AI Drug Discovery for Pharma and Biotech

Drug discovery

12

drugs

With orphan designations

Overview

A non-atherosclerotic, inflammatory vascular disorder affecting small- and medium-sized arteries and veins, strongly associated with tobacco use. Presents with distal limb ischemia, claudication, rest pain, ulcers, or gangrene. Diagnosis requires exclusion of diabetes, autoimmune diseases, and hypercoagulable states. Smoking cessation is the only definitive therapy; untreated cases risk progressive tissue necrosis [1][5][9][14].

Population

  • Primarily affects males aged 20–45 years (≥95% smokers), though incidence in females is rising [1][5][16].

  • More prevalent in regions with high tobacco use (Middle East, Asia, Eastern Europe) [2][14].

Burden

  • Amputation rates reach 20% despite treatment; recurrence common with continued smoking [2][5][10].

  • Causes severe disability due to chronic pain, infections, and limb loss, though life expectancy remains unaffected [1][6][16].

Therapies

  • First-line: Complete tobacco cessation (prevents progression and amputations) [1][9].

  • Pharmacotherapy: IV iloprost (superior to aspirin for pain/ulcer healing), oral bosentan (limited evidence) [3][11].

  • Adjunctive: Wound care, analgesia, and spinal cord stimulation for refractory pain [2][19].

Categories: rare circulatory system diseases, rare renal diseases, rare systemic and rheumatological diseases, rare transplant-related disorders

Research Papers

828 drug discovery papers about Buerger disease, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

828 drug discovery papers about Buerger disease, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:

2026-08-03 | [Exploration of effective substances and mechanisms of Corydalis Herba for treating thromboangiitis obliterans based on UHPLC-Q-TOF-MS/MS, network pharmacology, and molecular docking analyses].

Corydalis Herba is a traditional Tibetan medicine clinically applied to treat vasculitis and related conditions. Although many kinds of compounds have been isolated and identified from Corydalis Herba, there is no systematic mass spectrometry(MS) research on it, and its effective substances and mechanisms for treating thromboangiitis obliterans remain unknown. Therefore, one of the origins of Corydalis Herba, i.e., the dried herb of Corydalis hendersonii(CH), was used in this study, and ultra high performance liquid chromatography-quadrupole time-of-flight tandem mass spectrometry(UHPLC-Q-TOF-MS/MS), network pharmacology, and molecular docking methods were used to investigate its chemical constituents as well as its effective substances and mechanisms for treating thromboangiitis obliterans. A total of 107 chemical constituents of CH were identified, and 17 of them were unequivocally identified by comparison with reference compounds. The 107 compounds comprise 74 alkaloids, 10 flavonoids, 14 organic acids, 7 amides, and 2 amines, and 89 of them were new constituents of Corydalis Herba, including 8 potential new compounds. In addition, the electrospray ionization(ESI) MS fragmentation patterns of different types of benzyltetrahydroisoquinoline alkaloids were proposed. Finally, 16 core targets(SERPINE1, ICAM1, MMP9, VCAM1, SELE, F3, MPO, etc.), 85 active ingredients, 26 effective substances, and 10 key effective substances(protopine, naringenin, berberine, tetrahydropalmatine, kaempferol, higenamine, apigenin, luteolin, quercetin, and tetrahydrocoptisine) of CH in the treatment of thromboangiitis obliterans were determined by network pharmacology, molecular docking analyses, and literature retrieval. The results lay a solid foundation for further research on the quality control, pharmacological effects, mechanisms, and in vivo process of Corydalis Herba.

Open article ↗



2026-07-10 | Systemic Exposure and Multicomponent Pharmacokinetics of Mailuoning Oral Liquid: Integrated Ultra-High-Performance Liquid Chromatography-High-Resolution Mass Spectrometry Profiling and Ultra-High-Performance Liquid Chromatography-Tandem Mass Spectrometry Quantification.

