What CAMP4 Therapeutics does

CAMP4 Therapeutics is a clinical-stage biotechnology company developing antisense oligonucleotide (ASO) therapeutics designed to selectively upregulate gene expression by targeting regulatory RNAs (regRNAs). Founded in 2016 and headquartered in Cambridge, Massachusetts, the company is publicly traded on NASDAQ under the ticker CAMP. Its core technology is the RAP Platform® (RNA Actuating Platform), which systematically maps regRNAs across the human genome and generates ASO leads that modulate those regRNAs to restore protein production in diseases of haploinsufficiency. The proprietary regRNA catalog spans key disease-relevant cell types including CNS, liver, heart, skeletal muscle, and immune system.

CAMP4's clinical pipeline is anchored by CMP-002, an ASO candidate for SYNGAP1-related disorder (a rare CNS disease affecting over 10,000 individuals in the US with no approved disease-modifying therapy), which is on track for Phase 1/2 initiation in H2 2026 with retained commercial rights. The second pipeline asset, CMP-001, targets the CPS1 gene in urea cycle disorders, has completed Phase 1 analysis, and is being evaluated for partnership. In December 2025, CAMP4 entered a strategic research collaboration with GSK covering ASO drug candidates for neurodegenerative and kidney diseases, receiving $17.5M upfront and eligibility for up to $440M in milestones plus royalties.

The company generates no product revenue. Its business model rests on three near-term economic levers: (1) milestone payments and royalties from the GSK collaboration, (2) potential future partnership economics on CMP-001 and additional platform-derived programs, and (3) eventual direct commercialization of CMP-002 in rare disease CNS channels (patient communities, specialty prescribers, clinical trial sites). As of March 2026, CAMP4 held approximately $109.5M in cash, extending its operational runway into 2028, supported by a $30M public offering (December 2025), a $50M initial tranche of an oversubscribed private placement (September 2025), and prior venture capital including Series A ($45M, 2021) and Series B ($100M, 2022) rounds. The company employs 11–50 people and is expanding into a ~44,000 sq ft laboratory and office facility in Watertown, Massachusetts.

Name

CAMP4 Therapeutics

Legal name

CAMP4 Therapeutics Corporation

Website

https://camp4tx.com

Company type

Public

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Short description

CAMP4 Therapeutics is a clinical-stage biotechnology company developing antisense oligonucleotide therapeutics that selectively upregulate gene expression by targeting regulatory RNAs (regRNAs) to treat haploinsufficiency disorders, with lead candidate CMP-002 entering Phase 1/2 for SYNGAP1-related disorder in H2 2026.

Ownership category

akta.pro rank

Product category

Antisense oligonucleotide therapeutics for genetic diseases

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industries

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), Neurology/CNS Gene Therapies (HLAAACAH), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)

Keywords

• Antisense oligonucleotide therapeutics
• Regulatory RNA targeting
• Gene expression upregulation
• Rare genetic disease therapeutics
• Haploinsufficiency drug discovery

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Product Revenue (future): CAMP4 is a pre-commercial, clinical-stage biotech company with no current product revenue. The company is developing ASO therapeutics and plans to commercialize its pipeline candidates directly or through partnerships. CMP-002 for SYNGAP1-related disorder and CMP-001 for urea cycle disorders represent the primary pipeline assets. The company retains commercial rights for CMP-002.
2. Licensing and Milestone Payments: CAMP4 receives upfront payments, milestone payments, and royalties from strategic partnerships. The GSK collaboration includes a $17.5 million upfront payment, potential milestones up to $440 million, and royalty payments on future commercialization. The company is also exploring partnership opportunities for CMP-001 in urea cycle disorders.
3. Public Equity Offering: The company raised $30 million in December 2025 through an underwritten public offering of 5 million shares at $6.00 per share. This supplements the September 2025 private placement that raised $50 million initially, with potential for another $50 million upon reaching certain milestones. These are equity financing events to support clinical development rather than revenue-generating operations.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

CAMP4 Therapeutics develops antisense oligonucleotide (ASO) therapeutics designed to upregulate gene expression by targeting regulatory RNAs (regRNAs), with a focus on diseases caused by haploinsufficiency. Its lead clinical programs include CMP-002 for SYNGAP1-related CNS disorders and CMP-001 for urea cycle disorders, developed through its proprietary RAP® platform.

Product overview

CAMP4 Therapeutics is pioneering a new class of antisense oligonucleotide (ASO) medicines designed to selectively upregulate gene expression by modulating regulatory RNAs (regRNAs). Their core technology is the RAP Platform®, which enables systematic mapping of regRNAs across the human genome and rapid development of ASO therapeutic candidates. The company's pipeline includes CMP-002 for SYNGAP1-related disorder (lead CNS program, clinical initiation anticipated H2 2026) and CMP-001 for urea cycle disorders (in Phase 1/2). They also have a strategic research collaboration with GSK for neurodegenerative and kidney diseases, leveraging their platform to identify and develop ASO drug candidates for multiple gene targets.

