Cover Story: It is an RNA world

Table 4. Delivery platforms. Platforms in clinical development to deliver RNA therapeutics in vivo.

Platform

Company

Program

Description

Stage

Encapsulation platforms

Lipid nanoparticle

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY); Genzyme Corp. unit of Sanofi (Euronext:San; NYSE:SNY)

Patisiran

Stable nucleic acid lipid particles: cationic lipids carrying RNAi therapeutic; the delivery technology is licensed from Tekmira Pharmaceuticals Corp. (TSX:TKM; NASDAQ:TKMR)

Phase III

Silence Therapeutics plc (LSE:SLN)

Atu027

siRNA-lipoplex: with cationic lipids, co-lipids and pegylated lipids

Phase II

Nanoparticle

ProNAi Therapeutics Inc.; Marina Biotech Inc. (Pink:MRNA)

 

PNT2258

SMARTICLES: anionic, pH-tunable liposomes containing DNA-based oligonucleotides that disrupt transcription

Senesco Technologies Inc.

(OTCQB:SNTI)

SNS01-T

Polyethylenimine (PEI)-based nanoparticles carrying gene-encoding DNA plasmid and an siRNA

Phase I/II

Arrowhead Research Corp. (NASDAQ:ARWR)

CALAA-001

RONDEL: a cyclodextrin-coated, polymer-based nanoparticle carrying siRNA

Phase I

ARC-520

Dynamic polyconjugate (DPC): amphipathic nonlipid polymer-based nanoparticles with siRNA-cholesterol conjugate and N-acetylgalactosamine (GalNAc), hepatocyte-targeting ligand

Bio-Path Holdings Inc.

(NASDAQ:BPTH)

Liposomal Grb-2

Neutral lipid liposomal formulation for antisense oligonucleotide delivery

MiNA Therapeutics Ltd.

MTL-501

Triethanolamine-core poly(amidoamine) dendrimer-conjugated small activating RNA

Preclinical

Rexahn Pharmaceuticals Inc. (NYSE-M:RNN)

RX-0201-nano

Nanoliposomal formulation for antisense oligonucleotide delivery

Marina

Multiple

DiLA2: lipid formulations from dialkylated amino acids for

delivery of siRNA

Lipid nanoparticle

Dicerna Pharmaceuticals Inc. (NASDAQ:DRNA)

DSR-PH1

EnCore: lipid nanoparticle-formulated siRNA

Conjugation platforms

GalNAc conjugate

Alnylam; Genzyme unit of Sanofi

ALN-TTRsc

GalNAc-siRNA conjugate

Phase II

Regulus Therapeutics Inc. (NASDAQ:RGLS)

RG-101

GalNAc-anti-microRNA conjugate

Phase I

Cholesterol conjugate

Arrowhead

ARC-520

DPC

Local administration

Enema

Atlantic Healthcare Ltd.; Isis Pharmaceuticals Inc. (NASDAQ:ISIS); Orphan Australia Pty. Ltd.

Alicaforsen

Delivery of antisense oligonucleotide

Phase III

Topical eye drops

Gene Signal International S.A.

Aganirsen

Intravitreal injection

iCo Therapeutics Inc. (TSX-V:ICO; OTCQX:ICOTF)

iCo-007

Injection of RNAi therapeutic

Phase II

Topical on skin

CoDa Therapeutics Inc.

Nexagon

Delivery of antisense oligonucleotide

Completed Phase II

Intravitreal injection

TransDerm Inc.

TD101

Injection of RNAi therapeutic

Completed

Phase I

Topical on skin

or eye drops

SBI Biotech Co. Ltd.; Biocon Ltd. (NSE:BIOCON; BSE:BIOCON); BioSpring GmbH

QPI-1007

Phase I