Fibrosis Drug Pipeline Across Indications — PatSnap Eureka
Fibrosis Drug Pipeline Across Liver, Lung, Kidney, Heart & Skin
Organ fibrosis collectively accounts for an estimated 45% of deaths in the developed world. Despite decades of preclinical advances, only pirfenidone and nintedanib carry regulatory approval — both solely for IPF. Discover the shared pathway strategies reshaping cross-indication drug discovery.
Key Data Signals from the Fibrosis Patent & Literature Dataset
Visualising the distribution of therapeutic modalities and cross-organ target breadth identified across retrieved patent filings and academic publications.
Therapeutic Modality Distribution — Fibrosis Pipeline Dataset
Small molecules are the most densely represented class; cell-based therapies are the only modality with ongoing clinical trials (liver fibrosis/cirrhosis).
Cross-Organ Molecular Target Coverage
TGF-β/Smad is the only target implicated across all five organ indications; PPAR and HDAC cover four each.
Seven Drug Classes Targeting Shared Fibrotic Pathways
From approved small molecules to emerging oligonucleotide therapies, the fibrosis pipeline spans a diverse range of modalities — many explicitly designed to address cross-indication targets. Explore the PatSnap analytics platform to map competitive landscapes across each class.
Kinase Inhibitors, PPAR Agonists & Novel Scaffolds
Small molecules are the most densely represented class in this dataset. Pirfenidone (approved IPF) shows preclinical evidence for NASH-associated liver fibrosis via inhibition of TNF-α-induced apoptosis. Nintedanib (tyrosine kinase inhibitor) is approved for IPF and systemic sclerosis-associated ILD. PXS-5505, a pan-lysyl oxidase inhibitor targeting LOX/LOXL2-mediated collagen crosslinking, demonstrated activity in skin and lung in systemic sclerosis rodent models and is in early clinical development for myelofibrosis. Pan-PPAR agonist lanifibranor reduced lung fibrosis in a TGFβ-overexpressing transgenic mouse model of scleroderma. The California Institute for Biomedical Research has filed patents covering small molecule libraries explicitly spanning IPF, liver cirrhosis, kidney fibrosis, cardiac fibrosis, and keloid formation.
Pirfenidone & nintedanib approved (IPF); PXS-5505 early clinicalIL-11 Antibodies, BMP-7, END55 & Collagen-Targeting Constructs
IL-11 neutralizing antibodies targeting IL-11 or IL-11RA show anti-fibrotic activity in human fibroblasts and murine cardiac and renal fibrosis models. BMP-7 is retrieved as a multi-organ anti-fibrotic biologic with beneficial effects in renal, pulmonary, and cardiovascular fibrosis models; Thrasos Innovation has filed patents on BMP agonist peptides for renal fibrosis and diabetic nephropathy. END55, an endostatin-derived recombinant fusion protein produced in plants, demonstrated anti-fibrotic effects in murine skin and lung fibrosis models and in human organ cultures. Collagen-binding peptide-conjugated micelles (CBP-micelles) and VWF-A3 domain constructs have been engineered to preferentially accumulate at fibrotic sites in lung and kidney.
Predominantly preclinical; IL-11 antibodies in early translationASOs, siRNA Lipid Nanoparticles & miRNA Delivery
ONTs offer distinct advantages for targeting otherwise undruggable fibrotic pathways. ASOs targeting TGF-β pathway components are validated by the Royal Holloway group, citing recent ASO approvals in Duchenne muscular dystrophy and SMA as platform validation. Biogen researchers used siRNA-formulated lipid nanoparticles to silence genes upregulated in liver and kidney fibrosis, identifying five novel modifiers: Egr2, Atp1a2, Fkbp10, Fstl1, and Has2 — all reducing Col1a1 expression. Co-delivery of miR-29b and germacrone via cyclic RGD-modified PEG-PLGA nanoparticles demonstrated HSC targeting and collagen suppression in CCl4-induced liver fibrosis models. Eli Lilly researchers reviewed direct lung ONT delivery strategies, noting reduced systemic side effects.
Preclinical proof-of-concept in murine modelsHDAC Inhibitors & the miR-132/PPARγ Epigenetic Relay
HDAC inhibitors are documented to suppress myofibroblast differentiation in cardiac, pulmonary, hepatic, and conjunctival fibrosis, establishing epigenetic regulation as a cross-organ mechanism. A University of Georgia Research Foundation patent describes an epigenetic relay in which loss of miR-132 leads to transcriptional silencing of PPARγ, driving hepatic stellate cell activation — with nucleoside compounds and conjugated antibodies claimed as interventions. PatSnap analytics can be used to map patent families covering HDAC isoform-selective inhibitors across organ indications.
