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Supporting the submission of new drug applications this year, the key clinical trial results of innovative RNAi therapy are positive

 Last Update: 2021-11-13
 Source: Internet
 Author: User

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On November 5, Dicerna Pharmaceuticals announced that a pivotal clinical trial of the RNAi drug candidate nedosiran has achieved positive results

Primary hyperoxaluria (PH) is an ultra-rare and life-threatening genetic disease.

Nedosiran binds to mRNA to inhibit the expression of liver lactate dehydrogenase (LDH), which is a key metabolic enzyme that leads to the overproduction of oxalate

▲In patients with PH1 subtypes, nedosiran significantly reduced oxalate excretion in the urine of patients (picture source: Dicerna's official website)

This phase 2 clinical trial reached the primary endpoint.

In the trial, nedosiran was generally well tolerated, and the overall adverse event (AE) characteristics were consistent with previous trial data

Dr.

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