Here are 1-3 brief analogies for Camp4 Therapeutics (CAMP):

• Ionis Pharmaceuticals, but for switching genes on.
• Alnylam Pharmaceuticals, but focused on turning genes *on* instead of off.

• RNA Actuating Platform (RAP Platform): A proprietary technology platform for identifying regulatory RNA sequences and designing antisense oligonucleotide (ASO) drug candidates to upregulate gene expression.
• CMP-CPS-001: A clinical-stage RNA Actuator product candidate in Phase 1 trials, designed to treat Urea Cycle Disorders (UCDs) by increasing the expression of the CPS1 enzyme.
• SYNGAP1 Program: A preclinical RNA Actuator program focused on developing therapeutics for SYNGAP1-related disorders.

Camp4 Therapeutics (CAMP) is a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapeutics. As such, the company is currently in the research and development phase and does not have commercial products available for sale. Therefore, it does not currently have "major customers" in the traditional sense of entities purchasing its products or services.

However, if their product candidates receive regulatory approval and are commercialized, their ultimate "customers" would be patients. Based on the company's current development pipeline and stated goals, the categories of patients they aim to serve include:

1. Patients with Urea Cycle Disorders (UCDs): These are individuals suffering from severe, inherited metabolic diseases caused by mutations in genes necessary to convert ammonia into urea. CMP-CPS-001 is their lead product candidate targeting this group.
2. Patients with SYNGAP1-related disorders: This is their first preclinical program targeting individuals with this specific neurological disorder.
3. Patients with a broad range of other genetic diseases characterized by deficient protein levels: This includes individuals with haploinsufficient diseases or recessive partial loss-of-function disorders, where a modest increase in protein expression can provide therapeutic benefit.

Josh Mandel-Brehm, President & Chief Executive Officer

Mr. Mandel-Brehm is the President & Chief Executive Officer of CAMP4 Therapeutics. He was appointed CEO in January 2017. Previously, he was an entrepreneur partner with Polaris Partners. Mr. Mandel-Brehm held key business development and operations leadership roles at Biogen and Genzyme. He co-founded Vico Therapeutics, where he currently serves as a board Director, and is also a board Director at ProMIS Neuroscience.

Kelly Gold, Chief Financial Officer

Ms. Gold is the Chief Financial Officer at CAMP4 Therapeutics. She joined CAMP4 in 2017 and was promoted to CFO in April 2022, after holding roles of increasing responsibility, including Chief Business Officer and SVP, Finance. At CAMP4, she has played a key role in raising a combined $90 million through company financings. Prior to CAMP4, Ms. Gold held various corporate finance and business planning roles at Biogen, providing financial leadership for late-stage and marketed rare disease programs. Before Biogen, she worked in the Healthcare and Latin American Investment Banking groups at Deutsche Bank, where she advised on M&A, public and private equity offerings, and leveraged finance transactions. She began her career as a mechanical engineer designing biocontainment systems.

Yuri Maricich, MD, Chief Medical Officer

Dr. Maricich serves as Chief Medical Officer at CAMP4. He is a licensed physician-scientist, clinical/therapeutic developer, investor, and strategist, with a background that includes clinical practice as an internal medicine physician.

Daniel Tardiff, PhD, Chief Scientific Officer

Dr. Tardiff was elevated to Chief Scientific Officer, effective October 1, 2025. He joined CAMP4 in 2023 and has been instrumental in leading the company's drug discovery efforts.

Michelle Gates, SVP, Chief People Officer

Ms. Gates was appointed to the newly created role of Senior Vice President and Chief People Officer in July 2021.

