TTR x RBP4

Last update 08 May 2025

Basic Info

Related Targets

TTRRBP4

Drugs associated with TTR x RBP4

ERM-001

Target

RBP4 x TTR

Mechanism

RBP4 inhibitors [+1]

Active Org.

Ermaris Bio, Inc.

Originator Org.

Ermaris Bio, Inc.

Active Indication

Geographic Atrophy

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

100 Clinical Results associated with TTR x RBP4

100 Translational Medicine associated with TTR x RBP4

0 Patents (Medical) associated with TTR x RBP4

146

Literatures (Medical) associated with TTR x RBP4

01 Dec 2025·Current Heart Failure Reports

Biomarkers in Subclinical Transthyretin Cardiac Amyloidosis

Review

Author: Roth, Lori R ; Hendren, Nicholas ; Pedretti, Rose ; Saelices, Lorena ; Grodin, Justin L ; Kozlitina, Julia ; Gulati, Jaskeerat S

PURPOSE OF REVIEWThe most common type of cardiac amyloidosis is transthyretin amyloidosis (ATTR-CM). Early forms of the disease can often go undetected. Effective pharmacological treatments are available for ATTR-CM. However, current treatment options may be more effective when used earlier in the disease, making early detection paramount. Below, we discuss updates with regards to the role that blood-based biomarkers play in detecting subclinical cardiac amyloidosis.RECENT FINDINGSCarriers of amyloidogenic mutations in the TTR gene are at a heightened risk of developing heart failure and have higher mortality rates compared with noncarrier counterparts. Conventional biomarkers, such as the cardiac troponins and natriuretic peptides, may be useful to monitor subclinical cardiac amyloidosis. In addition, recent studies have demonstrated links between amyloidogenic TTR carrier status and low levels of circulating transthyretin (TTR) and retinol-binding protein 4 (RBP4). Laboratory advances have also allowed for the development of peptide-based detection methods. Probes targeting transthyretin aggregates and nonnative TTR peptides have shown promise in differentiating ATTR from non-ATTR amyloidosis populations. Finally, recent studies have identified neurofilament light chains as potential biomarkers for detecting polyneuropathy-predominant amyloidosis. Conventional biomarkers, such as cardiac troponin and natriuretic peptides may indicate evolving amyloid deposition in early ATTR-CM. However, they are non-specific and emerging biomarkers such as serum transthyretin levels, retinol-binding protein 4, transthyretin aggregates, nonnative TTR, and neurofilament light chains may hold promise in characterizing subclinical ATTR.

04 Feb 2025·Investigative Ophthalmology & Visual Science

Hepatic Satellite Cell Activation and Alteration of Vitamin A Status Are Relevant to the Aggravation of Retinopathy by T2DM

Article

Author: Chang, Han-Hsin ; Lin, David Pei-Cheng ; Chang, Min-Chun

PurposeType 2 diabetes mellitus (T2DM) leads to diabetic retinopathy (DR) and hepatic impairments. The potential mutual interaction and the intermediator between these two injuries are not well elucidated. Both the retina and liver are involved in vitamin A metabolism, suggesting a potential involvement of vitamin A and its metabolites in this mutual interaction. This study aimed to elucidate the impact of either DR or hepatic impairment on the pathogenesis and vitamin A status of each during injury progression.MethodsA streptozotocin (STZ)-high-fat diet (HFD)-induced T2DM rodent model was applied to examine via electroretinography (ERG) retinal and hepatic histopathology at 0, 12, 16, 20, 24, 28, and 30 weeks after T2DM induction. The levels of retinol in the retina, liver, serum, all-trans-retinal in the retina, and retinyl palmitate in the liver were measured at various time points after T2DM induction.ResultsRetinal dysfunction, evidenced by reduced ERG responses, appeared at week 12, followed by photoreceptor and ganglion cell damage after the 16th week. Hepatic impairments began with hepatic stellate cell activation and decreased retinyl palmitate storage, concurrent with reduced retinal retinol and increased all-trans-retinal. Serum retinol levels remained stable, but reductions in transthyretin (TTR) and retinol-binding protein 4 (RBP4) were found, likely disrupting vitamin A transport in the serum.ConclusionsThese results provide novel insights into hepatic injury and vitamin A status, implicating both in the aggravation of retinopathy under the influence of T2DM. The current results may raise clinical awareness on hepatic issues and vitamin A involvement during DR progression.

02 Apr 2024·Amyloid

Circulating transthyretin and retinol binding protein 4 levels among middle-age V122I TTR carriers in the general population

Article

Author: Shao, Zhili ; Liu, Chia-Feng ; Saelices, Lorena ; Grodin, Justin L. ; Farr, Maryjane A. ; Hendren, Nicholas S. ; Garg, Sonia ; Kozlitina, Julia ; Ji, Alan X. ; Drazner, Mark H. ; Tang, W. H. Wilson ; Berry, Jarett D. ; De Lemos, James A.

BACKGROUNDHereditary transthyretin cardiac amyloidosis (ATTRv-CA) has a long latency phase before clinical onset, creating a need to identify subclinical disease. We hypothesized circulating transthyretin (TTR) and retinol binding protein 4 (RBP4) levels would be associated with TTR carrier status and correlated with possible evidence of subclinical ATTRv-CA.METHODSTTR and RBP4 were measured in blood samples from V122I TTR carriers and age-, sex- and race-matched non-carrier controls (1:2 matching) among Dallas Heart Study participants (phases 1 (DHS-1) and 2 (DHS-2)). Multivariable linear regression models determined factors associated with TTR and RBP4.RESULTSThere were 40 V122I TTR carriers in DHS-1 and 54 V122I TTR carriers in DHS-2. In DHS-1 and DHS-2, TTR was lower in V122I TTR carriers (p < .001 for both), and RBP4 in DHS-2 was lower in V122I TTR carriers than non-carriers (p = .002). Among V122I TTR carriers, TTR was negatively correlated with markers of kidney function, and limb lead voltage (p < .05 for both) and TTR and RBP4 were correlated with atrial volume in DHS-2 (p < .05).CONCLUSIONSV122I TTR carrier status is independently associated with lower TTR and RBP4 in comparison with non-carriers. These findings support the hypothesis that TTR and RBP4 may correlate with evidence of subclinical ATTRv-CA.

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