Citeline Drug ID Generic Drug Name Drug Names

142511 AB-729 AB 729; AB-729; AB729; GalNAc RNAi therapy,

150947 ALN-HSD Arbutus
ALN HSD; ALN-HSD; ALNHSD; NASH therapy,
168355 ALN-REGN4 Alnylam; NASH
ALN REGN4; therapy, Regeneron
ALN-REGN E1; ALN-REGN-E1; ALN-
197840 AMG-609 REGN4; ALN-REGNE1; ALNREGN4;
AMG 609; AMG-609; AMG609 cemdisiran +
71131 AMT-130 AAV5-miHTT, Uniqure; AMT 130; AMT-130; AMT130;
71748 APN-401 Huntington's diseaseAPN401
APN 401; APN-401; therapy, AMT
117164 ARC-HIF2 ARC HIF2; ARC-HIF2; ARCHIF2; ARO-HIF2; AROHIF2
134469 ARO-AAT ARO AAT; ARO-AAT; AROAAT; TAK 999; TAK-999;
144959 ARO-ANG3 TAK999
ARO ANG3; ARO-ANG3; AROANG3
144958 ARO-APOC3 ARO APOC3; ARO-APOC3; AROAPOC3
170030 ARO-HSD ARO HSD; ARO-HSD; AROHSD
41612 bamosiran bamosiran; glaucoma therapy, Sylentis; SYL-040003;
128189 belcesiran SYL-040012;
belcesiran; DCR SYL040003; SYL040012
A1AT; DCR-A1AT; DCRA1AT; DRNA
82653 cemdisiran 17, Dicerna; liver disease therapy,
ALN CC5; ALN-CC5; ALNCC5; cemdisiran Dicerna
40196 cotsiranib cotsiranib; cotsiranib (Intradermal); cotsiranib
161518 DCR-CM2 (intratumoral);
DCR-CM2; Cutasil; LY
DCRCM2; Cutasil (Intradermal);
3819469; Cutasil
LY-3819469;
131679 DCR-HBVS LY3819469
DCR-HBVS; DCR-S219; DCRS-219; DCRS219;
73087 fitusiran DsiRNA-HBV, GalXC,ALN-AT3;
ALN AT3; ALN-APC; Dicerna; RG 6346; fitusiran;
ALNAT3; RG-6346;SAR
80245 givosiran 439774;
ALN AS1;SAR-439774; SAR439774
ALN-AS1; ALNAS1; Givlaari; givosiran
82624 hepatitis B siRNA, Life hepatitis B siRNA, Life Technologies; hepatitis B
119878 Technologies
inclisiran siRNA,
ALN-PCS Suzhou
(SC); Ribo Life Science;
ALN-PCS01 (SC); RB HBV008; RB-
ALN-PCSsc; ALN-
135209 INT-1B3 PCSsc (SC); anticholesterolaemic,
INT 1B3; INT-1B3; INT1B3 Alnylam-2 (SC);
134467 JNJ-3989 ARO HBV; ARO-HBV; AROHBV; JNJ 3989; JNJ
90855 lumasiran 73763989; JNJ-3989; ALNGO1;
ALN GO1; ALN-GO1; JNJ-73763989; JNJ3989;
lumasiran; Oxlumo
161517 LY-3561774 DCR-CM1; DCRCM1; LY 3561774; LY-3561774;
160766 NBF-006 LY3561774
NBF 006; NBF-006; NBF006
67955 ND L02-s0201 BMS 986263; BMS-986263; BMS986263; fibrotic
113336 nedosiran diseases
DCR programme,
- PHsc; NittoDCR-PH1
DCR PHXC; Denko; fibrotic diseases
(sc); DCR-PH1
118311 olpasiran (subcutaneous);
AD-01765; AD01765; DCR-PHsc; DCR-PHXC;
AMG 890, DRNA 16.1;
Amgen; AMG-890;
91919 OLX-10010 (SC) AMG890;
BMT 101; ARC-LPA
BMT-101; programme; ARO-LPA;Hugel;
BMT101; cp-asiRNA, AROLPA;
OLX
64853 patisiran 10010 (Intradermal); OLX 10010 (SC); OLX
ALN TTR02; ALN-TTR, second-generation; ALN- 101; OLX
133971 Pbi-shRNA, Gradalis TTR02; ALNTTR02;
EWS/FLI1 amyloidosis
type 1 Lipoplex, therapy,
Gradalis; Alnylam-2;
EWS/FLI1 type 1
42022 QPI-1007 LPX, Gradalis; Pbi-shRNA, Gradalis
QPI 1007; QPI-1007; QPI1007; RB RQ007; RB-RQ007;
128349 retinitis therapy, Sylentis RBRQ
retinitis007; RBRQ-007;
therapy, Sylentis;RBRQ007;
SYL 1801;SR 061; SR-061;
SYL-1801;
71740 siG12D LODER SYL1801
KRAS-LODER; siG12D LODER; siG12D-LODER
143816 SLN-124 iron overload GalNAc-siRNA therapy, Silence
143817 SLN-360 Therapeutics;
cardiovascularSLN 124;GalNAc-siRNA
disease SLN-124; SLN124
therapy, Silence
160122 SRN-14 Therapeutics; SLN 360; SLN-360; SLN360
GL2 800; GL2-800; GL2800; PRDM14 siRNA/uPIC,
41131 stathmin-1 shRNA, Gradalis AccuRna;
anticancerSRN 14; SRN-14;
bifunctional SRN14
shRNA, Gradalis; pGBI-2;
30445 teprasiran shRNASTMN1;
15NP; stathmin-1
AKIi-5; AKLi-5; shRNA,
AtuRNAi, Gradalis;
acute kidney STMN1
injury;
