrants.
- The private placement agreement includes the potential for an additional $50 million in gross proceeds from the issuance of up to 32,721,172 shares of common stock or pre-funded warrants, contingent upon achieving specific milestones, such as regulatory clearance for a Phase 1/2 clinical trial in SYNGAP patients.
Inbound Investments
- In September 2025, Camp4 Therapeutics received an initial $50 million from a private placement led by Coastlands Capital, with participation from institutional investors including Janus Henderson Investors, Balyasny Asset Management, Vivo Capital, 5AM Ventures, Adage Capital Management LP, Trails Edge Capital Partners, and the SynGAP Research Fund.
- 5AM Ventures VII, L.P. invested approximately $4.5 million in Camp4 Therapeutics, purchasing 2,941,176 shares of common stock on September 11, 2025.
- Polaris Management Co. VII, L.L.C. and Director Amir Nashat each made investments of approximately $2 million by purchasing 1,307,189 shares on September 11, 2025.
Capital Expenditures
- Since 2019, Camp4 Therapeutics' capital expenditure has decreased significantly by 81%.
- The company's capital expenditure reached its peak in 2020.
- The primary focus of capital allocation, reflected in significant research and development expenses, is for the development of biopharmaceutical product candidates, including clinical and preclinical study costs.
