An Opportunity
For Patient Impact

icon of a pill bottleThe Need for New Treatment Options


Diverse disease types are not all addressed by current treatments.


No existing therapeutics remove amyloid and reverse disease.

Current Challenges

In some types of amyloidosis, approved therapies reduce amyloid formation and may slow progression.

Lack of removal of toxic amyloid accumulated in organs leads to progress organ failure and increased mortality.

Lack of therapies that address all stages of disease.

icon of outline of a man with heartThe Need for Better Diagnosis


Vast majority of patients are undiagnosed.

icon of a hour glass

Short survival window drives need for earlier diagnosis.

Current Challenges

There are no amyloid specific diagnosis agents that work across all amyloid types and provide a complete picture of the disease.

Disease progresses unchecked and toxic amyloid accumulates.

It can take years and a multiple of diagnostic tests for patients to get diagnosed.


Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.

Proven Treatments in Systemic Amyloidosis

Modulating amyloid proteins is a validated target in systemic amyloidosis. Therapies recently approved in ATTR amyloidosis have proven that reducing or stabilizing the pre-cursor protein leads to slowing disease progression. In AL amyloidosis, off-label plasma cell targeting agents including chemotherapy and Autologous Stem Cell Transplant, which both reduce the precursor protein, have also been shown to improve clinical outcomes. Reducing or stabilizing the pre-cursor protein in AL and ATTR amyloidosis results in the reduction of formation of new amyloid, but does not directly address already deposited toxic amyloid.


There is a substantial unmet need to improve the diagnosis of amyloidosis in patients. The majority of systemic amyloidosis patients see more than four specialists and take more than 18 months to get an accurate diagnosis, with 25% of patients taking more than five years to get a correct diagnosis. Unfortunately, due to this prolonged process, a significant number of patients die without a diagnosis. Of the patients that do receive a diagnosis, there are a number of diagnostic procedures required with clear challenges in getting an early and accurate diagnosis. A misdiagnosis can be deadly and delayed diagnosis leads to poorer patient outcomes.

30 Subtypes


4 most Common

Types of Systemic Amyloidosis


Wild-type Transthyretin Amyloidosis

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>400K patients in US, EU, & Japan

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13% of HFpEF patients may have wtATTR

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Mainly manifests in heart and ligaments & tendons

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Median Survival: 3-5 years


Hereditary Transthyretin Amyloidosis

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>50K patients in US, EU, & Japan

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3-4% of African Americans carry the V122I (V142I) mutation

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Manifests in heart, nerves, GI, and kidneys

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Median Survival: 3-15 years; 3-5 years with cardiac involvement


Light Chain Amyloidosis

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>50K patients in US, EU & Japan

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12-30% of MM patients have co-existing AL​

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Manifests in almost every organ: >70% have cardiac and/or kidney

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Median Survival: 5 years; 1-2 years in cardiac patients


Leukocyte Chemotactic Factor 2 Amyloidosis

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Unknown prevalence

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ALECT2 deposits found in~3% of Hispanic descendants

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Manifests mainly in kidney and liver

Other Types of Systemic Amyloidosis

icon meaning prevalence
Rare: <50K in aggregate in US, EU & Japan

icon meaning manifest

Manifests in various organs, with about half affecting kidneys

AA (Apo) Serum amyloid A All organs except CNS
AH Immunoglobulin heavy chain All organs except CNS
Ab2M b2-Microglobulin, wild type b2-Microglobulin Musculoskeletal system ANS
AApoAI variant, Apolipoprotien A I, variants Heart, liver, kidney, PNS, testis

larynx (C-terminal variants), skin (C-terminal variants)

AApoAII Apolipoprotien A II, variants Kidney
AApoAIV Apolipoprotien A IV, wild type Kidney medulla and systemic
AApoCII Apolipoprotien C II, variants Kidney
AApoCII Apolipoprotien C II, variants Kidney
AGel Gelsolin, variants PNC, cornea
ALys Lysozyme, variants Kidney
AFib Fibinogen a, variants Kidney, primarily
ACys Cystatin C, variants PNS, skin
ACal (Por)calcitonin C-cell thyroid tumors
AIAPP Islet amyloid polypeptide** Islets of Langerhans, insulinomas
AANF Atrial natriuretic factor Cardiac atria
APro Prolactin Pituitary prolactinomas, aging pituitary
Alns Insulin Iatrogenic, local injection
ASPC*** Lung surfactant protein Lung
AGal7 Galectin 7 Skin
ACor Corneodesmosin Cornified epithelia, hair follicles
AMed Lactadherin Senile aortic, media
AKer Kerato-epithelin Cornea, hereditary
ALac Lactoferrin Cornea
AOAAP Odontogenic ameloblast-associated protein Odontogenic tumors
ASem1 Semenogelin 1 Vesicula seminalis
AEnf Enfurvitide latrogenic