Regulus Therapeutics

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.

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

Product category

MicroRNA Therapeutics

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro industry

PRIMARYHLAAADAB

siRNA Therapeutics (gene silencing)

Healthcare & Life Sciences/Biotechnology & Biologics/RNA Therapeutics (mRNA, siRNA, ASO)

Keywords

• MicroRNA therapeutics
• Rare disease biopharmaceuticals
• ADPKD drug development
• RNA-targeting therapeutics
• Oligonucleotide-based drugs

Headquarters

HQ city

San Diego

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Infrastructure

Revenue model

1. 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

Core 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

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

One partnership is on record.

• Novartis AGflagshipStrategic or Co-development Partner · 1 January 2024Novartis 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

Direct 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

• Xx.com/RegulusRx
• Facebookfacebook.com/134644253293544
• LinkedInlinkedin.com/company/regulus-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds15 records

Investors10 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

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

Frequently asked questions about Regulus Therapeutics