66785 tivanisiran p53
SYL-1001; SYL1001; SYL1001 DP; tivanisiran QPI
siRNA, Quark; PFT-101B; PFTi; QP-1002;
146103 VIR-2218 ALN HBV02; ALN-HBV02; ALNHBV02; BRII 835; BRII-
90304 zilebesiran 835;
AGT BRII835; VIR 2218;
siRNA, Alnylam; VIR-2218;
ALN AGT; ALNVIR2218
AGT01; ALN-
AGT; ALN-AGT01; ALNAGT01; zilebesiran
Summary Global Status
AB-729 is a GalNAc RNAi therapy under development by Phase II Clinical Trial
Arbutus
ALN-HSD Biopharma
is an RNAi fortherapeutic
hepatitis Btargeting
surface antigen and/or
HSD17B13, under Phase I Clinical Trial
development by Alnylam in collaboration with
ALN-REGN-E1 (ALN-REGN4) is developing a combination Regeneron forof Phase II Clinical Trial
cemdisiran
AMG-609 isand pozelimab,
a small under
interfering RNA development
(siRNA) thatbyselectively
Alnylam in Phase I Clinical Trial
targets a variant allele of patatin like phospholipase
AMT-130 comprises an AAV5 vector carrying an artificial domain Phase II Clinical Trial
micro-RNA
APN-401 is specifically
a siRNA based tailored to silence
autologous the huntingtin
cellular therapy gene, Phase I Clinical Trial
targeting
ARC-HIF2Cbl-b, under development
(ARO-HIF2) by Apeironunder
is a RNAi therapeutic, Biologics for Phase I Clinical Trial
development by Arrowhead
ARO-AAT (TAK-999) Pharmaceuticals
is an RNAi (formerly to
therapeutic designed Phase III Clinical Trial
knock down the hepatic production of the mutant
ARO-ANG3 is a double stranded oligomer RNAi molecule alpha-1 Phase II Clinical Trial
targeting
ARO-APOC3 angiopoietin-like protein 3oligomer
is a double stranded (ANGPTL3),
RNAiunder
molecule Phase II Clinical Trial
targeting apolipoprotein C-III leveraging the targeted
ARO-HSD is a RNAi therapeutic that reduces production RNAiof Phase II Clinical Trial
HSD17B13, a hydroxysteroid
Bamosiran (SYL-040012) is a dehydrogenase,
small interfering under
RNA (siRNA) Phase II Clinical Trial
agent, under development by Sylentis
Belcesiran (DCR-A1AT) is an investigational for thetherapy,
treatment of
under Phase II Clinical Trial
development by Dicernaisby
Cemdisiran (ALN-CC5) using therapeutic
a RNAi its GalXC RNAi technology
targeting the C5 Phase II Clinical Trial
component of the complement pathway, under development
Cotsiranib (STP-705; STP-705L) is a small interfering RNA Phase II Clinical Trial
(siRNA) therapeutic
DCR-CM2 targeting
(LY-3819469) is anTGF-ß1 and COX-2
sc administered mRNA,
RNAi Phase I Clinical Trial
therapeutic,
RO-7445482under development
(RG-6346; DCR-HBV)by Dicerna forSubstrate
is a Dicer Phase II Clinical Trial
siRNA
Fitusiran(DsiRNA), under
(ALN-AT3; development
SAR-439774) by siRNA
is an Dicerna using its'
therapeutic Phase III Clinical Trial
targeting
Givosiranantithrombin
(ALN-AS1) is(AT), undertherapeutic
an RNAi development by Alnylam,
targeting Launched
aminolevulinate
RB-HBV008 synthase
(SR-016; 1 (ALAS-1)
RBD-1016) is adeveloped by Alnylam
siRNA molecule acts Phase I Clinical Trial
by
Inclisiran (ALN-PCSsc; KJX-839) is an RNA interference for
reducing HBsAG, under development by Suzhou Ribo Registered
(RNAi)
INT-1B3therapeutics directed to proprotein
is an LNP-formulated, chemically convertase
modified miR- Phase I Clinical Trial
193a-3p
JNJ-3989miRNA mimic,JNJ-73763989)
(ARO-HBV; under development by InteRNA
is a RNAi therapeutic Phase II Clinical Trial
that inhibits viral RNA polymerase, under development
Lumasiran (ALN-GO1) is a subcutaneously administered by Launched
