Antisense Oligonucleotides and RNA Interference - Challenges in Delivery of Therapeutic Genomics and Proteomics - page 325

Table 7.1 Clinical Status of siRNA Formulations

S. No. Company and

Strategic Alliances

Product Details Clinical Status as

on Dec. 2009

Drug Target/
Tissue

Indication Route of

Delivery

Delivery System

1. Acuity/later licensed

by Opko

siRNA Cand5/
Bevasiranib

Terminated at
Phase III

VEGF/Eye AMD Intravitreal

injections

Naked siRNA

VEGF/Eye Diabetic

retinopathy

Intravitreal
injections

Naked siRNA

2. Sirna Therapeutics/

Later acquired by
Allergan

Sirna-027/Now
AGN-745

Terminated at
Phase II

VEGF/Eye AMD Intravitreal

injections

Naked siRNA

3. Silence Therapeutics/

Quark/Pfizer

RTP-801i Phase II VEGF/Eye AMD Intravitreal

injections

Naked siRNA

Atu027 Phase I Targets PKN3

molecule in
cancer cells

Cancer Intravenous siRNA incorporated

in AtuPLEX
delivery platform

AKIi-5 Phase I/II P53 gene/

kidney

Acute kidney
injury in kidney
transplantation

Intravenous Chemically

modified siRNA
with AtuRNAi
technology DGFi Phase I/II Delayed graft

function
in kidney
transplantation

Intravenous

4. Alnylam

Pharmaceuticals

ALN-RSV01 Phase III RSV

nucleocapsid
/lungs

Respiratory
syncytial virus
(RSV) infection

Intranasal Naked siRNA

ALN-VSP Phase I Kinesin spindle

protein (KSP)
and VEGF/liver

Liver cancer Intravenous Two siRNA

molecules
formulated in lipid
nanoparticles