Preclinical stageMuse Cells, hFSSC Secretome & Clinical Liver Trials
Stem and progenitor cell therapies are under active clinical investigation in liver fibrosis/cirrhosis, encompassing different cell sources, dosing, and delivery methods. Life Science Institute, Inc. (Japan) has filed a patent for SSEA-3-positive pluripotent stem cells (Muse cells) derived from mesenchymal tissue as a cell product for organ fibrosis including liver. Human fetal skin-derived stem cell (hFSSC) secretome demonstrated anti-fibrotic effects via HSC suppression and liver regeneration in CCl4-induced rat models. According to ClinicalTrials.gov, multiple stem cell trials in liver cirrhosis are registered globally.
Clinical trials ongoing (liver); earlier stage for other organsNanoparticles, Collagen-Binding Platforms & Herbal Derivatives
A dedicated literature stream addresses poor bioavailability and off-target effects via organ-targeted delivery: HSC-targeted nanoparticles loaded with drugs and nucleic acids for liver fibrosis; collagen-binding micelles and VWF-A3 domain constructs exploiting leaky vasculature for lung/kidney targeting; and machine perfusion supplementation with anti-fibrotic drugs proposed for kidney transplant interstitial fibrosis prevention. A substantial body of retrieved academic literature — predominantly from Chinese institutions — covers natural product compounds including naringenin, artemisinin, germacrone, epoxyeicosatrienoic acids, and pyrroloquinoline quinone (PQQ) evaluated across multiple organ fibrosis models at the preclinical stage. The PatSnap chemicals & materials platform supports structure-activity relationship analysis for natural product scaffolds.
Preclinical; machine perfusion approach novel for transplant settingKey Targets Appearing Across Multiple Organ Indications
The following targets appear across multiple retrieved results and multiple organ indications, signaling their relevance to cross-indication strategies. Data derived from patent filings and academic literature retrieved via PatSnap Eureka.
Identify Cross-Indication Repurposing Opportunities
PatSnap Eureka's AI search surfaces shared pathway signals across organ indications instantly.
Academic Institutions Lead Discovery; Biotech Drives IP Commercialisation
Retrieved results are overwhelmingly literature-driven (academic papers), with a smaller but strategically significant patent component reflecting growing IP commercialisation activity.
RWTH Aachen / Bonn University — SFB/TRR57 Consortium
Multiple publications spanning 13 years covering both liver and kidney fibrosis mechanisms and therapeutic modulation, representing one of the most sustained academic research programmes in the retrieved dataset.
MRC-London Institute of Medical Sciences
Identified IL-11 as an unexpected central mediator of cardiac and renal fibroblast activation. Demonstrated that genetic deletion of IL-11 receptor (Il11ra1) is protective in mouse models — a finding with significant therapeutic implications for neutralizing antibody development.
University of Chicago & University of Pittsburgh
Engineered collagen-targeting therapeutics (CBP-micelles, VWF-A3 domain constructs, END55 recombinant fusion protein) demonstrating anti-fibrotic effects in murine skin and lung fibrosis models and in human organ cultures — advancing targeted delivery as a cross-organ platform.
Biogen, Eli Lilly & Boehringer Ingelheim
Pharmaceutical contributors include Biogen (siRNA LNP identification of five novel fibrosis modifiers: Egr2, Atp1a2, Fkbp10, Fstl1, Has2), Eli Lilly (pulmonary ONT delivery review), and Boehringer Ingelheim (nintedanib clinical development challenges), reflecting industry engagement with both established and emerging modalities.
Accelerate Cross-Indication Fibrosis Research with AI-Native Intelligence
The fibrosis drug pipeline challenge is fundamentally a data problem: shared pathway signals are buried across thousands of organ-specific patent families, literature reviews, and clinical records. PatSnap Eureka applies AI to surface cross-indication connections that would take research teams months to identify manually.
For fibrosis specifically, Eureka enables teams to identify which TGF-β pathway inhibitors have been claimed across multiple organ indications, track assignee patent filing velocity for emerging modalities like oligonucleotide therapies, and map the competitive white space between approved agents (pirfenidone, nintedanib) and the preclinical pipeline. The PatSnap customer base includes leading pharmaceutical and biotech organisations leveraging these capabilities for indication expansion decisions.
Bioinformatics approaches have already demonstrated the value of cross-indication analysis: a drug repurposing pipeline applied across nine fibrotic diseases by the Cyprus Institute of Neurology and Genetics identified unique and shared genes and candidate repurposed substances. PatSnap Eureka enables R&D teams to run equivalent analyses against a live, continuously updated database of 2B+ data points — as documented by WHO-aligned global disease burden frameworks and supported by PatSnap's open API for programmatic data access.
Fibrosis Drug Pipeline — Key Questions Answered
The central cellular effector in all five indications is the myofibroblast, generated primarily through activation of hepatic stellate cells (HSCs) in the liver, portal fibroblasts, and analogous resident fibroblast populations in lung, kidney, heart, and skin. Once activated, myofibroblasts synthesize excess extracellular matrix — predominantly collagens I, III, and fibronectin — leading to progressive architectural disruption and organ failure.
Pirfenidone and nintedanib are approved only for idiopathic pulmonary fibrosis (IPF). No drug carries regulatory approval specifically for liver, kidney, cardiac, or dermal fibrosis. Nintedanib is also approved for systemic sclerosis-associated ILD.
