Skip to main content

Table 2 The global RNA interference pipeline*

From: A status report on RNAi therapeutics

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
  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.
  2. *All data from corporate websites, press releases and
  3. †Enrollment ongoing
  4. ‡From Koldehoff et al., 2007