Expert Opinion on Therapeutic Patents最新文献

Introduction: Factor XII (FXII) is a liver-derived plasma zymogen that autoactivates on anionic surfaces to FXIIa, which drives the contact blood coagulation pathway, kallikrein-kinin signaling, fibrinolysis, and classical complement. Although congenital FXII(a) deficiency is largely asymptomatic, dysregulated activity is linked to thrombosis, hereditary angioedema (HAE), and neuroinflammation, making FXII(a) an attractive therapeutic target.

Areas covered: This review provides a brief overview of FXII/FXIIa structure and function to highlight its suitability as a therapeutic target. It then summarizes patents published between 2020 and 2025 (patent search using Espacenet, Google Patents, and SciFinder) covering FXII(a)-targeting agents across diverse modalities, including small molecules, proteins and peptides, monoclonal antibodies, oligonucleotides, and siRNAs.

Expert opinion: Patent analysis indicates that most FXII(a) inhibitors remain in early preclinical development, though a growing subset has shown in vivo efficacy in models of thrombosis, HAE, sepsis, and neuroinflammation. The breadth and pace of 2020-2025 filings, together with accumulating translational data, should accelerate progression from patents to clinical candidates, particularly for contact-activation indications (e.g. device-related thrombosis). Resolving full-length FXII/α-FXIIa structures would further enable allosteric inhibitors design.

Introduction: Over the past decade, significant efforts have been made to develop and patent selective fatty acid amide hydrolase (FAAH) inhibitors with favorable pharmacokinetic and safety profiles to modulate pain, inflammation, and neurological disorders. Recently, attention has shifted toward dual inhibitors that combine FAAH inhibition with other targets, such as COXs, MAGL, and sEH, aiming to improve therapeutic outcomes. This review highlights the most significant patents from the last 10 years in this evolving field of research.

Areas covered: Patents of selective and nonselective FAAH inhibitors published from 2015 to 2025. Patent searches were conducted on Espacenet, WIPO (World Intellectual Property Organization), and Google Patents databases, while literature search was performed using the Artificial Intelligence (AI) visual tools Connected Papers and Research Rabbit.

Expert opinion: The search for novel and clinically relevant FAAH inhibitors starts with newly disclosed chemical entities. However, reducing translation gaps also requires advances in identifying key biomarkers and developing relevant animal models that mimic target diseases. Additionally, disclosing of innovative chemical templates, including those for allosteric modulation of FAAH, and the identification of suitable and innovative multitarget directed ligands will likely establish FAAH inhibitors as a validated therapeutic option for several diseases.

Introduction: Alzheimer's disease (AD) is a multifactorial neurodegenerative disorder with a complex and not fully elucidated etiology. An exponential rise in its incidence underscores the urgent need for effective therapeutic strategies. AD imposes a significant economic burden on public healthcare systems and on patient's families.

Areas covered: This manuscript focuses on the review of potent acetylcholinesterase (AChE) inhibitors, either through chemical synthesis or isolation from natural sources, aimed at restoring acetylcholine levels. Most of the compounds discussed act as multitarget agents and are categorized into four groups: drug derivatives (9 patents), heterocyclic scaffolds (16 patents), natural products from plant extracts (12 patents), and synthetic compounds inspired by natural templates (18 patents).

Expert opinion: AChE inhibition remains a compelling target in AD drug design, as it enhances acetylcholine levels and can alleviate cognitive decline. Furthermore, inhibitors that interact with the peripheral anionic site (PAS) of AChE may reduce β-amyloid self-aggregation, thereby preventing the deposition of neurotoxic peptides in the brain. However, targeting AChE alone is insufficient for the development of effective therapeutics. A multitarget approach, combining AChE inhibition with pharmacophores addressing β-amyloid aggregation, neuroinflammation, oxidative stress, and other pathological hallmarks, holds greater promise for the development of more efficient anti-Alzheimer's agents.

