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Phage Display Derived Monoclonal Antibodies: From Bench to Bedside.

Monoclonal antibodies (mAbs) have become one of the most important classes of biopharmaceutical products, and they continue to dominate the universe of biopharmaceutical markets in terms of approval and sales. They are the most profitable single product class, where they represent six of the top ten selling drugs. At the beginning of the 1990s, an in vitro antibody selection technology known as antibody phage display was developed by John McCafferty and Sir. Gregory Winter that enabled the discovery of human antibodies for diverse applications, particularly antibody-based drugs. They created c

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… In 2018, lanadelumab has received the US FDA approval for the prevention of the angioedema attacks in patients with hereditary angioedema. … Currently, it is being evaluated in two active phase III clinical trials to prevent hereditary angioedema attacks in pediatric patients as well as in adolescent and adult patients suffering from acquired angioedema (AAE) due to C1-INH deficiency ( NCT04070326 and NCT04206605 ). …

Structure and function of therapeutic antibodies approved by the US FDA in 2025.

In 2025, the 50th anniversary of the invention of hybridoma technology, the United States Food and Drug Administration (US FDA) approved a total of 44 new molecular entities (NMEs), of which 10 were therapeutic antibody-based molecules. Additionally, the US FDA approved 31 small molecules (of which one was a peptide), and 3 new nonantibody therapeutic proteins, but no new chimeric antigen receptor (CAR)-T-cell therapeutics. Of the new antibody-based biologics, two were antibody-drug conjugates, one was a T-cell engager-bispecific antibody, two were Fc-modified, half-life extended antibodies, a

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… BK then binds to its GPCR B2, causing vasodilation, increased vascular permeability, and angioedema, hallmark components of HAE. C1-INH inhibits this pathway at multiple points as shown in the figure, as noted with boxed arrows. … Hereditary angioedema FXIIa activates the KKS by transforming plasma prekallikrein (PK), which is noncovalently bound to the nonenzymatic high-molecular-weight kininogen (HMWK), into active kallikrein [ 279 ] ( Fig. 4 ). …

Schnitzler's Syndrome-Diagnostic Experience, Approaches to Therapy, and Patient Management according to a Multicenter Russian Cohort.

The objectives of the study were to present the experience of diagnosis, management, and therapy with IL-1 inhibitors in patients with Schni … pathy was detected in 6 (35.3%) patients; enlarged liver, in 6 (35.3%); pericarditis, in 4 (23.5%); angioedema, in 6 (35.3); redness and dryness in the eyes, in 3 (17.6%); sore throat, in 2 (11.8%); abdominal pain, in 1 (5.9%), distal polyneuropathy, in 2 (11.8%); paraesthesia, in 1 (5.9%); and chondritis of the auricles, in 1 (5.9%). Monoclonal gammopathy w …

Фактор XII-А Новая терапевтическая цель? Систематический обзор.

Фактор XII — это молекула нечеткой физиологической функции, которая привлекает все больший интерес к исследованиям в различных медицинских дисциплинах. В последние годы был накоплен значительный объем данных о вкладе фактора XII в патогенез подстрекательских и протромботических заболеваний. Было продемонстрировано, что FXII играет защитную роль в коагуляции на основе FXII во время хостинговой защиты от инфекций и защищает от многоорганических неудач в моделях сепсиса животных. В случае синдрома острой дыхательной недостаточности (АРДС) деятельность FXII способствует высвобождению провоспалител

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… Timothy J Craig et al., 2023, USA [ 39 ] | 80 patients with type I or type II HAE | The role of garadacimab as prophylaxis for HAE. | Garadacimab administration significantly reduced hereditary angioedema attacks. 3. Results 3.1. … Efficacy and safety of garadacimab, a factor XIIa inhibitor for hereditary angioedema prevention (VANGUARD): A global, multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023;401:1079–1090. doi: 10.1016/S0140-6736(23)00350-1. …

Skin manifestations associated with systemic diseases - Part I.

The skin demonstrates what is happening in the body in many diseases, as it reflects some internal processes on the surface. In this sense, skin as an organ, goes beyond its protective and barrier functions, as it provides clues for the identification of some systemic diseases. The dermatologist then raises diagnostic hypotheses for conditions related to all systems and refers them to the appropriate specialty. With easy access to examination by trained eyes and biopsies, the skin can present specific or non specific alterations on histopathology. In the first case this combination establishes

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… Occasional manifestations: DLE Urticaria/Angioedema 7. Specific lesions in mucous membranes: Lichen planus ACLE Neutrophilic dermatoses CCLE Erythema multiforme Seborrheic dermatitis Vitiligo Table 1 Specific and non specific lesions of lupus erythematosus. … Occasional manifestations: DLE | Urticaria/Angioedema 7. …

Real-World Dermatologic Adverse Events of CAR T-Cell Therapy: A Decade-Wide Disproportionality Analysis of the FDA Adverse Event Reporting System.