Differentiator

Problem solved

Functional benefit

Products and services

• CMP-002 Clinical-stage antisense oligonucleotide therapeutic designed to upregulate SYNGAP1 gene expression for the treatment of SYNGAP1-related CNS disorders. Intended for patients with haploinsufficiency-driven neurodevelopmental disease.
• CMP-001 Antisense oligonucleotide therapeutic designed to upregulate expression of a key urea cycle enzyme gene for the treatment of urea cycle disorders, a class of rare metabolic diseases of haploinsufficiency.

Quantifiable outcome

• CMP-002 produced statistically significant improvements in seizure threshold and severity in SYNGAP1 haploinsufficient mouse models compared to untreated mice in preclinical studies
• +3 more outcomes

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Two partnerships are on record, tiered minor and core.

• Michael MacLeanminorOthers · 24 March 2026Michael MacLean was appointed to the Board of Directors as a Class III director effective April 1, 2026, expanding the board from eight to nine members. MacLean brings over 35 years of financial leadership experience in biotechnology, having served as CFO at Avidity Biosciences and Akcea Therapeutics, and Chief Accounting Officer at Biogen.
• GSKcoreStrategic or Co-development Partner · 18 December 2025CAMP4 Therapeutics entered a strategic research collaboration with GSK to develop antisense oligonucleotide (ASO) drug candidates for neurodegenerative and kidney diseases. CAMP4 received a $17.5 million upfront payment and is eligible for milestone payments up to $440 million and royalties. GSK will handle further development and commercialization. The collaboration leverages CAMP4's RAP Platform to identify regulatory RNAs and develop ASO candidates for multiple gene targets relevant to neurodegenerative and kidney disease indications.

Scale indicators12 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Stoke Therapeutics: Stoke is the most direct peer: it develops ASOs to upregulate gene expression in haploinsufficiency disorders, including programs in SYNGAP1-related disorder. It is the closest comparable by both mechanism (ASO-based gene upregulation) and target indication (SYNGAP1).
• Ionis Pharmaceuticals: Ionis is the pioneer and largest dedicated ASO therapeutics company, with approved products (SPINRAZA, TEGSEDI, WAYLIVRA) and a deep pipeline across neurological, metabolic, and rare genetic diseases. CAMP4 is a downstream platform peer leveraging ASO chemistry pioneered by Ionis.
• Wave Life Sciences: Wave is a clinical-stage oligonucleotide therapeutics company developing stereopure ASOs and siRNAs for rare and neurological diseases, including programs in Huntington's disease and ALS. Highly comparable in modality, stage, and CNS focus.
• ProQR Therapeutics: ProQR develops RNA-targeted oligonucleotide therapeutics for genetic diseases, including programs in cystic fibrosis and rare eye and metabolic disorders. Directly comparable as a small-cap clinical-stage RNA therapeutics company pursuing rare genetic indications.

Broad incumbents

• Alnylam Pharmaceuticals: Alnylam is the leading RNAi therapeutics company with multiple approved products (ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA). It represents the broader-incumbent RNA therapeutics category against which CAMP4's regRNA-targeting ASOs compete for partnerships and investor mindshare.
• Biogen: Biogen is a large biopharma with significant CNS and rare disease exposure, developed and commercializes SPINRAZA (the landmark ASO), and was an early investor in CAMP4 (2020 financing). It is a strategic incumbent whose CNS franchise overlaps directly with CAMP4's lead indication.
• Sarepta Therapeutics: Sarepta is a leading genetic medicines company with approved exon-skipping ASOs and AAV gene therapies for Duchenne muscular dystrophy and broader neuromuscular/rare disease programs. Comparable as a rare genetic disease therapeutics developer with oligonucleotide expertise.

Emerging players

• Korro Bio: Korro Bio is an early-stage company developing RNA editing (ADAR-mediated) therapeutics to upregulate or modify protein expression in genetic diseases, including indications overlapping with haploinsufficiency. Competes with CAMP4 for the same target biology via a different RNA-based mechanism.
• Vico Therapeutics: Vico is an early-stage ASO company developing allele-selective oligonucleotides for CNS disorders, including repeat-expansion diseases. It is a co-founded company of CAMP4's CEO Josh Mandel-Brehm and represents a smaller-scale, similarly focused ASO peer.
• Solid Biosciences: Solid Biosciences is a clinical-stage genetic medicines company developing AAV gene therapies and other modalities for rare neuromuscular and CNS disorders. Comparable as a small-cap clinical-stage peer pursuing disease-modifying treatments for rare genetic diseases.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/media
• LinkedInlinkedin.com/company/camp4-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles14 records

Funding overview

Funding rounds6 records

Investors16 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about CAMP4 Therapeutics