Mailuoning oral liquid (MLN) is a state-approved multi-herb formula that has been prescribed for 25 years to clear heat, nourish yin, invigorate blood, and resolve stasis. Although randomized controlled trials and real-world evidence support its efficacy in thromboangiitis obliterans, deep-vein thrombosis, and the convalescent phase of cerebral infarction, a systematic search identified no pharmacokinetic data on its bioactive constituents. This lack of pharmacokinetic data impedes evidence-based dose refinement and international acceptance. Here, we employed ultra-high-performance liquid chromatography with Quadrupole-Exactive Orbitrap high-resolution mass spectrometry (UHPLC-Q-Exactive Orbitrap HRMS) to track 39 MLN-derived compounds in rat plasma, intestinal contents, and 10 tissues; 15 with prominent systemic exposure were designated as key pharmacokinetic markers. A selective and sensitive UHPLC-tandem MS assay was subsequently developed and fully validated for simultaneous quantification of these 15 circulating components. The assay exhibited excellent linearity (r ≥ 0.9974) for all analytes, with precision, accuracy, extraction recovery, and matrix effects meeting standard bioanalytical validation criteria. All compounds demonstrated stability under the tested storage conditions. Following oral administration of MLN at 8.1 and 16.2 mL·kg-1 in rats, all 15 components were rapidly absorbed, reaching peak plasma concentrations within approximately 1 h. Systemic exposure increased in a clear dose-dependent manner, with structurally related compounds showing similar pharmacokinetic profiles. Notably, the iridoids secoxyloganin, harpagide, and secologanic acid yielded the highest AUC0-24 h values, indicating they are the principal systemic exposure markers. This study establishes the first comprehensive pharmacokinetic profile for MLN and provides a robust, validated analytical platform. By creating a crucial link between quantitative systemic exposure and efficacy, our work supplies a scientific foundation for refining dosage regimens, establishing quality specifications, and promoting the evidence-based clinical application of this widely prescribed traditional Chinese medicine formula.

Open article ↗



2026-07-01 | Preliminary clinical observation of a novel anticoagulation regimen in the acute phase of thromboangiitis obliterans (Buerger's disease)

Preliminary clinical observation of a novel anticoagulation regimen in the acute phase of thromboangiitis obliterans (Buerger's disease)

Open article ↗



2026-06-26 | BUERGER’S DISEASE - A GENERAL OVERVIEW WITH A FOCUS ON NEW THERAPEUTIC APPROACHES

Introduction: Buerger's disease (thromboangiitis obliterans, TAO) is an inflammatory disease affecting small and medium-sized blood vessels, primarily in the extremities. This condition leads to their gradual narrowing and occlusion, resulting in impaired blood flow and tissue ischemia. It most often affects young adults, particularly men. It is strongly associated with tobacco use, which is considered a key risk factor for disease development and progression. Clinical presentation includes limb pain, intermittent claudication, ulceration, and, in advanced cases, tissue necrosis. Although treatment is primarily centered on smoking cessation and symptom relief, recent advances in therapeutic approaches show promising potential. Purpose: The aim of this review is to discuss Buerger's disease in the context of its pathogenesis, diagnosis, and clinical presentation, and to analyze current therapeutic options. Methodology: A narrative literature review was conducted in PubMed to identify studies on thromboangiitis obliterans. The search strategy included the keywords "Buerger’s disease" (accounting for variant - "Buerger disease"), "thromboangiitis obliterans" and "treatment". Additionally, reference lists of selected articles were screened to identify further relevant publications. Studies published between 2000 and 2025 and directly related to the topic were included. Conclusions: Advances in the understanding and management of Buerger’s disease have led to the development of new therapeutic approaches. However, current evidence remains limited, and further well-designed studies are needed to establish optimal treatment strategies and improve patient outcomes.

Open article ↗



2026-05-22 | Buerger’s Disease- Ayurved Chikista -Case Study

Buerger’s disease is a segmental, progressive, occlusive, inflammatory disease of small and medium sized vessels with superficial thrombophlebitis often may present as Raynaud’s phenomenon with micro abscesses, along with neutrophil and giant cell infiltration, with skip lesions. Buerger's disease[Thromboangiitis Obliterans ] is a non-atherosclerotic inflammatory disease of unknown etiology, which has a strong association with tobacco. More common in lower limbs than upper limbs Incidence is Very commonly seen in young and middle aged males (20 – 40 yrs.) Seen only in smokers and tobacco users . Hormonal influence, familial factors Hypersensitivity to cigarette, Altered autonomic functions, Lower socioeconomic group, Recurrent minor feet injuries, Poor hygiene. Pathogenesis Smoke- carbon monoxide and nicotinic acid Vasospasm &hyperplasia of intima Thrombosis of vessels Obliteration of vessels Panarteritis – segmental Artery ,vein and nerve are together involved ,Nerve involvement due to blockage of vasa nervosa causes rest pain . Artery involvement leads to features like claudication pain. With blockage – plenty of collaterals open up Collaterals maintain the blood supply to the ischemic area this is known as compensatory peripheral vascular disease. Disease progression due to smoking leads to blockage of collaterals also leading to severe ischemia known as decompensatory peripheral vascular disease or critical limb ischemia. we present here one case successfully treated with the help of classical ayurveda management.

Open article ↗



2026-08-03 | [Exploration of effective substances and mechanisms of Corydalis Herba for treating thromboangiitis obliterans based on UHPLC-Q-TOF-MS/MS, network pharmacology, and molecular docking analyses].

