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RNA interference

RNA interference

RNA interference (RNAi) is a mechanism of controlling gene activity used in most eukaryotic cells. RNAi works through small interfering RNAs (siRNA) or micro RNA (miRNA) which target mRNA transcripts. RNAi drugs use siRNA or miRNA.

RNA interference (RNAi) is a mechanism of controlling gene activity used in most eukaryotic cells. Small interfering RNAs (siRNA) are double stranded RNA (dsRNA) molecules that trigger direct homology-dependent control of gene activity. The small dsRNA molecules are recognized by RNAi enzymatic machinery which leads to degradation of target mRNA. In mammalian cells siRNA are produced from cleavage of longer dsRNA precursors by the RNase II endonuclease Dicer. siRNA molecules can also be synthesized by chemical or biological methods.

Micro RNA (miRNA) are another arm of RNAi. miRNA are single stranded molecules that regulate expression of multiple mRNAs while siRNA molecules inhibit one specific target mRNA. The single-stranded miRNA guide the miRISC complex to target mRNAs and result in gene silencing by translational repression, degradation or cleavage. Whereas siRNA must be completely complementary to target mRNA miRNA can be partially complementary to its target RNA.

Approved RNAi drugs

  • Givosiran (Givlaari) from Alynlam Pharmaceuticals is a treatment for acute hepatic porphyria, a rare genetic condition that leads to the buildup of toxic polypyrin molecules (FDA approved)
  • Leqvio (inclisiran) from Novartis , an siRNA drug for treatment of hypercholesterolemia or mixed dyslipidemia (European Commission approval)
  • Lumasiran (Oxlumo) from Alnylam Pharmaceuticals is a treatment for the rare genetic disorder known as primary hyperoxaluria type 1 (FDA approved, EMA approved)
  • Patisiran (Onpattro) from Alnylam is an RNAi treatment for a rare genetic disorder known as hereditary transthyretin amyloidosis that causes buildup of the protein transthyretin in the nervous system (FDA approved)

Timeline

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May 3, 2021
BioSpace
Alnylam Announces Positive Early Results on Clinical Outcome Measures from ILLUMINATE-A Phase 3 Study of OXLUMO® (lumasiran) - read this article along with other careers information, tips and advice on BioSpace
Benitec Biopharma Inc.
April 27, 2021
www.prnewswire.com:443
/PRNewswire/ -- Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "the Company"), a development-stage, gene therapy-focused, biotechnology company developing...
BioSpace
April 11, 2021
BioSpace
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced positive interim results from the ongoing Phase 1 study of ALN-AGT, a subcutaneous investigational RNAi therapeutic targeting liver-expressed angiotensinogen for the treatment of hypertension.
Angus Liu
March 10, 2021
FierceBiotech
Scientists at Northwestern University have developed a novel RNA interference drug that targets the cancer-promoting Bcl2L12 gene in glioblastoma tumors. A new study in a small number of patients showed that it can penetrate the blood-brain barrier.

References

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