RNAi therapeutic, developed by Alnylam,
LY-3561774 (DCR-CM1) is an sc administered drug utilizing its ESC- Phase I Clinical Trial
candidate,
NBF-006 isunder development
a novel by Dicerna
lyophilized lipid by using
nanoparticle its GalXC
formulation, Phase I Clinical Trial
under development
ND-L02-s0201 by Nitto Denko
(BMS-986263) for the treatment
is a vitamin A-coupledof KRAS
lipid Phase II Clinical Trial
nanoparticle-containing
Nedosiran (DCR-PHXC)siRNA against HSP-47,
is an RNAi-based under
inhibitor of lactate Phase III Clinical Trial
dehydrogenase
Olpasiran (AD-01765; A (LDHA), under is
AMG-890) development by Dicerna
the lead compound in the Phase II Clinical Trial
ARC-LPA programme of RNAi therapeutics
OLX-101 (BMT-101) is a RNAi based connective tissue designed to Phase II Clinical Trial
growth factor
Patisiran inhibitor(CTGF
(ALN-TTR02; inhibitor)
GZ-438027; and angiogenesis
GENZ-438027) is a 2nd- Launched
generation
Gradalis siRNA therapy
is developing targeting the
a bifunctional transthyretin
short hairpin RNA gene, Phase I Clinical Trial
(shRNA) EWS/FLI1 type 1 lipoplex for the treatment
QPI-1007 (SR-061) is a small interfering RNA (siRNA), is of Phase III Clinical Trial
under development
SYL-1801 is a smallby Suzhoutargeting
molecule Ribo LifeNRARP
Scienceadministered
as a Phase I Clinical Trial
topically,
Silenseedunder development
is developing siG12D by Sylentis using its
LODER (Local RNA
Drug EluteR), Phase II Clinical Trial
a siRNA local-delivery
SLN-124 implant,
is a GalNAc-siRNA for the treatment
conjugate targetingof pancreatic
TMPRSS6, Phase I Clinical Trial
under
SLN-360development by Silence
is a GalNAc-siRNA Therapeutics
conjugate for the
targeting treatment
LPA, under Phase I Clinical Trial
development
SRN-14 (GL2-800)by Silence
is an Therapeutics for the treatment
siRNA/uPIC involves of
unit poly ion Phase I Clinical Trial
complex
Gradalis is(uPIC) containing
developing PRDM14-specific
a bifunctional chimera
short hairpin RNAsiRNA, Phase I Clinical Trial
(shRNA) construct
Teprasiran (QPI-1002)directed at stathminand
is a temporary 1 (STMN1),
reversibleasmall Phase III Clinical Trial
interfering RNA (siRNA) molecule against
Tivanisiran (SYL-1001) is under development by p53, under
Sylentis Phase III Clinical Trial
using its RNA interference technology for the
ALN-HBV02 (VIR-2218; BRII-835) is a siRNA therapeutic,treatment of dry Phase II Clinical Trial
under development
Zilebesiran (ALN-AGT) by Alnylam
is an sc utilizing Vir Biotech's
administered ESC+
long-lasting Phase II Clinical Trial
RNAi therapeutic targeting angiotensinogen (AGT), under
Development Status Drug Disease
Active Infection, hepatitis-B virus
Active Infection, hepatitis-B
Non-alcoholic virus
steatohepatitis
Active Non-alcoholic steatohepatitis
Paroxysmal nocturnal haemoglobinuria
Active Paroxysmal nocturnal haemoglobinuria
Non-alcoholic steatohepatitis
Active Huntington's disease
Active Cancer, colorectal
Active Cancer,
Cancer, solid,
renal unspecified
Active Hepatic dysfunction, alpha-1 antitrypsin deficiency
Active related
Hyperlipidaemia, unspecified
Active Hyperlipidaemia, unspecified
Hypertriglyceridaemia
Active Hypertriglyceridaemia
Non-alcoholic steatohepatitis
Active Non-alcoholic
Glaucoma steatohepatitis
Active Glaucoma
Hepatic dysfunction, alpha-1 antitrypsin deficiency
Active related
Berger's disease
Active Berger's disease
