Nitto Denko Brings RNAi Therapeutics to Clinic as New Star Rises on RNA(i) Therapeutics Firmament

There is more evidence from the San Diego biotech scene that investment is flocking back to RNAi Therapeutics, from all sorts of sources.  Following successful public offerings by the likes of Arrowhead Research and Alnylam and multi-million dollar VC rounds in two smaller RNAi Therapeutics companies earlier this year, the investment this time is coming from a large diversified company and private investors.

Nitto Denko Tackles Fibrosis

With the initiation of clinical development for an RNAi Therapeutics in fibrosis, not Merck, not Novartis or Takeda, but Japanese materials manufacturer Nitto Denko has become the largest company yet to enter an RNAi Therapeutics into the clinic which originated from internal R&D.  The newest addition to the RNAi Therapeutics pipeline is based on research from Prof. Niitsu’s laboratory at Sapporo Medical University and involves a vitamin A-targeted liposome for gene knockdown of a collagen-specific chaperone (Hsp47) in fibrogenic cells.  Subsequent to the seminal Nature Biotech publication on gene knockdown in hepatic stellate cells for not just the stabilization, but even the reversal of liver fibrosis (Sato et al. 2008), a more recent publication applying the same formulation in pancreatic fibrosis indicates that Nitto Denko is considering ND-L02-s0201 for fibroses beyond the liver.  

Of note, the RNAi trigger design came from Quark Pharmaceuticals (Tuschl workarounds with lots of non-canonical modifications) and Nitto Denko itself has become the world’s largest clinical supplier of oligonucleotides following its 2011 acquisition of Avecia.

Fibrosis is becoming a hot area in Oligonucleotide Therapeutics with a number of companies (RXi Pharmaceuticals, Excaliard/Pfizer, Sirnaomics, BMT etc) chasing fibroses in the skin, eye, liver, and beyond.  As fibrosis is a complication, often the most debilitating consequence of  many diseases involving inflammation (think fatal diseases such as idiopathic pulmonary fibrosis), and little progress has been made in reversing it, the expanded target space available to oligonucleotide therapeutics could bring unique opportunities at changing the way we think about the disease.

RNAi Start-up Arcturus Therapeutics Raises $1.3M in Private Investor Round

Coincidental with the Nitto Denko trial initiation, San Diego-based Arcturus Therapeutics announced that it has raised $1.3M in a private investor round.  Arcturus Therapeutics, curiously founded by former Nitto Denko employees, is said to be developing a liver-targeted RNAi Therapeutics for an unnamed rare/orphan disease indication.  It appears that the value of Arcturus is based on a nanoparticle delivery technology.  Although the nature of the formulation is yet to be disclosed, my impression is that it is a liposomal one given the background of the company founders and the liver indication.

It is very good seeing examples like that where the RNAi Therapeutics appetite is reaching down to the grass-roots levels, unfathomable even just 1 ½ years ago.  While I once worried that the RNAi Therapeutics dry spell (2008-2011) could prove disastrous to innovation in the industry, some seeds were able to survive.  I look forward to following the progress of these technologies and companies as they grow.