Background: Chimeric antigen receptor (CAR) T-cell therapy has transformed haematological-malignancy care, but its dermatologic safety profile remains incompletely characterised across products and indications. Methods: We analysed 8,431,841 deduplicated FDA Adverse Event Reporting System reports from 2016 Q3 to 2026 Q1. Seven approved CAR T-cell products were identified. The primary outcome was any dermatologic adverse event, defined using the MedDRA Skin and subcutaneous tissue disorders system organ class. Secondary outcomes included broad severe cutaneous adverse reactions, narrow Stevens-

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… A 14-category dermatologic adverse-event panel, adapted from Storgard et al. [ 6 ], included bullous dermatosis, eczematous dermatitis, psoriasiform reactions, rash, severe cutaneous eruption, toxic eruption, urticaria/angioedema, hair changes, hyperhidrosis, pruritus, skin lesions, vascular cutaneous … The lowest estimates were observed for chronic or T-cell-mediated dermatoses, including psoriasiform events (ROR 0.014), pruritus (0.058), eczematous dermatitis (0.072), urticaria/angioedema (0.111) and hair changes (0.118). …

Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.

Gene editing is a groundbreaking therapeutic approach that can potentially treat a broad spectrum of genetic and acquired diseases. This review highlights recent clinical trials employing advanced gene editing technologies such as CRISPR-Cas9, zinc-finger nucleases (ZFNs), and base editors across multiple disease areas including metabolic disorders, autoimmune diseases, muscular dystrophies, and inherited eye disorders. Central to the success of these therapies is the development of efficient and safe delivery systems, including lipid nanoparticles (LNPs), viral vectors (adenoviral and lentivi

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… are advancing toward precise, durable therapies, with ex vivo base editing strategies being explored for primary immunodeficiencies like X-linked chronic granulomatous disease (X-CGD), while in vivo CRISPR-based treatments delivered via LNPs are under investigation for conditions such as hereditary angioedema … Table 5 Ongoing clinical trials employing CRISPR-Cas9 and base editing for the treatment of chronic granulomatous disease and hereditary angioedema Trial ID Phase Disease Target Delivery system Intervention Gene editing method Country Reference NCT06325709 1/2 chronic granulomatous disease (CGD) autologous …

Interventions for preventing the progression of autosomal dominant polycystic kidney disease.

BACKGROUND: Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited disorder causing kidney disease. Current clini … and kidney volume (MD -0.08 L, 95% CI -0.75 to 0.59) but in three studies (560 participants) caused angioedema (RR 13.39, 95% CI 2.56 to 70.00), oral ulceration (RR 6.77, 95% CI 4.42 to 10.38), infections (RR 1.14, 95% CI 1.04 to 1.25) and diarrhoea (RR 1.70, 95% CI 1.26 to 2.29).Somatostatin analogues (6 studies, 138 participants) slightly improved SCr (MD …

FDA-Approved Drugs Containing Amide Functionality in the Last Five Years (2021-2025): Pharmaceutical Use, Trends and Synthetic Approaches.

The amide functional group remains a cornerstone of medicinal chemistry, serving as an indispensable scaffold in the design of modern therapeutics. This review presents an analysis of FDA-approved drugs (small molecules and peptides with MW ®), amides form the structural backbone of peptidomimetics, enabling high-affinity binding to a viral protease. In precision oncology, as seen with adagrasib (Krazati®), the amide acts as a critical, metabolically stable linker that positions a covalent warhead for selective inhibition of a mutant kinase. This analysis underscores that amide's unique combin

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… Sebetralstat Approved on 7 July 2025 by the FDA [ 284 ], sebetralstat ( 85 , Ekterly ® , Figure 68 a) is an oral drug developed by KalVista Pharmaceuticals for the on-demand treatment of hereditary angioedema (HAE) attacks [ 284 , 285 ]. … Hereditary angioedema (HAE) attacks are characterized by sudden, painful swelling episodes in various parts of the body. These attacks can affect the face, limbs, abdomen, and, most seriously, the airways, potentially leading to life-threatening situations [ 286 ]. …

Monoclonal Antibodies Approved for Cancer Therapy

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… Hypersensitivity reactions, generally manifesting as bronchospasm or angioedema, are another potentially dangerous adverse effect. … Signs and symptoms include bronchospasm, ARDS, pulmonary infiltrates, cardiac disorders, angioedema, and anaphylaxis. …

Antibodies to watch in 2018.