Cancer, basal cell
Active Cancer, skin, unspecified
Alimentary/metabolic disease, unspecified
Active Alimentary/metabolic disease, unspecified
Infection, hepatitis-B virus
Active Infection,
Haemophiliahepatitis-B
A virus
Active Haemophilia
Porphyria A
Active Porphyria
Infection, hepatitis-B virus
Active Infection, hepatitis-B virus
Atherosclerosis
Active Atherosclerosis
Cancer, solid, unspecified
Active Cancer,
Infection,solid, unspecified
hepatitis-B virus
Active Infection, hepatitis-B
Hyperoxaluria virus
Active Hyperoxaluria
Hyperlipidaemia, unspecified
Active Hyperlipidaemia,
Cancer, colorectalunspecified
Active Cancer,
Cirrhosis,lung, non-small cell
hepatic
Active Cirrhosis, hepatic
Hyperoxaluria
Active Hyperoxaluria
Cardiovascular disease, unspecified
Active Cardiovascular
Keloid scarring disease, unspecified
Active Keloid scarring
Amyloidosis, transthyretin-related hereditary
Active Amyloidosis,
Cancer, transthyretin-related
sarcoma, Ewing's hereditary
Active Neuropathy, ischaemic optic
Active Neuropathy, ischaemic age-related,
Macular degeneration, optic unspecified
Active Cancer, pancreatic
Active Cancer, pancreatic
Myelodysplastic syndrome
Active Thalassaemia
Cardiovascular disease, unspecified
Active Cancer, breast
Active Cancer, solid, unspecified
Active Acute renal failure
Active Acute
Dry eyerenal failure
syndrome
Active Dry eye syndrome
Infection, hepatitis-B virus
Active Infection, hepatitis-B
Hypertension, virus
unspecified
Hypertension, unspecified
Company Originator / Company HQ Company HQ
Arbutus Biopharma Licensee
Originator Country
Canada State
BC
Arbutus
Alnylam Biopharma Originator
Originator Canada
USA BC
MA
Regeneron
Regeneron Licensee
Licensee USA
USA NY
NY
Regeneron
Amgen Licensee
Originator USA
USA NY
CA
uniQure Originator Netherlands
Apeiron Biologics Originator Austria
Apeiron Biologics
Arrowhead Pharmaceuticals Originator
Originator Austria
USA CA
Arrowhead Pharmaceuticals Originator USA CA
Arrowhead
Arrowhead Pharmaceuticals
Pharmaceuticals Originator
Originator USA
USA CA
CA
Arrowhead
Arrowhead Pharmaceuticals
Pharmaceuticals Originator
Originator USA
USA CA
CA
Arrowhead
Arrowhead Pharmaceuticals
Pharmaceuticals Originator
Originator USA
USA CA
CA
Arrowhead
PharmaMarPharmaceuticals Originator
Originator USA
Spain CA
PharmaMar
Dicerna Originator
Originator Spain
USA MA
Dicerna
Alnylam Originator
Originator USA
USA MA
MA
Alnylam
Sirnaomics Originator
Originator USA
USA MA
MD
Sirnaomics
Eli Lilly Originator
Licensee USA
USA MD
IN
Eli Lilly
Dicerna Licensee
Originator USA
USA IN
MA
Dicerna
Alnylam Originator
Originator USA
USA MA
MA
Alnylam
Alnylam Originator
Originator USA
USA MA
MA
Alnylam
Suzhou Ribo Life Science Originator
Licensee USA
China MA
Suzhou
Alnylam Ribo Life Science Licensee
Originator China
USA MA
Alnylam
InteRNA Technologies Originator
Originator USA
Netherlands MA
InteRNA
Arrowhead Technologies
Pharmaceuticals Originator
Originator Netherlands
USA CA
Arrowhead
Alnylam Pharmaceuticals Originator
Originator USA
USA CA
MA
Alnylam
Dicerna Originator
Originator USA
USA MA
MA
Eli Lilly
Nitto Denko Licensee
Originator USA
Japan IN
Nitto Denko Squibb
Bristol-Myers Originator
Licensee Japan
USA NY
Bristol-Myers Squibb
Dicerna Licensee
Originator USA
USA NY
MA
Dicerna
Amgen Originator
Licensee USA
USA MA
CA
Amgen
Hugel Licensee
Licensee USA
South Korea CA
OliX Pharmaceuticals
Alnylam Originator
Originator South
USA Korea MA
