Regulus Therapeutics
Regulus Therapeutics is a clinical-stage biopharmaceutical company developing microRNA-targeting therapeutics for rare kidney diseases, with lead asset RGLS8429 for ADPKD. Founded in 2007 in San Diego, it was pre-revenue and acquired by Novartis in 2025 for up to $1.7 billion.
- Company typePrivate
- Founded2007
- HeadquartersSan Diego, United States
- Headcount51–100
- GTM typeB2B
- OfferingHardware or Manufacturing
What Regulus Therapeutics does
Regulus Therapeutics is a clinical-stage biopharmaceutical company founded in 2007 and headquartered in San Diego, California, that develops therapeutics targeting microRNAs for the treatment of rare kidney diseases. The company's lead asset, RGLS8429, is a microRNA-targeting drug candidate for Autosomal Dominant Polycystic Kidney Disease (ADPKD), which completed a Phase 1 Single Ascending Dose (SAD) study with positive safety and pharmacokinetic data and progressed into a Phase 1b Multiple Ascending Dose (MAD) study with first patient dosed in November 2022.
The company's core technology is a proprietary microRNA-targeting drug discovery platform described as a novel therapeutic approach to address root causes of rare kidney diseases. Regulus monetized its pipeline through successive private equity financings rather than commercial product sales — raising approximately $34.6 million in November 2021, $4.5 million via an ATM sale in November 2022, and an oversubscribed $100 million private placement in March 2024 led by Adage Capital Partners and RA Capital Management, with additional participation from NEA, Federated Hermes, Vivo Capital, Deep Track Capital, and Octagon Capital. The company was publicly traded on NASDAQ under the symbol RGLS prior to its 2025 acquisition by Novartis AG for up to $1.7 billion, after which it operates as a wholly-owned subsidiary contributing to Novartis' San Diego-based RNA targeting and xRNA platform capabilities.
Regulus was pre-revenue throughout its standalone history, with no commercialized products and customer-facing activity limited to clinical trial enrollment, scientific conferences, and investor relations communications. The intended end market — patients with ADPKD and other rare kidney diseases — represents a defined but constrained therapeutic population, with the global ADPKD market estimated at US$1.93 billion in 2024.
Regulus Therapeutics firmographics
Firmographics- Name
- Regulus Therapeutics
- Legal name
- Regulus Therapeutics Inc.
- Website
- https://regulusrx.com
- Company type
- Private
- Founded year
- 2007
- Operating status
- Acquired
- Headcount range
- 51–100 employees
- Short description
- Regulus Therapeutics is a clinical-stage biopharmaceutical company developing microRNA-targeting therapeutics for rare kidney diseases, with lead asset RGLS8429 for ADPKD. Founded in 2007 in San Diego, it was pre-revenue and acquired by Novartis in 2025 for up to $1.7 billion.
- Ownership category
- akta.pro rank
Regulus Therapeutics industry classification
Industry- Product category
- MicroRNA Therapeutics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- siRNA Therapeutics (gene silencing) (HLAAADAB)
Keywords
Where Regulus Therapeutics is headquartered
LocationHeadquarters
- HQ city
- San Diego
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Regulus Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure
Revenue model
- Pipeline Development Funding: Regulus raises capital through private placements to fund its microRNA-targeting drug development pipeline. The company is pre-revenue and does not have commercialized products.
Go-to-market motion1 record
Marketing channels2 records
Regulus Therapeutics product offering
Product offeringCore offering
Regulus Therapeutics is a clinical-stage biopharmaceutical company developing microRNA-targeting therapeutics, with its lead candidate RGLS8429 in clinical development for autosomal dominant polycystic kidney disease (ADPKD). The company leverages a proprietary platform of anti-miR oligonucleotides designed to selectively inhibit disease-driving microRNAs, primarily within the miR-17 family, for rare disease indications.
Product overview
Regulus Therapeutics is a biotech company specializing in microRNA-targeting drug development. The company's primary focus is on RGLS8429, an investigational drug for Autosomal Dominant Polycystic Kidney Disease (ADPKD). Regulus Therapeutics was acquired by Novartis for approximately $1.7 billion in 2025, integrating into Novartis' San Diego operations as part of their xRNA platform development.
Differentiator
Problem solved
Functional benefit
Products and services
- RGLS8429
Quantifiable outcome
- RGLS8429 Phase 1 SAD study demonstrated positive safety and pharmacokinetic data
- +1 more outcomes
Companies that use Regulus Therapeutics
Customer profileSegments1 record
Ideal customer profiles2 records
Regulus Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature1 record
Regulus Therapeutics partnerships and signals
Strategic signalPartnerships
One partnership is on record.