The pace of antibody therapeutics development accelerated in 2017, and this faster pace is projected to continue through 2018. Notably, the annual number of antibody therapeutics granted a first approval in either the European Union (EU) or United States (US) reached double-digits (total of 10) for the first time in 2017. The 10 antibodies granted approvals are: brodalumab, dupilumab, sarilumab, guselkumab, benralizumab, ocrelizumab, inotuzumab ozogamicin, avelumab, duvalumab, and emicizumab. Brodalumab, however, had already been approved in Japan in 2016. As of December 1, 2017, nine antibody

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… The FDA has granted lanadelumab Breakthrough Therapy and orphan drug designations, and lanadelumab has been granted orphan drug designation in the EU, for treatment of hereditary angioedema attacks. … In May 2017, Shire reported key results from the 26-week Phase 3 HELP study ( NCT02586805 ), which evaluated the efficacy and safety of lanadelumab in preventing acute angioedema attacks in patients with Type I and Type II HAE. 49 Patients were administered 300 mg lanadelumab every 2 weeks or every 4 …

CRISPR-Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics.

CRISPR-Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used across early CRISPR clinical translation. In parallel, early clinical experience has begun to demonstrate the feasibility of in vivo editing, including subretinal delivery for CEP290-associated inherited retinal degeneration (EDIT-101 programme) and hepatocyte-targ

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… NTLA-2002 applies the same platform to KLKB1 in hereditary angioedema (HAE), achieving dose-dependent kallikrein suppression and roughly 90–95% reductions in monthly attack rates after a one-time infusion [ 71 , 107 ]. … Similarly, NTLA-2002 targets KLKB1 in hereditary angioedema, where enrollment is defined clinically and by baseline attack burden, supported by kallikrein–kinin pathway biomarkers [ 71 ]. …

Vaccine Allergy? Skin Testing and Challenge at a Tertiary Pediatric Hospital in Melbourne, Australia.

BACKGROUND: The rate of true vaccine allergy is unknown. Children with potential IgE-mediated adverse events following immunization (AEFI) should undergo allergy investigation that may include skin testing or challenge. Previous protocols tend to be highly conservative and often suggest invasive testing for all, a practice not evidence based, technically difficult, and unpleasant in children. It has more recently been suggested that skin testing may be restricted to those with allergic-like events within the first hour and those with a history of anaphylaxis. OBJECTIVE: We aimed to describe t

Advances in antisense oligonucleotide treatment for cancer.

RNA therapeutics, including antisense oligonucleotides (ASOs), have emerged as a promising class of drugs, with several already approved for clinical use. To date, most approved ASO-based RNA therapies target non-malignant disorders such as neurodegenerative diseases, and only a single therapy in this class has been approved for cancer. Notably, nearly half of existing RNA therapeutics act by modulating RNA splicing. Given the growing evidence implicating aberrant RNA splicing in cancer pathogenesis, the development of ASO-based therapeutics for oncologic indications is expected to accelerate.

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… Donidalorsen, approved in August 2025, is also a GalNAc-conjugated 2′-MOE ASO for the prevention of hereditary angioedema (HAE) attacks [ 75 ]. It binds to prekallikrein (PKK) mRNA and induces RNase H1–mediated degradation, thereby reducing the production of PKK protein [ 76 ]. … In Phase III clinical trials, donidalorsen treatment significantly reduced the rate of hereditary angioedema attacks compared with placebo [ 77 ]. …

Anxiety in the Medically Ill: A Systematic Review of the Literature.

BackgroundAlthough anxiety is highly represented in the medically ill and its occurrence has relevant clinical implications, it often remains undetected and not properly treated. This systematic review aimed to report on anxiety, either symptom or disorder, in patients who suffer from a medical illness.MethodsEnglish-language papers reporting on anxiety in medically ill adults were evaluated. PubMed, PsycINFO, Web of Science, and Cochrane databases were systematically searched from inception to June 2021. Search term was "anxiety" combined using the Boolean "AND" operator with "medically ill/c

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… Among patients with hereditary angioedema, 15–49.9% had anxious symptoms (Fouche et al., 2014 * ; Banerji et al., 2020 * ) which were severe in 7.6–25% of cases (Banerji et al., 2020 * ; Fouche et al., 2014 * ), mild in half of them, and moderate in about 25% (Fouche et al., 2014 * ). … Discussion Moderate or severe anxiety occurs particularly among patients with chronic kidney disease, end-stage renal disease, hip pathology, systemic lupus erythematosus patients, hereditary angioedema and chronic urticarial, metastatic breast cancer, bladder cancer. …

The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data.