Alnylam
Gradalis Originator
Originator USA
USA MA
TX
Kunshan RiboQuark Licensee China
Suzhou Ribo Life Science
PharmaMar Licensee
Originator China
Spain
Silenseed Originator Israel
Silenseed
Silence Therapeutics Originator
Originator Israel
UK
Silence
Silence Therapeutics
Therapeutics Originator
Originator UK
UK
NanoCarrier Originator Japan
Gradalis Originator USA TX
Quark Pharmaceuticals Originator USA CA
Quark Pharmaceuticals
PharmaMar Originator
Originator USA
Spain CA
PharmaMar
Alnylam Originator
Originator Spain
USA MA
Brii Biosciences
Alnylam Licensee
Originator China
USA MA
Alnylam Originator USA MA
Company HQ City Company HQ Postal Code Company
Burnaby V5J 5J8 (Subsidiary/Acquiree)
Burnaby
Cambridge V5J 5J8
02142
Tarrytown
Tarrytown 10591
10591
Tarrytown
Thousand Oaks 10591
91320-1799
Amsterdam 1105 BA
Vienna 1030
Vienna
Pasadena 1030
91101
Pasadena 91101
Pasadena
Pasadena 91101
91101
Pasadena
Pasadena 91101
91101
Pasadena
Pasadena 91101
91101
Pasadena
Madrid 91101
28770 Sylentis
Madrid
Cambridge 28770
02140 Sylentis
Cambridge
Cambridge 02140
02142
Cambridge
Gaithersburg 02142
20879
Gaithersburg
Indianapolis 20879
46285
Indianapolis
Cambridge 46285
02140
Cambridge
Cambridge 02140
02142
Cambridge
Cambridge 02142
02142
Cambridge
Kunshan 02142
215300
Kunshan
Cambridge 215300
02142
Cambridge
Nijmegen 02142
6534
Nijmegen
Pasadena 6534
91101
Pasadena
Cambridge 91101
02142
Cambridge
Cambridge 02142
02140
Indianapolis 46285
New York 10154
New York
Cambridge 10154
02140
Cambridge
Thousand Oaks 02140
91320-1799
Thousand
ChuncheonOaks 91320-1799
200-821
Seoul
Cambridge 153-777
02142
Cambridge
Dallas 02142
75201
Kunshan
Kunshan
Madrid 215300
28770 Sylentis
7177871
London 7177871
W6 0NB
London
London W6
W6 0NB
0NB
Kashiwa 277-0871 AccuRna
Dallas 75201
Fremont 94555
Fremont
Madrid 94555
28770 Sylentis
Madrid
Cambridge 28770
02142 Sylentis
Shanghai
Cambridge 02142
Cambridge 02142
Company Company Company Company
(Subsidiary/Acquiree (Subsidiary/A (Subsidiary/Acquiree) HQ (Subsidiary/Acquiree) HQ

Spain Madrid 28760

Spain Madrid 28760

Spain Madrid 28760

Japan Tokyo

Spain Madrid 28760

Spain Madrid 28760
Drug Country Drug Country Current Status Launch Year Highest Status Reached
Canada Region
North America Phase II Clinical Trial Phase II Clinical Trial
Australia
UK Oceania
Europe, EU Phase
Phase II Clinical
Clinical Trial
Trial Phase
Phase II Clinical
Clinical Trial
Trial
UK
USA Europe, EU
North America Phase
Phase II ClinicalTrial
I Clinical Trial Phase
Phase II ClinicalTrial
I Clinical Trial
Netherlands
USA Europe, EU
North America Phase I Clinical Trial
Phase I Clinical Trial Phase I Clinical Trial
Phase I Clinical Trial
USA North America Phase II Clinical Trial Phase II Clinical Trial
Austria Europe, EU Phase I Clinical Trial Phase I Clinical Trial
USA
USA North
North America
America Phase
Phase II Clinical
Clinical Trial
Trial Phase
Phase II Clinical
Clinical Trial
Trial
USA North America Phase III Clinical Trial Phase III Clinical Trial
Germany
Australia Europe,
Oceania EU Phase
Phase II
II Clinical
Clinical Trial
Trial Phase
Phase II
II Clinical
Clinical Trial
Trial
New Zealand
Australia Oceania
Oceania Phase
Phase II Clinical Trial
II Clinical Trial Phase
Phase II Clinical Trial
II Clinical Trial
Canada
New Zealand North America
Oceania Phase
Phase II Clinical Trial
II Clinical Trial Phase
Phase II Clinical Trial
II Clinical Trial
USA
Germany North America