- Novartis AGflagshipNovartis acquired Regulus Therapeutics for up to $1.7 billion as part of its strategy to expand capabilities in RNA targeting small molecule drug discovery. The acquisition is part of Novartis' broader expansion in San Diego, including a $1.1 billion research hub, strengthening capabilities in gene therapy and xRNA platforms.
Scale indicators4 records
Recent moves5 records
Expansion highlights3 records
Regulus Therapeutics competitors and assessment
Company assessmentDirect peers
- Alnylam Pharmaceuticals: Leading clinical and commercial-stage RNA interference (siRNA) therapeutics company. Directly comparable to Regulus as an RNA-targeting platform developing oligonucleotide drugs for serious diseases, with a far larger pipeline and commercial footprint.
- Arrowhead Pharmaceuticals: Clinical-stage biotechnology company developing RNAi-based therapeutics using targeted RNAi molecules (TRiM™). Directly comparable to Regulus as an RNA-targeting platform with multiple clinical-stage candidates across rare and chronic diseases.
- Ionis Pharmaceuticals: Pioneer in antisense oligonucleotide (ASO) RNA-targeted drug discovery with a large clinical pipeline and multiple approved products. Directly comparable to Regulus as a leader in RNA-targeting therapeutic modalities.
- Dicerna Pharmaceuticals: Developer of GalNAc-conjugated RNAi therapeutics (acquired by Novo Nordisk). Closely comparable to Regulus as a clinical-stage RNA-targeting platform using a conjugate delivery approach for liver and rare disease indications.
- Wave Life Sciences: Clinical-stage RNA therapeutics company developing stereopure oligonucleotides for rare and neurological diseases. Comparable to Regulus as a small-cap, clinical-stage RNA-modality platform.
- ProQR Therapeutics: Clinical-stage RNA editing and oligonucleotide therapeutics company focused on rare genetic diseases. Comparable to Regulus as a small-cap, clinical-stage RNA-based platform developing treatments for serious diseases with high unmet need.
Emerging players
- BridgeBio Pharma: Clinical-stage biopharmaceutical company targeting genetic diseases, including programs relevant to kidney disease. Comparable to Regulus as a clinical-stage rare-disease biotech with a focus on genetically validated targets.
Broad incumbents
- Otsuka Pharmaceutical: Markets tolvaptan (Jinarc) — the only currently approved disease-modifying therapy for ADPKD. Comparable to Regulus as a direct competitor in the ADPKD commercial market, with established nephrology infrastructure.
- Vertex Pharmaceuticals: Large commercial-stage biopharmaceutical company with growing rare disease and nephrology interests. Comparable to Regulus as a broader rare-disease biotech acquirer/innovator that has historically engaged in M&A and platform-based drug development.
- Moderna: Commercial-stage biotechnology company pioneering mRNA therapeutics and vaccines with an R&D pipeline expanding into rare diseases. Comparable to Regulus as a broader RNA-based therapeutics platform, though at significantly larger scale.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat3 records
Key risks5 records
Key highlights6 records
Customer concentration
Regulus Therapeutics social profiles
Digital presenceRegulus Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Regulus Therapeutics leadership team
Management profileNumber of profiles
Profiles6 records
Regulus Therapeutics funding detail
Funding detailFunding overview
Funding rounds15 records
Investors10 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Regulus Therapeutics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Regulus Therapeutics
What does Regulus Therapeutics do?
Regulus Therapeutics is a clinical-stage biopharmaceutical company developing microRNA-targeting therapeutics, with its lead candidate RGLS8429 in clinical development for autosomal dominant polycystic kidney disease (ADPKD). The company leverages a proprietary platform of anti-miR oligonucleotides designed to selectively inhibit disease-driving microRNAs, primarily within the miR-17 family, for rare disease indications.
Is Regulus Therapeutics a public or private company?
Regulus Therapeutics is a private company. It is classified as corporate owned and is currently acquired.
When was Regulus Therapeutics founded?
Regulus Therapeutics was founded in 2007. It employs 51 to 100 people.
Where is Regulus Therapeutics based?
Regulus Therapeutics is headquartered in San Diego, United States, in the North America region.
How does Regulus Therapeutics make money?
One revenue line is on record: pipeline Development Funding.
Who are Regulus Therapeutics's main competitors?
Direct peers on record are Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Ionis Pharmaceuticals, Dicerna Pharmaceuticals, Wave Life Sciences and ProQR Therapeutics. BridgeBio Pharma is listed as an emerging player. Broad incumbents are Otsuka Pharmaceutical, Vertex Pharmaceuticals and Moderna.
Does Regulus Therapeutics have an API?
No public API is recorded for Regulus Therapeutics.
What industry is Regulus Therapeutics in?
Regulus Therapeutics's product category is MicroRNA Therapeutics. Its primary akta.pro industry code is HLAAADAB, siRNA Therapeutics (gene silencing). Its NAICS code is 541714 and its SIC code is 2836.