ObjectivesTo investigate the drugs and indications that have passed through the UK's Early Access to Medicines Scheme (EAMS) to date, the type of evidence the regulator considers when accepting a drug into the EAMS, and potential risks to patients.DesignAnalysis of publicly available data: MHRA Public Assessment Reports; Electronic Medicines Compendium database; interactive Drug Analysis Profiles database; Eudravigilance database.SettingUnited Kingdom.ParticipantsThe 51 'scientific opinions' available on the MHRA website in June 2024.Main outcome measuresPublic Assessment Reports, pharmacovigi

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… The majority of indications were cancers (60%, 29), followed by severe atopic dermatitis (4), Pompe Disease (2), transthyretin amyloidosis (2) and 11 other diseases (Crohn's, myasthenia gravis, sickle cell, angioedema, spinal muscular atrophy, hyperoxaluria, Sars Cov-2, familial chylomicronemia, haemophilia … Scemblix | Philadelphia chromosome-positive chronic myeloid leukaemia | Novartis 8 | Voxelotor | Oxbryta | Sickle cell disease | Pfizer 9 | Abrocitinib | Cibinqo | Severe atopic dermatitis | Pfizer 10 | Avalglucosidase alfa | Nexviazyme | Pompe disease | Aventis 11 | Berotralstat | Orladeyo | Hereditary angioedema …

The patient journey of patients with Fabry disease, Gaucher disease and Mucopolysaccharidosis type II: A German-wide telephone survey.

BACKGROUND: Lysosomal Storage Diseases (LSD) are rare and multisytemic diseases which are caused by lysosomal enzyme deficiencies leading into accumulation of waste products due to an interruption in the decomposition process. Due to the low prevalence and therefore limited disease awareness as well as the fact that LSD patients present with unspecific symptoms the final diagnosis is often made after a long delay. The aim of this German-wide survey was to characterize the period between onset of symptoms and final diagnosis regarding e.g. self-perceived health, symptom burden and false diagnos

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… Compared to previous decades, the diagnosis of rare diseases is now made much earlier due to increased awareness, which has been exemplarily shown for hereditary angioedema [ 19 ]. … Improvement in diagnostic delays over time in patients with hereditary angioedema: findings from the Icatibant Outcome Survey. Clin Transl Allergy. 2018;8:42 10.1186/s13601-018-0229-4 Associated Data Supplementary Materials Survey questionnaire in German. …

Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia.

Gene therapy has transitioned from a long-awaited promise to a clinical reality, offering transformative treatments for rare congenital diseases and certain cancers, which have a significant impact on patients' lives. Current approaches focus on gene replacement therapy, either in vivo or ex vivo, mostly utilizing viral vectors to deliver therapeutic genes into target cells. However, refining these techniques is essential to overcome challenges and complications associated with gene therapy to ensure long-term safety and efficacy. Slovenia has witnessed significant advancements in this field s

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… A notable example is the successful in vivo CRISPR-Cas9–mediated disruption of the KLKB1 gene using lipid nanoparticle (LNP) delivery in patients with hereditary angioedema. … CRISPR-Cas9 in vivo gene editing of KLKB1 for hereditary angioedema. N Engl J Med. 2024;390(5):432–41. 38294975 10.1056/NEJMoa2309149 45. …

Hair Dye Ingredients and Potential Health Risks from Exposure to Hair Dyeing.

Given the worldwide popularity of hair dyeing, there is an urgent need to understand the toxicities and risks associated with exposure to chemicals found in hair dye formulations. Hair dyes are categorized as oxidative and nonoxidative in terms of their chemical composition and ingredients. For several decades, the expert panel's Cosmetic Ingredient Review (CIR) has assessed the safety of many of the chemicals used in hair dyes; however, a comprehensive review of hair dye ingredients and the risk of exposure to hair dyeing has not been documented. Herein, we review the safety of the various ch

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… ( Table 3 ). 61 − 63 Angioedema is a type I hypersensitivity reaction characterized by edema of the skin and subcutaneous tissues that damages the airways and gastrointestinal tract and may even lead to life-threatening laryngeal swelling. … Angioedema, an unusual reaction to hair dye . pan african medical journal 2018 , 30 , 103 10.11604/pamj.2018.30.103.12061 . 30364413 PMC6195235 Isik S. M. D. ; Caglayan-Sozmen S. M. D. ; Anal O. M. D. ; Karaman O. M. D. ; Uzuner N. M. D. …

Systemic mastocytosis - a diagnostic challenge.

Mastocytosis refers to a group of disorders characterized by the infiltration of clonally derived mast cells to the skin or extracutaneous tissues resulting in a heterogeneous clinical picture. It is a rare hematologic disorder in all its forms. The exact incidence is unknown; it affects patients of any age and males and females equally. Its molecular pathogenesis is incompletely understood. The clinical features of mastocytosis result from both chronic and episodic mast cell mediator release, signs and symptoms arising from diffuse or focal tissue infiltration, and, occasionally, the presence

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… Our patient found herself inserted in a category of aggressive SM presenting three C findings (hematopoietic dysfunction with bicytopenia, diffuse bone lesions and weight loss due to malabsorption). 7 , 8 , 9 The differential diagnosis is important to exclude other pathologies such as angioedema, carcinoid …