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Accelerating Amyloidosis Drug Development

Amyloidosis presents a significant therapeutic challenge due to its complex pathogenesis and limited treatment options. Protheragen is a specialized partner in the development of innovative therapeutics for Amyloidosis, offering a full spectrum of preclinical drug development services. From target validation and lead optimization to comprehensive IND-enabling studies, Protheragen integrates scientific rigor with advanced technology platforms to support the translation of novel concepts into clinical candidates. Our team combines deep expertise in protein misfolding disorders with robust capabilities in in vitro and in vivo modeling, pharmacokinetics, toxicology, and biomarker development. Protheragen’s operations adhere to stringent regulatory standards, ensuring data integrity and facilitating seamless advancement toward clinical trials. By leveraging state-of-the-art methodologies and a proven track record in complex disease biology, Protheragen delivers tailored solutions that address the unique challenges of Amyloidosis drug discovery. Driven by a commitment to scientific excellence and innovation, Protheragen partners with pharmaceutical and biotech organizations to accelerate the development of effective therapies, ultimately aiming to improve outcomes for patients affected by Amyloidosis.

What is AmyloidosisTargets for AmyloidosisDrug Discovery and Development ServicesWhy Choose Us

What is Amyloidosis

Amyloidosis is a group of disorders characterized by the abnormal extracellular deposition of misfolded protein fibrils, known as amyloid, in various tissues and organs. These deposits result from the aggregation of normally soluble proteins—such as immunoglobulin light chains, serum amyloid A, transthyretin, or beta-2 microglobulin—into insoluble, beta-pleated sheet structures. The underlying causes vary by type and include plasma cell dyscrasias, chronic inflammatory diseases, genetic mutations, and long-term dialysis. The accumulation of amyloid disrupts normal tissue architecture and function, leading to progressive organ dysfunction. Clinically, amyloidosis presents with a wide range of symptoms depending on the organs involved, most commonly affecting the heart, kidneys, liver, nervous system, and joints. Diagnosis relies on tissue biopsy with Congo red staining, followed by identification of the amyloid type using specialized techniques. Additional laboratory and imaging studies assess organ involvement. Treatment is tailored to the amyloidosis subtype and may include therapies that reduce precursor protein production (such as antisense oligonucleotides or small interfering RNA for transthyretin amyloidosis), stabilize native proteins (transthyretin stabilizers), or target underlying plasma cell disorders (monoclonal antibodies for AL amyloidosis). Early recognition and intervention are essential to improve outcomes and limit irreversible organ damage.

Launched Drugs

Structure Generic Name CAS Registry Number Molecular Formula Molecular Weight
eplontersen (Rec INN; USAN) 1637600-16-8
img-1446711-81-4-acoramidis-rec-inn-usan acoramidis (Rec INN; USAN) 1446711-81-4 C15 H17 F N2 O3 292.305
vutrisiran (Rec INN; USAN) 1867157-35-4
daratumumab SC; daratumumab/hyaluronidase-fihj; daratumumab/vorhyaluronidase alfa
inotersen (Rec INN; USAN) 1492984-65-2
patisiran (Rec INN; USAN) 1420706-45-1
img-594839-88-0-free-acid951395-08-7-tafamidistafamidis-meglumine-prop-innm-usan tafamidis; tafamidis meglumine (Prop INNM; USAN) 594839-88-0 (free acid); 951395-08-7 C14 H7 Cl2 N O3 . C7 H17 N O5 503.33

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Targets for Amyloidosis

Targets in Clinical or Later Phases of Development

Target Name Gene Symbol
transthyretin TTR
Amyloid beta A4 precursor protein-binding family (APP-BP) (nonspecified subtype)
ATP binding cassette subfamily B member 1 ABCB1
amyloid beta precursor protein APP
cereblon CRBN
DCX (DDB1-CUL4-X-box) E3 protein ligase complex
Matrix Metalloproteinase (MMP) (nonspecified subtype)
caspase 3 CASP3
Nuclear factor kappa-light-chain-enhancer of activated B cells
nuclear receptor subfamily 3 group C member 1 NR3C1

Amyloidosis is driven by the misfolding and aggregation of specific precursor proteins, which accumulate as amyloid fibrils in tissues and organs. The principal amyloidogenic targets include amyloid beta precursor protein (APP), transthyretin (TTR), beta-2-microglobulin (B2M), and leukocyte cell derived chemotaxin 2 (LECT2). Each plays a distinct role in disease subtypes: APP is central to Alzheimer’s disease via abnormal proteolytic processing that generates neurotoxic amyloid-beta peptides; TTR destabilization leads to familial and wild-type transthyretin amyloidosis through tetramer dissociation and fibril formation; B2M accumulates in patients with renal failure, causing dialysis-related amyloidosis; and LECT2 aggregation is implicated in systemic amyloidosis, particularly affecting kidneys and liver. These proteins’ structural features, post-translational modifications, and interactions with cellular pathways are fundamental to amyloidogenesis and tissue damage. Targeting these proteins offers significant therapeutic promise. For TTR amyloidosis, small molecule stabilizers (e.g., tafamidis), gene silencing agents (patisiran, inotersen), and fibril disruptors are in clinical use or advanced development. In Alzheimer’s disease, therapies focus on secretase inhibitors and anti-amyloid-beta antibodies, with several agents approved or in late-stage trials. Strategies to reduce B2M levels or prevent its aggregation are employed for dialysis-related amyloidosis, while LECT2 lacks specific therapies, highlighting an unmet need. Quantifying these proteins and their misfolded forms also provides valuable biomarkers for diagnosis, prognosis, and treatment monitoring, establishing these targets as central to both therapeutic and diagnostic advances in amyloidosis.