Europe, EU Phase
Phase II Clinical Trial
II Clinical Trial Phase
Phase II Clinical Trial
II Clinical Trial
Spain
New Zealand Europe,
Oceania EU Phase
Phase II
II Clinical
Clinical Trial
Trial Phase
Phase II
II Clinical
Clinical Trial
Trial
Sweden
Canada Europe, EU
North America Phase
Phase II Clinical Trial
II Clinical Trial Phase
Phase II Clinical Trial
II Clinical Trial
France
USA Europe, EU
North America Phase
Phase II Clinical Trial
II Clinical Trial Phase
Phase II Clinical Trial
II Clinical Trial
USA
Singapore North America
Asia Phase IIIClinical
Phase ClinicalTrial
Trial Phase IIIClinical
Phase ClinicalTrial
Trial
USA
Australia North America
Oceania Phase
Phase IIIClinical
ClinicalTrial
Trial Phase
Phase IIIClinical
ClinicalTrial
Trial
Hong Kong
Australia Asia
Oceania Phase
Phase III ClinicalTrial
II Clinical Trial Phase
Phase III ClinicalTrial
II Clinical Trial
Brazil
Canada South America
North America Phase III
Launched Clinical Trial 2021 Phase III
Launched Clinical Trial
Germany
Australia Europe, EU
Oceania Launched
Phase I Clinical Trial 2020 Launched
Phase I Clinical Trial
China
Austria Asia
Europe, EU Phase I Clinical Trial
Registered Phase I Clinical Trial
Registered
Belgium
Belgium Europe,
Europe, EU
EU Registered
Phase I Clinical Trial Registered
Phase I Clinical Trial
Netherlands
Australia Europe,
Oceania EU Phase
Phase IIIClinical
ClinicalTrial
Trial Phase
Phase IIIClinical
ClinicalTrial
Trial
Hong Kong
Germany Asia
Europe, EU Phase II
Launched Clinical Trial 2021 Phase II
Launched Clinical Trial
USA
USA North
North America
America Launched
Phase I Clinical Trial 2020 Launched
Phase I Clinical Trial
USA
USA North
North America
America Phase
Phase II Clinical
Clinical Trial
Trial Phase
Phase II Clinical
Clinical Trial
Trial
USA
Argentina North
South America
America Phase
Phase II ClinicalTrial
I Clinical Trial Phase
Phase II ClinicalTrial
I Clinical Trial
Belgium
Australia Europe, EU
Oceania Phase III
Phase II Clinical
ClinicalTrial
Trial Phase III
Phase II Clinical
ClinicalTrial
Trial
France
Australia Europe,
Oceania EU Phase
Phase III ClinicalTrial
II Clinical Trial Phase
Phase III ClinicalTrial
II Clinical Trial
Canada
South Korea North
Asia America Phase
Phase II
II Clinical
Clinical Trial
Trial Phase
Phase II
II Clinical
Clinical Trial
Trial
South Korea
Belgium Asia
Europe, EU Phase II
Launched Clinical Trial 2020 Phase II
Launched Clinical Trial
Bulgaria
USA Europe,
North EU
America Launched
Phase I Clinical Trial 2021 Launched
Phase I Clinical Trial
China Asia Phase III Clinical Trial Phase III Clinical Trial
China
Spain Asia
Europe, EU Phase
Phase III ClinicalTrial
I Clinical Trial Phase
Phase III ClinicalTrial
I Clinical Trial
Israel Asia Phase II Clinical Trial Phase II Clinical Trial
USA
UK North America
Europe, EU Phase IIIClinical
Phase ClinicalTrial
Trial Phase IIIClinical
Phase ClinicalTrial
Trial
UK
USA Europe, EU
North America Phase I Clinical Trial
Phase I Clinical Trial Phase I Clinical Trial
Phase I Clinical Trial
Japan Asia Phase I Clinical Trial Phase I Clinical Trial
USA North America Phase I Clinical Trial Phase I Clinical Trial
Australia Oceania Phase III Clinical Trial Phase III Clinical Trial
Austria
Estonia Europe,
Europe, EU
EU Phase
Phase III
III Clinical
Clinical Trial
Trial Phase
Phase III
III Clinical
Clinical Trial
Trial
Germany
New Zealand Europe,
Oceania EU Phase III Clinical Trial
Phase II Clinical Trial Phase III Clinical Trial
