Table 2

The global RNA interference pipeline*

Sponsor

Program (clinical stage)

Status

Target

Indication

Number enrolled

Alnylam/Cubist/Kyowa Kirin

ALN-RSV (phase IIb)

Ongoing

RSV nucleocapsid

Adult RSV infection

354

Pfizer/Quark

PF-04523655 (phase II)

Ongoing

RTP801

(1) AMD, diabetic macular edema

244†

Quark

QPI 1002 (phase II)

Ongoing

p53

(1) Acute kidney injury, delayed graft function

56†

Zabecor

Excellair (phase II)

Ongoing

Syk kinase

Asthma

?

Alnylam

ALN-VSP (phase I)

Ongoing

VEGF, KSP

Primary and secondary liver cancer

55†

Calando

CALAA-01 (phase I)

Ongoing

RRM2

Cancer

36†

Silence

Atu-027 (phase I)

Ongoing

PKN3

Cancer (GI, lung other)

33†

Sylentis

SYL040012 (phase I)

Ongoing

β2 adrenergic receptor

Glaucoma

?

Alnylam

ALN-TTR (phase I)

Ongoing

TTR

TTR amyloidosis

Enrollment begins H1, 2010

Opko

Bevasiranib (phase III)

Terminated

VEGF-A

AMD

522

Allergan/SIRNA

AGN211745 (phase II)

Terminated

VEGFRI

AMD

164

Tekmira

ApoB SNALP (phase I)

Completed

ApoB

Hypercholesterolemia

23

Transderm

TD101 (phase I completed)

Completed

Mutant K6a

Pachyonychia congenita

1

Univ. Duisberg-Essen‡

Bcr-abl (phase I completed)

Unknown

Bcr-abl oncogene

CML

1

AMD = age-related macular degeneration; CML = chronic myeloid leukemia; GI = gastrointestinal; KSP = kinesin spindle protein; PKN = protein kinase N3; RRM2 = ribonucleotide reductase M2; RSV = respiratory syncytial virus; RTP = ; TTR = transthyretin; VEGF = vascular endothelial growth factor; VEGFRI = vascular endothelial growth factor receptor I.

*All data from corporate websites, press releases and http://www.clinicaltrials.gov webcite

†Enrollment ongoing

‡From Koldehoff et al., 2007

Vaishnaw et al. Silence 2010 1:14   doi:10.1186/1758-907X-1-14