Corydalis Herba is a traditional Tibetan medicine clinically applied to treat vasculitis and related conditions. Although many kinds of compounds have been isolated and identified from Corydalis Herba, there is no systematic mass spectrometry(MS) research on it, and its effective substances and mechanisms for treating thromboangiitis obliterans remain unknown. Therefore, one of the origins of Corydalis Herba, i.e., the dried herb of Corydalis hendersonii(CH), was used in this study, and ultra high performance liquid chromatography-quadrupole time-of-flight tandem mass spectrometry(UHPLC-Q-TOF-MS/MS), network pharmacology, and molecular docking methods were used to investigate its chemical constituents as well as its effective substances and mechanisms for treating thromboangiitis obliterans. A total of 107 chemical constituents of CH were identified, and 17 of them were unequivocally identified by comparison with reference compounds. The 107 compounds comprise 74 alkaloids, 10 flavonoids, 14 organic acids, 7 amides, and 2 amines, and 89 of them were new constituents of Corydalis Herba, including 8 potential new compounds. In addition, the electrospray ionization(ESI) MS fragmentation patterns of different types of benzyltetrahydroisoquinoline alkaloids were proposed. Finally, 16 core targets(SERPINE1, ICAM1, MMP9, VCAM1, SELE, F3, MPO, etc.), 85 active ingredients, 26 effective substances, and 10 key effective substances(protopine, naringenin, berberine, tetrahydropalmatine, kaempferol, higenamine, apigenin, luteolin, quercetin, and tetrahydrocoptisine) of CH in the treatment of thromboangiitis obliterans were determined by network pharmacology, molecular docking analyses, and literature retrieval. The results lay a solid foundation for further research on the quality control, pharmacological effects, mechanisms, and in vivo process of Corydalis Herba.

Open article ↗



2026-07-10 | Systemic Exposure and Multicomponent Pharmacokinetics of Mailuoning Oral Liquid: Integrated Ultra-High-Performance Liquid Chromatography-High-Resolution Mass Spectrometry Profiling and Ultra-High-Performance Liquid Chromatography-Tandem Mass Spectrometry Quantification.

Mailuoning oral liquid (MLN) is a state-approved multi-herb formula that has been prescribed for 25 years to clear heat, nourish yin, invigorate blood, and resolve stasis. Although randomized controlled trials and real-world evidence support its efficacy in thromboangiitis obliterans, deep-vein thrombosis, and the convalescent phase of cerebral infarction, a systematic search identified no pharmacokinetic data on its bioactive constituents. This lack of pharmacokinetic data impedes evidence-based dose refinement and international acceptance. Here, we employed ultra-high-performance liquid chromatography with Quadrupole-Exactive Orbitrap high-resolution mass spectrometry (UHPLC-Q-Exactive Orbitrap HRMS) to track 39 MLN-derived compounds in rat plasma, intestinal contents, and 10 tissues; 15 with prominent systemic exposure were designated as key pharmacokinetic markers. A selective and sensitive UHPLC-tandem MS assay was subsequently developed and fully validated for simultaneous quantification of these 15 circulating components. The assay exhibited excellent linearity (r ≥ 0.9974) for all analytes, with precision, accuracy, extraction recovery, and matrix effects meeting standard bioanalytical validation criteria. All compounds demonstrated stability under the tested storage conditions. Following oral administration of MLN at 8.1 and 16.2 mL·kg-1 in rats, all 15 components were rapidly absorbed, reaching peak plasma concentrations within approximately 1 h. Systemic exposure increased in a clear dose-dependent manner, with structurally related compounds showing similar pharmacokinetic profiles. Notably, the iridoids secoxyloganin, harpagide, and secologanic acid yielded the highest AUC0-24 h values, indicating they are the principal systemic exposure markers. This study establishes the first comprehensive pharmacokinetic profile for MLN and provides a robust, validated analytical platform. By creating a crucial link between quantitative systemic exposure and efficacy, our work supplies a scientific foundation for refining dosage regimens, establishing quality specifications, and promoting the evidence-based clinical application of this widely prescribed traditional Chinese medicine formula.

Open article ↗



2026-07-01 | Preliminary clinical observation of a novel anticoagulation regimen in the acute phase of thromboangiitis obliterans (Buerger's disease)

Preliminary clinical observation of a novel anticoagulation regimen in the acute phase of thromboangiitis obliterans (Buerger's disease)

