Novartis has recently announced its acquisition of U.S.-based Regulus Therapeutics in a $1.7 billion deal. This acquisition strengthens the Swiss pharmaceutical giant’s position in kidney disease treatments. The deal gives Novartis access to Regulus’  patent portfolio focused on chronic kidney disease (CKD) and related disorders and RNA-targeted drug pipeline.

According to market estimates, the global CKD market was valued at $41.21 billion in 2024 and is projected to reach $86.83 billion by 2037. The expected growth highlights the commercial significance of Novartis’ latest move.

Farabursen: Kidney disease treatment

The transaction, announced by Novartis, centers on Regulus’ lead asset, RGLS8429 (farabursen), an investigational microRNA inhibitor targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), the most common genetic cause of renal failure worldwide.

Now in a Phase 1b trial, farabursen’s early results show favorable safety, promising pharmacokinetics, and encouraging biomarker responses. The therapy aims to preserve kidney function and slow disease progression in ADPKD patients. In addition to farabursen, Novartis will acquire Regulus’ broader portfolio of microRNA-based therapeutic technologies, strengthening its position in the nephrology space.

Clinical and regulatory outlook

As mentioned, farabursen has advanced through target validation, lead optimization, IND-enabling studies, and is progressing through Phase 1 clinical development. Recently, Regulus reported positive topline results from the fourth and final cohort of its Phase 1b multiple-ascending dose study. In this cohort, 26 patients received a fixed 300 mg dose biweekly for three months. Results showed consistent responses in urinary polycystin 1 and 2 levels and halted kidney volume growth— aligning with the findings from a previous weight-based cohort. Based on this data, Regulus has selected the 300 mg fixed dose for its planned Phase 3 trial.

According to a company release, the U.S. Food and Drug Administration has reviewed and agreed on key aspects of the program, including Chemistry, Manufacturing and Controls (CMC), non-clinical and clinical pharmacology plans, and the Phase 3 trial design. The pivotal study will use a 12-month height-adjusted total kidney volume (htTKV) endpoint for potential Accelerated Approval and a 24-month estimated glomerular filtration rate (eGFR) endpoint for Full Approval. Other Regulus programs in nephrology and CNS remain in early discovery stages.

M&A as innovation strategy

Novartis continues to drive progress in kidney health as reflected in their recent FDA approvals in renal care: Vanrafia® in IgA nephropathy (IgAN) in April 2025, and Fabhalta® for C3 glomerulopathy (C3G) and IgAN in March 2025 and August 2024, respectively.

This acquisition move by Novartis is part of a broader trend seen in recent industry deals. Companies like Roche, AstraZeneca, and Pfizer are following suit, with Roche’s $4.3 billion acquisition of Spark Therapeutics, AstraZeneca’s purchase of Neogene Therapeutics, and Pfizer’s partnership with Beam Therapeutics all demonstrating the growing reliance on M&A to accelerate innovation.

Novartis and Regulus: The patents behind the deal

Novartis’ acquisition is reinforced by Regulus’ patent application WO2024215846A1, which outlines a method for treating polycystic kidney disease (PKD) with a chemically modified oligonucleotide. The compound is designed for subcutaneous delivery which offers enhanced stability and therapeutic potential.

From this point forward, we take a closer look at Regulus’ patent portfolio and highlight recent filings related to kidney health.

Regulus’ Patenting Activity

Regulus saw its most active filing year in 2016, which likely reflects a strategic push to protect its RNA-targeting platform as it entered critical stages of clinical development. That same year, the company secured a $30 million loan from Oxford Finance LLC to boost its clinical and preclinical pipeline. In addition, Regulus announced their expanded partnership with GlaxoSmithKline for the phase II study of their lead candidate RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for hepatitis C.

By the end of 2016, Regulus also announced two new treatments:  RGLS5040, a candidate for the treatment of cholestatic diseases, and RGLS4326 for the treatment of ADPKD. Notably, in October 2021, Regulus announced the discontinuation of its first-generation ADPKD candidate, RGLS4326, in favor of advancing RGLS8429, a next-generation compound targeting miR-17.

Top Legal Representatives

Regulus Therapeutics’ top legal representatives reflect a global patent strategy, with leading firms from the UK (Carpmaels & Ransford), Japan (Saegusa & Partners), and Canada (Smart & Biggar). The U.S. presence is notable through Rebecca B. Scarr from McNeill PLLC, and Spruson & Ferguson, while firms from China, Taiwan, and South Korea highlight Regulus’ active engagement across key Asian markets.