Key Risks to Camp4 Therapeutics

1. Unproven Technology and Regulatory Uncertainty: Camp4's core RNA Actuating Platform (RAP Platform) and its approach of developing antisense oligonucleotides (ASOs) directed towards regulatory RNAs (regRNAs) to upregulate gene expression is unproven. No regulatory authorities have yet approved ASOs specifically targeting regRNAs, which creates significant uncertainty regarding the safety, efficacy, and eventual regulatory pathway for their product candidates.
2. Early Stage of Product Development and High Risk of Clinical Trial Failure: Camp4 Therapeutics is in the early stages of product development. Their lead product candidate, CMP-CPS-001, is only in an ongoing Phase 1 clinical trial, and their SYNGAP1 program is preclinical. The success of their business depends heavily on the successful development and regulatory approval of their product candidates, a process that is lengthy, costly, and has a high rate of failure at each stage of clinical development.

Camp4 Therapeutics' lead product candidate, CMP-CPS-001, targets urea cycle disorders (UCDs).

The global market for urea cycle disorders treatment was estimated at $1.37 billion in 2025 and is projected to grow to $1.43 billion in 2026, with a compound annual growth rate (CAGR) of 3.8%. This market is expected to reach $1.71 billion by 2030, growing at a CAGR of 4.6%.

For SYNGAP1-related disorders, the other program in Camp4 Therapeutics' pipeline, a quantifiable addressable market size in monetary terms is not available. Therefore, the market size for SYNGAP1-related disorders is null.

Here are 3-5 expected drivers of future revenue growth for Camp4 Therapeutics (CAMP) over the next 2-3 years:

• Milestone payments from strategic partnerships for CMP-CPS-001: As Camp4 Therapeutics advances its lead product candidate, CMP-CPS-001 for Urea Cycle Disorders (UCDs), through clinical trials, successful data readouts (such as the anticipated Phase 1 data in Q1 and H2 2025) could attract strategic partners. These partnerships typically involve upfront payments and milestone payments tied to development and regulatory achievements, which would contribute to revenue within the specified timeframe.
• Upfront and research payments from collaborations leveraging the RNA Actuating Platform (RAP): Camp4 Therapeutics' proprietary RAP Platform, designed to identify and characterize regulatory RNAs for a broad range of genetic diseases, represents a valuable asset. Licensing this platform or entering into research collaborations with other pharmaceutical companies could generate revenue through upfront fees and ongoing research funding agreements.
• Milestone payments from the advancement of the SYNGAP1 program: The company expects to initiate final Good Laboratory Practice (GLP) toxicology studies for its SYNGAP1-related disorders program in 2025, paving the way for clinical trial applications. Progression of this program into clinical development could lead to partnership opportunities and associated milestone payments, contributing to future revenue growth.
• Out-licensing of additional preclinical assets identified by the RAP Platform: Beyond its lead programs, Camp4 Therapeutics' RAP Platform has the potential to identify numerous novel regulatory RNA targets and generate new preclinical candidates for various genetic diseases. The out-licensing of these early-stage assets to other companies for further development could provide revenue through upfront payments and future milestones.

Share Repurchases

• Camp4 Therapeutics has not reported significant share repurchase activity over the last 3-5 years.

Share Issuance

• The number of shares outstanding for Camp4 Therapeutics increased by 547.76% in one year, indicating substantial share issuance.
• In October 2024, Camp4 Therapeutics completed an Initial Public Offering (IPO), pricing $75 million at $11 per share.
• The company also completed a follow-on equity offering of $30 million and filed for another follow-on offering of $100 million.

Inbound Investments

• In December 2025, Camp4 Therapeutics signed a research, collaboration, and license agreement with GSK, receiving an upfront payment of $17.5 million.
• The agreement with GSK also includes eligibility for up to $440 million in milestones and tiered royalties on future net sales for neurodegenerative and kidney disease indications.
• Camp4 Therapeutics received approximately $100.17 million in funding from various investors, including Janus Henderson Investors, Balyasny Asset Management L.P., Vivo Capital, LLC, Adage Capital Management, L.P., Trails Edge Capital Partners, LLC, and SYNGAP Research Fund.

Capital Expenditures

• Camp4 Therapeutics Corporation's Capital Expenditure was -$279.0 thousand in 2025, reflecting a 36.7% year-over-year increase.
• In the last 12 months (as of March 2026), capital expenditures were -$279,000.