Open article ↗



2026-06-26 | BUERGER’S DISEASE - A GENERAL OVERVIEW WITH A FOCUS ON NEW THERAPEUTIC APPROACHES

Introduction: Buerger's disease (thromboangiitis obliterans, TAO) is an inflammatory disease affecting small and medium-sized blood vessels, primarily in the extremities. This condition leads to their gradual narrowing and occlusion, resulting in impaired blood flow and tissue ischemia. It most often affects young adults, particularly men. It is strongly associated with tobacco use, which is considered a key risk factor for disease development and progression. Clinical presentation includes limb pain, intermittent claudication, ulceration, and, in advanced cases, tissue necrosis. Although treatment is primarily centered on smoking cessation and symptom relief, recent advances in therapeutic approaches show promising potential. Purpose: The aim of this review is to discuss Buerger's disease in the context of its pathogenesis, diagnosis, and clinical presentation, and to analyze current therapeutic options. Methodology: A narrative literature review was conducted in PubMed to identify studies on thromboangiitis obliterans. The search strategy included the keywords "Buerger’s disease" (accounting for variant - "Buerger disease"), "thromboangiitis obliterans" and "treatment". Additionally, reference lists of selected articles were screened to identify further relevant publications. Studies published between 2000 and 2025 and directly related to the topic were included. Conclusions: Advances in the understanding and management of Buerger’s disease have led to the development of new therapeutic approaches. However, current evidence remains limited, and further well-designed studies are needed to establish optimal treatment strategies and improve patient outcomes.

Open article ↗



2026-05-22 | Buerger’s Disease- Ayurved Chikista -Case Study

Buerger’s disease is a segmental, progressive, occlusive, inflammatory disease of small and medium sized vessels with superficial thrombophlebitis often may present as Raynaud’s phenomenon with micro abscesses, along with neutrophil and giant cell infiltration, with skip lesions. Buerger's disease[Thromboangiitis Obliterans ] is a non-atherosclerotic inflammatory disease of unknown etiology, which has a strong association with tobacco. More common in lower limbs than upper limbs Incidence is Very commonly seen in young and middle aged males (20 – 40 yrs.) Seen only in smokers and tobacco users . Hormonal influence, familial factors Hypersensitivity to cigarette, Altered autonomic functions, Lower socioeconomic group, Recurrent minor feet injuries, Poor hygiene. Pathogenesis Smoke- carbon monoxide and nicotinic acid Vasospasm &hyperplasia of intima Thrombosis of vessels Obliteration of vessels Panarteritis – segmental Artery ,vein and nerve are together involved ,Nerve involvement due to blockage of vasa nervosa causes rest pain . Artery involvement leads to features like claudication pain. With blockage – plenty of collaterals open up Collaterals maintain the blood supply to the ischemic area this is known as compensatory peripheral vascular disease. Disease progression due to smoking leads to blockage of collaterals also leading to severe ischemia known as decompensatory peripheral vascular disease or critical limb ischemia. we present here one case successfully treated with the help of classical ayurveda management.

Open article ↗



Access all drug discovery papers and probability of success in trials forecasts:

Access all drug discovery papers and probability of success in trials forecasts:

Drug Discovery Landscape

12 orphan drug designations for Buerger disease.

12 orphan drug designations for Buerger disease.

Drug

Therapy type

Regulator

Orphan designation

Approval

Sponsor

circular RNA therapy expressing Hepatocyte Growth Factor

RNAs

FDA

2025-10-23

Shanghai CirCode Biomed Co., Ltd.

Granulocyte colony-stimulating-factor (G-CSF)

proteins

FDA

2022-06-03

Vasogenesis Inc

E-selectin/Adeno-Associated Virus (E-sel/AAV2) Gene Therapy

gene therapies

FDA

2021-10-12

Ambulero Inc

GCSF-mobilized autologous CD34 cells for intramuscular injection

cell therapies

FDA

2021-03-04

Lisata Therapeutics

fully human nicotine-specific monoclonal antibody

antibodies

FDA

2018-08-22

Antidote Therapeutics, Inc.

Autologous adipose tissue-derived mesenchymal stem cells [VascoStem]

cell therapies

EMA

2017-03-20

SPC GmbH

autologous adipose tissue derived mesenchymal stem cells

cell therapies

FDA

2016-05-04

Biostar Stem Cell Research Institute

Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells

cell therapies

EMA

2015-05-21

Voisin Consulting Life Sciences

allogeneic ex-vivo expanded placental adherent stromal cells

cell therapies

FDA

2011-08-22

Pluri Biotech Ltd.

Autologous bone marrow-derived mononuclear cell fraction

cell therapies

EMA

2010-09-20

t2cure GmbH

Bone marrow-derived mononuclear cells

cell therapies

FDA

2010-05-17

t2cure GmbH

pVGI.1(VEGF2)

gene therapies

FDA

1999-11-09

Corautus Genetics, Inc.

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228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.

Explority AI logo

228 Park Ave S,
New York, USA.

At Explority, we build first-of-its-kind AI to bring clarity to the earliest and riskiest stages of pharmaceutical research by forecasting which therapies are most likely to succeed. Explority AI web and mobile applications are properties of the Explority AI Inc., a company registered in the United States (File No. 10320493).
For all questions: support@explority.ai

Copyright © 2026 Explority AI Inc.