Ewing's Sarcoma: Development of RNA Interference-Based Therapy for Advanced Disease

Table 1

RNAi drugs in human clinical trials [113–115].

Company	Product name	Disease	Target	Stage
Alnylam	ALN-RSV1	Respratory syncytial virus infection	Nucleocapsid (N) gene of RSV genome	Expanded; phase II
Alnylam	ALN-VSP	Liver cancers and solid tumors	Kinesin spindle protein (KSP), VEGF	Phase I
Alnylam	ALN-TTR01	TTR-mediated amyloidosis	TTR	Phase I
Alnylam	ALN-PCS	hypercholesterolemia	PCSK9	Phase I
Benetec/City of Hope	—	AIDS lymphoma	rHIV7-shI-TAR-CCR5RZ	Phase I
Calando Pharmaceuticals	CALAA-01	Cancer and solid tumors	M2 subunit of ribonucleotide reductase (RRM2)	Phase I
Cequent Pharmaceuticals	CEQ508	FAP	β-Catenin	Phase I
Duke University	—	Metastatic melanoma	LMP2, LMP7, and MECL1	Phase I
OPKO Health	Bevasiranib	Wet age-related macular degeneration	VEGF	Expanded; phase III
OPKO Health	Bevasiranib	Diabetic macular edema	VEGF	Phase II
Quark Pharmaceuticals	PF4523655/RTP801i14	Wet age-related macular degeneration	RTP801	Phase II
Quark Pharmaceuticals	PF4523655/RTP801i14	Diabetic macular edema	RTP801	Phase II
Quark Pharmaceuticals	QPI-1002/Akli5/I5NP	Acute kidney injury	P53	Phase I/IIa
Quark Pharmaceuticals	QPI-1002/DGFi	Delayed graft function in kidney transplantation	P53	Phase I/II
Sirna Therapeutics (MERCK)/Allergan	Sirna-027/AGN-745	Wet age-related macular degeneration	VEGFRI	Phase II
Silence Therapeutics	Atu027	Lung cancers	Protein kinase N3 (PKN3)	Phase I
SENETEK	—	Brain tumors glioblastomas	Tenascin-C	Phase I
Tekmira	ApoB SNALP	High LDL cholesterol	Apo B lipoprotein	Phase I/II
Tekmira	TKM-PLK1	Advanced solid tumor	PLK1	Phase I
TransDerm, Inc	TD101	Pachyonichia congenita	Keratin 6a (K6a)	Phase I
University of Duisbur-Essen	—	Chronic myeloid leukaemia	bcr-abl	Single patient
Gradalis, Inc.	FANG	Advanced cancer	Furin	Phase I
Gradalis, Inc.	FANG	Ovarian	Furin	Phase II
Gradalis, Inc.	FANG	Melanoma	Furin	Phase II
Gradalis, Inc.	FANG	Colon cancer	Furin	Phase II