Introduction: Bromodomain-containing protein 9 (BRD9) is an epigenetic reader component of the non-canonical BAF (BRG1/BRM-Associated Factors) chromatin remodeling complex, involved in the regulation of transcription. The ncBAF complex differs from the other two complexes, the canonical BAF and PBAF, as it contains unique subunits encoded by genes including BRD9, GLTSCR1 (Glioma Tumor Suppressor Candidate Region 1), and GLTSCR1L (GLTSCR1-Like). In recent years, BRD9 has emerged as a promising therapeutic target in several diseases.

Areas covered: This review explores the most compelling patents released from 2019 to 2025 concerning compounds, classified as small molecules and protein degraders (PROTACs), interfering with BRD9 activity. Relevant patents were identified through searches in European Patent Office (EPO) and World Intellectual Property Organization WIPO databases.

Expert opinion: The patent landscape reflects a growing interest in BRD9 as an epigenetic target for its key role in various pathologies. The recent patent data show how selective BRD9 degraders represent a significant step forward in terms of efficacy and selectivity, with promising results in preclinical models of acute myeloid leukemia (AML), synovial sarcoma (SS), and Huntington's disease (HD). Despite several critical issues, the selective degradation of this epigenetic target shows great potential to be an innovative therapeutic strategy.

Introduction: LAG-3 is a molecule overexpressed on the surface of CD4+ and CD8+ cells in the tumor microenvironment that prevents T-cell activation and production of IL-2 and IFN-γ.

Areas covered: Using the patent databases of the State Patent and Trademark Office, the European Patent Office, the World Intellectual Property Organization, the Japanese Patent Office, the State Intellectual Property Office of China, and the Korean Intellectual Property Office, a detailed patent landscape of LAG-3 antagonists was generated, categorizing them as monospecific anti-LAG-3 antibodies, multispecific anti-LAG-3 antibodies, LAG-3 binding peptides, LAG-3 binding small molecules, and LAG-3 therapeutics.

Expert opinion: The trend shows that monospecific antibodies against LAG-3 continue to be the main antagonists, followed by multispecific t, treatment methods using known LAG-3 antagonists, LAG-3 binding peptides, and LAG-3 binding small molecules. The monospecific antibodies encelimab, miptenalimab, and the bispecific antibodies tobemstomig, IBI323, ABL501, fanastomig, and FS118 are added, during this period, to the potential drugs targeting LAG-3.

Introduction: Securing patents in multiple countries has become essential for the development of global medical products. However, differences in national patent systems result in varying patentability standards. Although global claim construction strategies have been applied in practice, these approaches have not yet been systematically organized.

Area covered: This study examines how the patent scope for patent families of international applications related to genome editing technologies filed in 2013, differs between Japan, where medical method patents are prohibited, and the United States, where such patents are permitted.

Expert opinion: For CRISPR-Cas system patents, claim structures varied significantly, even among the corresponding family patents. To navigate these differences, the following strategies were proposed for filing patents in countries that prohibit medical method patents such as Japan: Convert medical method claims in the U.S. into composition claims that include product inventions, as this process ensures that such claims allow for the enforcement of rights against the suppliers of infringing products.Clearly define the scope of the claimed use-inventions when specifying the characteristics of the product based on its effects.Explicitly describe cells produced by a specific manufacturing method within the claimed rights.

Introduction: With β-lactams remaining the most widely prescribed antibacterials worldwide, their continuing clinical efficacy remains an important therapeutic goal. Rapid spread of serine and metallo-ß-lactamases (SBLs and MBLs, respectively), which can inactivate β-lactams, is increasingly threatening this objective. Finding clinically useful inhibitors of MBLs, for which no FDA- carbapenem-resistant infections approved treatment currently exists, is of interest.

Areas covered: This article concisely reviews structurally novel xeruborbactam-inspired tricyclic boronate SBL/MBL inhibitors (as reported in US 2025/0223303) with promising inhibitory activities in vitro. The literature search was conducted using SciFinder and Patentscope. By introducing novel thioether-based C5 sidechains onto the previously optimized bicyclic boronate core, the inventors explored novel chemical space yielding SBL/MBL inhibitors with promising activities against carbapenem-resistant (CR) Escherichia coli, Klebsiella pneumoniae, and, importantly, Acinetobacter baumannii, when used in combination with meropenem and/or biapenem (at least with respect to taniborbactam, i.e. boronate inhibitor in late-stage clinical development).