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Drug Discovery and Development Services

In Vitro Efficacy Testing ServicesIn Vivo Model DevelopmentPK/PD Study ServicesIn Vivo Toxicity Assessment ServicesBiomarker Analysis Services
In Vitro Efficacy Testing Services

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Our In Vitro Efficacy Testing Service accelerates amyloidosis drug discovery by providing robust, sensitive platforms for screening and characterizing therapeutics. Leveraging advanced biochemical and biophysical assays—including biolayer interferometry, FRET, HTRF, chemiluminescence, ELISA, and surface plasmon resonance—we quantify key pharmacological parameters such as IC-50, EC-50, Kd, Ki, and MEC. These methods enable precise assessment of compound potency, binding affinity, aggregation inhibition, and functional cellular outcomes. Our tailored approach supports comprehensive profiling of candidate drugs targeting amyloidogenic proteins, ensuring efficient lead optimization and translational research to advance effective amyloidosis therapies.

Amyloid Beta Precursor Protein Caspase 3
Cereblon Nuclear Receptor Subfamily 3 Group C Member 1
Transthyretin

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Why Choose Us

Choosing Protheragen means partnering with a team that brings specialized expertise and unwavering dedication to the advancement of Amyloidosis therapeutics. At Protheragen, our professionals possess deep experience in Amyloidosis research and drug development, supported by advanced technology platforms that enable innovative and efficient solutions. Our proven track record in preclinical drug development services is a testament to our reliability and commitment to delivering results that meet the highest industry standards. Protheragen adheres strictly to rigorous quality standards and regulatory compliance, ensuring that every project is managed with the utmost integrity and attention to detail. We understand the unique challenges of Amyloidosis drug development and are committed to driving progress in this critical field. By choosing Protheragen, you gain a trusted partner dedicated to advancing new therapies for Amyloidosis, backed by a professional team and cutting-edge capabilities designed to bring promising treatments closer to patients in need.

FAQs for Our Services

Q: What are the main preclinical research challenges specific to developing new drugs for Amyloidosis?

A: Amyloidosis is characterized by the misfolding and deposition of various amyloid proteins, making it a heterogeneous and complex disease. Key preclinical challenges include developing relevant in vitro and in vivo models that accurately recapitulate human amyloid pathology, optimizing compound screening for efficacy against different amyloid types, and overcoming the lack of robust biomarkers for early disease detection and drug response. Our company leverages advanced transgenic animal models, high-content imaging, and state-of-the-art biochemical assays to address these challenges and generate translationally relevant data.

Q: What regulatory considerations are important in the preclinical development of Amyloidosis drugs?

A: Given Amyloidosis is a rare and often life-threatening disease, regulatory agencies such as the FDA and EMA may offer expedited pathways, including orphan drug designation and fast-track status. However, rigorous preclinical safety and efficacy data are still required. It is critical to demonstrate target engagement, mechanism of action, and safety in relevant models. Our team has extensive experience in designing preclinical programs that meet regulatory expectations, ensuring studies are GLP-compliant and that data packages are suitable for IND/CTA submissions.

Q: What are the key technical aspects to consider when conducting preclinical research for Amyloidosis?

A: Technical considerations include selecting appropriate amyloid protein targets (e.g., AL, ATTR, AA), establishing sensitive assays for detecting amyloid burden, and utilizing imaging modalities such as PET or MRI for in vivo tracking. Additionally, pharmacokinetic and pharmacodynamic profiling is essential to understand drug distribution and target engagement. Our services encompass assay development, advanced imaging, and bioanalytical platforms tailored for amyloid-specific research, ensuring comprehensive characterization of candidate therapeutics.

Q: What are the typical timeline and cost considerations for preclinical Amyloidosis drug development?

A: Preclinical development timelines for Amyloidosis therapeutics generally span 12 to 24 months, depending on the complexity of the compound and study requirements. Costs can vary significantly, typically ranging from $2M to $5M for a full IND-enabling package. Factors influencing cost and timeline include the need for specialized animal models, custom assay development, and regulatory documentation. Our company offers modular service packages, transparent budgeting, and project management expertise to optimize timelines and control costs.

Q: What are the critical success factors in preclinical drug development for Amyloidosis?

A: Success in preclinical Amyloidosis drug development hinges on selecting the right disease models, demonstrating robust and reproducible efficacy, ensuring safety and tolerability, and generating high-quality data that supports regulatory filings. Early engagement with regulatory authorities and the incorporation of translational biomarkers are also vital. Our integrated approach combines scientific rigor, regulatory insight, and cutting-edge technology to maximize the likelihood of advancing promising candidates to clinical development.

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