Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria

Gema Ariceta; Kelly Barrios; Bob D. Brown; Bernd Höppe; R. Roßkamp; Craig B. Langman

doi:10.1016/j.ekir.2021.01.029

February 6, 2021SHILAP Revista de lepidopterologíaOpen Access

Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria

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Key Points

To evaluate the safety and therapeutic potential of hepatic lactate dehydrogenase A (LDHA) inhibition using RNA interference across all known types of primary hyperoxaluria.
Evaluated clinical safety and tolerability outcomes from a Phase I trial assessing the RNA interference agent nedosiran.
Integrated and reviewed supporting published nonclinical data targeting hepatic LDHA.
Phase I clinical data together with nonclinical findings demonstrate that hepatic LDHA inhibition is a safe therapeutic approach.
Evidence supports LDHA targeting as a viable intervention applicable across all known genetic subtypes of primary hyperoxaluria.

Implication

Phase 1 trial data demonstrates safety of hepatic LDHA inhibition across all primary hyperoxaluria types, indicating a universal therapeutic target.

Abstract

Phase I clinical data on nedosiran and published nonclinical data together provide substantial evidence that LDHA inhibition is a safe therapeutic mechanism for the treatment of all known types of PH.

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Cite This Study

Ariceta et al. (2021) studied this question.

synapsesocial.com/papers/69d8288ef4e559c61eae2ac4 https://doi.org/10.1016/j.ekir.2021.01.029

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Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria

Key Points

Implication

Abstract

Discussion

Cite This Study

Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria

Key Points

Implication

Abstract

Discussion

Cite This Study

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