TGF-β/Smad signaling is identified as the dominant pro-fibrotic cytokine axis across liver, lung, kidney, heart, and skin fibrosis in multiple retrieved results. TGF-β1 activates Smad2/3 transcriptional programs, driving collagen synthesis and myofibroblast persistence. Retrieved results consistently note that indiscriminate inhibition of TGF-β carries systemic risk given its pleiotropic biology, creating demand for pathway-selective or tissue-targeted interventions.
IL-11 has been identified as an unexpected central mediator specifically in cardiac and renal fibroblast activation. Mouse models demonstrate that IL-11 overexpression drives heart and kidney fibrosis, while genetic deletion of the IL-11 receptor (Il11ra1) is protective. IL-11 neutralizing antibodies targeting IL-11 or IL-11RA have been developed and show anti-fibrotic activity in human fibroblasts and murine cardiac and renal fibrosis models.
Oligonucleotide therapies (ONTs) include antisense oligonucleotides (ASOs) targeting TGF-β pathway components, siRNA-formulated lipid nanoparticles (used by Biogen researchers to identify five novel fibrosis modifiers including Egr2, Atp1a2, Fkbp10, Fstl1, Has2), and miRNA-based delivery such as co-delivery of miR-29b and germacrone via cyclic RGD-modified nanoparticles for liver fibrosis. These are predominantly at the preclinical proof-of-concept stage in murine models.
Stem and progenitor cell therapies are under active clinical investigation in liver fibrosis/cirrhosis, with clinical trial summaries encompassing different cell sources, dosing, and delivery methods. Life Science Institute, Inc. (Japan) has filed a patent for SSEA-3-positive pluripotent stem cells (Muse cells) derived from mesenchymal tissue as a cell product for organ fibrosis, including liver fibrosis. For organ fibrosis broadly, these approaches remain at an earlier stage.
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References
- Research progress on drugs targeting the TGF-β signaling pathway in fibrotic diseases — Central South University, 2022
- Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides — Royal Holloway, University of London, 2018
- Integrins and cadherins as therapeutic targets in fibrosis — Baylor College of Medicine, 2014
- Pan-Lysyl Oxidase Inhibitor PXS-5505 Ameliorates Multiple-Organ Fibrosis — McMaster University, 2022
- IL-11 in cardiac and renal fibrosis: Late to the party but a central player — MRC-London Institute of Medical Sciences, 2020
- The Role of Nrf2 in Pulmonary Fibrosis: Molecular Mechanisms and Treatment Approaches — Tongji University, 2022
- HDAC Inhibitors: Therapeutic Potential in Fibrosis-Associated Human Diseases — Chonnam National University, 2019
- HIPK2 is a new drug target for anti-fibrosis therapy in kidney disease — James J. Peter Veterans Administration Medical Center, 2015
- The pan-PPAR agonist lanifibranor reduces development of lung fibrosis and attenuates cardiorespiratory manifestations — UCL Division of Medicine, 2021
- Antifibrotic effect of pirfenidone in a mouse model of human nonalcoholic steatohepatitis — Tokyo Medical and Dental University, 2017
- Identification of Novel Fibrosis Modifiers by In Vivo siRNA Silencing — Biogen, Inc., 2017
- Co-delivery of miR-29b and germacrone based on cyclic RGD-modified nanoparticles for liver fibrosis therapy — Wenzhou Medical University, 2020
- The Promising Therapeutic Potential of Oligonucleotides for Pulmonary Fibrotic Diseases — Eli Lilly and Company, 2022
- Engineered collagen-targeting therapeutics reverse lung and kidney fibrosis in mice — University of Chicago, 2022
- Ameliorating Fibrosis in Murine and Human Tissues with END55 — University of Pittsburgh, 2022
- Stem cells for treatment of liver fibrosis/cirrhosis: clinical progress and therapeutic potential — Lanzhou University First Hospital, 2022
- Common pathway signature in lung and liver fibrosis — Johns Hopkins University, 2016
- Drug Repurposing Through a Bioinformatics Pipeline Applied on Fibrotic Diseases — Cyprus Institute of Neurology and Genetics, 2020
- Pathologic Proteolytic Processing of N-Cadherin as a Marker of Human Fibrotic Disease — Duke University Medical Center, 2022
- Naringenin: A Promising Therapeutic Agent against Organ Fibrosis — Southwest Medical University, 2021
- Artemisinin and artemisinin derivatives as anti-fibrotic therapeutics — Northwestern University-Feinberg, 2021
- National Institutes of Health (NIH) — Fibrosis Research Resources
- World Health Organization (WHO) — Global Disease Burden Framework
- ClinicalTrials.gov — Registered Fibrosis Clinical Trials
All data and statistics on this page are sourced from the references above and from PatSnap's proprietary innovation intelligence platform. This page is derived from a limited set of patent and literature records retrieved across targeted searches and represents a snapshot of innovation signals within this dataset only.
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