Expert opinion: Due to the major societal importance of β-lactams for modern medicine, and the clearly demonstrated clinical potential of functionalized (bi)cyclic boronates as potent dual-acting SBL/MBL inhibitors when used in combination therapies, there is ample opportunity and scope for continued investigation of this pharmacophore, particularly in the context of discovering new therapeutic options for CR infections.

Introduction: CARM1 (Coactivator-associated arginine methyltransferase 1), also known as PRMT4 (Protein Arginine Methyltransferase 4), is a type I PRMT that regulates gene expression by methylating both histone and non-histone substrates. Its overexpression and functional dysregulation have been linked to the progression of several cancer types, including breast, prostate, and hematological malignancies, positioning CARM1 as a compelling target for therapeutic intervention. In this scenario, the development of selective and potent CARM1 inhibitors holds great promise for the treatment of cancer by modulating epigenetic pathways and altering oncogenic transcriptional programs. However, designing effective inhibitors is challenging due to the conserved nature of the PRMT catalytic domain and the need for high selectivity to minimize off-target effects.

Areas covered: This review aims at giving an overview of the recent patent literature of CARM1 inhibitors between 2018 and 2025. WIPO, EPO, USPTO, and SciFinder databases were used for the search of patents.

Expert opinion: Although the development of selective CARM1 inhibitors presents significant challenges, it remains a highly promising endeavor due to its potential to greatly advance anticancer drug discovery. Various strategies, including PROTACs can be employed to inactivate the protein, leading to antitumor effects.

Introduction: Inhibition of protein tyrosine phosphatase 1B (PTP1B) is a key strategy for improving insulin sensitivity in various cells. This strategy is supported by human genetic data. PTP1B inhibitors are considered an attractive target for the treatment of T2DM because they improve insulin receptor sensitivity and have the ability to reverse insulin resistance-related diseases.

Areas covered: This review provides an overview of the patents that were published between January 2019 and December 2023. The efficacy of potent PTP1B inhibitors for the treatment of T2DM is described in this review. The latest patented studies of PTP1B inhibitors (are summarized by using the keywords 'PTP1B inhibitors,' in PubMed, SciFinder, and Google Patents.

Expert opinion: There has been tremendous progress in PTP1B drug discovery. Progress has been made with natural products, semi-synthetic natural product derivatives and heterocyclic hybrid compounds. A number of protocols are being pursued in order to enhance the biological effects of PTP1B inhibitors. In addition, these new advances suggest that it may be possible to obtain small-molecule inhibitors of PTP1B with the required potency and selectivity. In addition, future efforts using an integrated medicinal chemistry and structural biology strategy are expected to lead to potent and selective PTP1B inhibitors.

Introduction: Galectin-1 (Gal-1) & Galectin-3 (Gal-3) are carbohydrate-binding proteins implicated in diverse pathological processes. These galectins have emerged as key therapeutic targets, with significant presence in drug discovery pipelines and clinical investigations. Their unique glycan-binding properties and immunomodulatory effects have prompted the development of inhibitors to modulate their function.

Areas covered: This review presents a comprehensive summary of 19 small-molecule patents targeting Gal-1 and Gal-3, published from January 2022 to June 2025. A systematic search of Reaxys and WIPO databases yielded patents spanning modified monosaccharides & disaccharides, non-carbohydrate chemotypes and metal-coordinated complexes.

Expert opinion: The patent landscape is dominated by β-D-galactopyranose and thiodigalactoside scaffolds, with limited innovation and unresolved issues in selectivity, drug-likeness, and efficacy, highlighted by GB0139's Phase II failure. Moreover, only a few patents provide structural evidence and strong in vitro/in vivo data, limiting confidence in their therapeutic outcome. By contrast, some diversification has emerged, including spirocyclic sugars, ruthenium-conjugates and non-carbohydrate ligands such as heterocycles and repurposed drugs. These highlight underexploited avenues with promise. Yet, robust biochemical data and structural proof of binding remain scarce. To move the field forward, future patents must diversify chemotypes, substantiate binding modes with crystallography or NMR, and demonstrate translation in relevant disease models.