Phase II Clinical Trial
China
USA Asia
North America Phase
Phase II
II Clinical
Clinical Trial
Trial Phase
Phase II
II Clinical
Clinical Trial
Trial
UK Europe, EU Phase I Clinical Trial Phase I Clinical Trial
Partnering Availability: Latest Change Latest Change Event Date
Country Date 7/27/2021 Phase Ia/Ib trial (AB-729-001) trial results 2021/03/15
7/2/2021 reported
Ongoing Phase I trial (ALN-HSD-001) 2019/12/31
2020/10/19
8/19/2021 confirmed
Initiation ofper Jun II
Phase presentation
trial for paroxysmal 2019/04/15
2021/08/13
nocturnal haemoglobinuria by Regeneron
6/25/2021 Initiation of Phase I trial (20200001) for 2021/03/09
2021/06/24
Argentina; Australia; Austria; NASH reported
8/31/2021 DSMB recommendation for Phase I/II trial 2021/04/27
2020/08/21
Belgium; Brazil; Canada; Chile; 7/8/2021 (CT-AMT-130-01)
Ongoing development in Huntington
confirmeddisease
per 2019/10/18
2019/03/30
Apeiron Biologics pipeline
8/1/2021 Interim results of Phase Ib trial 2018/02/01
2020/05/14
7/31/2021 (AROHIF21001)
Breakthrough therapyin renal cancer reported
designation for 2019/12/11
2021/07/29
alpha-1 antitrypsin deficiency reported
5/29/2021 Initiation of Phase IIb trial (AROANG3- 2021/04/14
2021/05/28
8/12/2021 Planned Phase IIb trial (AROAPOC3-2002) 2020/11/19
2001) in mixed dyslipidaemia reported 2021/03/01
for mixed dyslipidemia reported
6/24/2021 Interim results of Phase I/II 2019/06/20
2020/03/03
(AROHSD1001) trial reported
8/12/2021 Ongoing development confirmed per 2019/12/17
2014/07/10
Sylentis web page
7/29/2021 Phase I trial results reported 2010/10/26
2021/01/25
9/2/2021 EU orphan drug status for IgA nephropathy 2020/03/18
2021/08/20
reported
7/24/2021 Preclinical results reported 2019/03/12
2021/03/26
7/8/2021 Initiation of Phase I trial (J3L-MC-EZEA) 2021/01/17
2021/07/07
4/22/2021 reported
Expected timing of filing in the EU and the 2021/05/27
2020/11/16
8/4/2021 US reportedof Phase III trial (ATLAS-INH)
Completion 2019/10/31
2017/12/15
China; Hong Kong; India; 8/5/2021 for haemophilia
Results A and
from Phase III B reported and
(ENVISION) 2017/09/07
2021/08/04
Japan; Malaysia; Philippines; new approvals
8/26/2021 Ongoing PhaseinI trial
Japan and Israel for AHP
(RBHB1103-HK) for 2021/04/29
2021/04/15
Argentina; Australia; Brazil; chronic HBV infection reported
9/8/2021 Planned Phase III trial (VICTORION-2P) 2020/12/29
2020/12/11
Canada; Chile; China; 4/16/2021 for atherosclerosis
Receipt of grant from reported
the Netherlands 2020/02/06
2021/01/06
Enterprise Agency reported
9/3/2021 Phase II (AROHBV1001) study results 2020/05/06
2020/10/01
8/23/2021 reported
Results from Phase III trial (ILLUMINATE- 2020/09/02
2021/08/03
7/2/2021 A) and expected
Ongoing Phase I timing of launches
trial (17825) in
confirmed 2021/02/11
2021/01/29
8/2/2021 per ADA presentation
Ongoing Phase I (NBF-006- 2021/01/29
2019/02/07
Argentina; Australia; Austria; 8/31/2021 Initiation of Phase confirmed
001)development per clinical
II trial (IM025-017) for 2018/12/08
2019/04/03
Belgium; Brazil; Canada; Chile; hepatic cirrhosis
8/30/2021 Planned Phase IIreported
trial (PHYOX8) for 2018/06/01
2020/04/06
8/30/2021 hyperoxaluria reported
Initiation of Phase I trial (20190095) for 2019/08/02
2020/10/28
5/10/2021 Ongoing Phase IIa trial reported
cardiovascular disease (OLX10010-02) for 2020/10/28
2021/05/18
hypertrophic scarring reported
