Avidity Biosciences (RNA) Preclinical Data Demonstrates DM1 Activity in Vitro and Durable Reduction in DMK Expression in NHPs in Vivo.

Wells Fargo analyst Yanan Zhu reiterated an Overweight rating on Avidity Biosciences (NASDAQ: RNA) after the company presented preclinical data for its lead antibody oligonucleotideconjugate (AOC) product candidate AOC 1001 at the American Academy of Neurology(AAN) meeting over the weekend. The lead siRNA demonstrated activity in DM1 patient-derived cells in vitro while AOC 1001 demonstrated robust and durable reduction in DMPK expression in NHPsin vivo.

The analyst stated "In DM1patient-derived muscle cells, lead siRNA siDMPK.19 reduced DMPK mRNA levels inboth the cytoplasm and the nucleus, with cell fractionation assay demonstrating 80%reduction in the cytoplasm and 60% reduction in the nucleus. In myotubes cultured fromDM1 patients, siDMPK.19 reduced nuclear foci by 50% (nuclear foci are aggregates ofCUG-expanded DMPK mRNA in the nucleus). RNA splicing analysis demonstrated thatsiDMPK.19 partially corrected aberrant RNA splicing in DM1 myotubes, with 56.5%correction toward normal on the RNA Splicing Score. Based on these results, siDMPK.19was conjugated to transferrin receptor (TFRC) mAb to generate AOC 1001." "A single IV infusion of AOC1001 produced dose-dependent reduction of DMPKexpression in skeletal muscle at 4 weeks (roughly 75% reduction with 2 mg siRNA/kg),with the level of reduction sustained at least until 12 weeks. Expression proling in varioustissues at 4 weeks following a single dose of 6 mg siRNA/kg siDMPK demonstrated 75%to 90%+ reduction of DMPK expression in skeletal muscles, ~75% reduction in cardiacmuscle, modest reductions of ~25% in the spleen and liver, and no reductions in the brain."

For an analyst ratings summary and ratings history on Avidity Biosciences click here. For more ratings news on Avidity Biosciences click here.

Shares of Avidity Biosciences closed at $20.75 yesterday.

You May Also Be Interested In

• Goldman Sachs Upgrades Plains GP Holdings, L.P (PAGP) to Neutral
• Freedom Broker Starts Carnival Corporation (CCL) at Buy
• MaxLinear (MXL) PT Raised to $105 at Stifel