Regulus’ Global Patent Distribution

Regulus Therapeutics has built a strong global IP portfolio, with the most filings under the PCT followed by Japan, U.S., and Canada. Japan’s top position in the list might be attributed to Regulus’ exclusive license to the Sarnow patent estate covering anti-miR-122 therapies for hepatitis C, which is a public health concern in the country. Meanwhile, Canada has emerged as a global biotechnology leader, contributing significantly to advancements such as mRNA vaccine delivery systems and stem cell research, while also serving as a strategic hub and gateway to the North American biotech market.

Regulus Therapeutics’ patent filings center on biotech and pharmaceutical applications

Regulus Therapeutics concentrated its patent activity on biotechnology and pharmaceutical technologies. The largest portion, 33%, was classified under C12N, covering inventions involving microorganisms or enzymes.

Therapeutic compounds under A61P accounted for 31.1% of filings, while 29.6% were classified under A61K, relating to medical, dental, or hygiene preparations. Additional classifications included C07H (5.5%) for organic compounds and C12Q (0.4%) for testing processes involving enzymes or nucleic acids.

Regulus Therapeutics: Patents on RNA-based kidney disease treatments

Regulus has steadily expanded its portfolio, filing patents worldwide. Many of these focus on the modulation of microRNAs—small, non-coding RNA molecules that play a key role in regulating gene expression in kidney cells and form the basis of the company’s RNA-based therapeutic approach.

RNA-based treatment approaches for polycystic kidney disease

Polycystic kidney disease (PKD) is a genetic disorder that causes fluid-filled cysts to develop in the kidneys. U.S. Patent Application No. 2024/0294913 introduces a potential treatment for PKD using modified RNA molecules to block microRNAs that drive the disease. Specifically, it focuses on engineered oligonucleotides designed to inhibit the miR-17 family of microRNAs—key regulators linked to PKD progression. By targeting the miR-17 microRNA family, the compound may reduce kidney volume, limit cyst growth, and enhance kidney functions.

The patent application was filed on March 20, 2024, and was published on September 5, 2024. The inventors are Denis Drygin, Garth A. Kinberger, and Edmund Chun Yu Lee. McNeill PLLC is representing Regulus in this patent filing.

Refined RNA therapy designs to minimize off-target activity

Complementing the previous application, U.S. Patent Application No. 2025/0136979 focuses on improving the safety of RNA-based therapeutics. It describes novel oligonucleotides compositions designed to selectively inhibit microRNAs that begin with uracil or cytosine.

The compounds feature purine analogs engineered to avoid unintended hydrogen bonding, along with chemical modifications such as 2′-O-methoxyethyl and sugars to enhance stability and precision.

The patent application was filed on October 7, 2022, and was published on May 1, 2025. The application lists Denis Drygin, Garth A. Kinberger, and Edmund Chun Yu Lee as inventors. Regulus is represented by McNeill PLLC.

GalNAc-conjugated antiviral therapy for hepatitis C

Expanding beyond kidney-related indications, Regulus Therapeutics has also explored antiviral applications of its RNA-based technology.

U.S. Patent No. 12,005,120, discloses a GalNAc-conjugated oligonucleotide that targets microRNA-122 (miR-122)—a crucial host factor required for hepatitis C virus (HCV) replication. The compound described in the patent is engineered to retain strong antiviral efficacy while reducing the risk of hyperbilirubinemia, a common side effect associated with earlier RNA-based HCV treatments.

The patent was filed on May 8, 2018, and was granted on June 11, 2024. McNeill PLLC represented Regulus in the patent filing. The inventors listed are Charles R. Allerson, Steven S. Neben, and Timothy Wright.

Looking ahead

Regulus Therapeutics plans to begin its Phase 3 pivotal trial of farabursen in the third quarter of 2025. The study will support regulatory submissions for autosomal dominant polycystic kidney disease (ADPKD), a condition affecting an estimated 160,000 people in the U.S. and up to 7 million worldwide.

In Q1 2025, Regulus Therapeutics reported a net loss of $9.6 million, up from $8.5 million a year earlier, driven in part by rising R&D costs, which climbed to $6.8 million. These losses, compounded by lukewarm investor reaction to interim Phase 1b data for farabursen have intensified pressure on the company. Regulus’s shares plunged 24% following the update, despite favorable FDA feedback and promising biomarker trends from the trial’s fourth cohort.

While the FDA signaled alignment with the proposed Phase 3 design, the market response highlights skepticism about Regulus’s ability to carry the program forward alone. Continued operating losses and investor concerns likely accelerated Regulus’s acquisition by Novartis, providing the biotech with a financial runway and the backing needed to push farabursen toward regulatory approval. Regulus will continue to operate independently while the pending acquisition by Novartis moves forward.