8/26/2021 Results of Phase III trial (ALN-TTR02-006) 2019/11/11
2021/02/11
for transthyretin-mediated
9/8/2020 Ongoing development confirmed amyloidosis
from 2021/02/11
2016/11/25
company pipeline
5/5/2021 Planned Phase III trial for non-arteritic 2020/12/29
3/30/2021 anterior
Initiationischaemic
of Phase Ioptic neuropathy and
trial (SYL1801_I) 2020/12/29
2021/03/29
7/7/2021 reported
Additional details of Phase II trial 2018/05/04
2019/07/19
(SLSG12D-P2)
9/7/2021 Ongoing Phase for pancreatic cancer
I development confirmed 2018/02/26
2020/09/08
per Silence Therapeutics presentation
9/7/2021 Ongoing Phase I development confirmed 2020/06/26
2020/12/10
12/8/2020 per Silence
Ongoing Therapeutics
Phase presentation
I trial for breast cancer in 2019/01/15
2020/09/03
Japan reported
9/8/2020 Ongoing Phase I development confirmed 2020/09/03
2019/09/26
per company
7/9/2021 Ongoing pipeline confirmed per
development 2018/05/22
2019/03/26
Quark pipeline
5/29/2021 Initiation of Phase III trial (SYL1001_V) for 2018/06/14
2017/10/19
8/23/2021 dry eye syndrome
Completion of Phase reported
I/II trial (VIR-2218- 2017/06/01
2020/08/11
1001) for hepatitis-B virus reported for
7/5/2021 Details of Phase II trial (KARDIA-1) 2018/12/06
2021/06/30
hypertension reported 2021/06/29
Event Type Event Details
Global Status Advance Phase II Clinical Trial
Change in Licensee
Global Status Status
Advance Roivant
Phase I Sciences; No development reported
Clinical Trial
Lead
GlobalIdentified
Status Advance NASH
Phase therapy,
II ClinicalAlnylam
Trial
Global
Global Status
Status Advance
Advance Phase I Clinical Trial
Phase I Clinical Trial
Drug Added
Disease Phase Change Preclinical
Haemophilia B; No Development Reported
Global
DiseaseStatus
PhaseAdvance
Change Phase
Cancer,II melanoma;
Clinical TrialCancer, pancreatic; Cancer, renal, Cancer, colorectal;
New Disease
Global Status Advance NDR
Phase I Clinical Trial
IND Filing Review Designation Granted The
Expedited The US;
US; Cancer, renal; Clear cell
Hepatic dysfunction, renalantitrypsin
alpha-1 cell carcinoma
deficiency related;
New Mechanism
Disease Phase Change Breakthrough therapy designation
Mixed dyslipidemia; Phase II Clinical Trial
New
New Disease
Disease Mixed
Mixed dyslipidemia
dyslipidemia
Orphan Drug Status
Global Status Advance Granted The
Phase I/IILipoprotein
US; lipase deficiency
Clinical Trial
New Disease Name Granted
Nonproprietary Non-alcoholic
SYL-040012 steatohepatitis
Global Status Advance
Nonproprietary Name Granted Phase I/II Clinical Trial
DCR-A1AT
Orphan
Orphan Drug
Drug Status
Status Granted
Granted The
The US;
EU; Hepatic
Berger'sdysfunction,
disease alpha-1 antitrypsin deficiency related
Disease
Disease Phase
Phase Change
Change Berger's disease;
Cancer, liver; PhasePhase II Clinical
I Clinical Trial Trial
New Disease
Global Status Advance Cancer,I Clinical
Phase basal cell
Trial
IND Filing
Global Status Advance The
PhaseUS;II Cardiovascular
Clinical Trial disease, unspecified; Alimentary/metabolic
New Licensee
Development Restarted Roche; Worldwide
Development
New ApprovalSuspended Phase III Clinical
Israel, Japan; Trial for the treatment of AHP in adolescents and
Porphyria;
New Approval
Global Status Advance adults aged
Phase 12yrTrial
I Clinical and older
Development
First ApprovalRestarted Phase I Clinical Trial
The EU; Atherosclerosis; Hypercholesterolaemia; Heterozygous familial
New
GlobalFiling
Status Advance hypercholesterolaemia;
Phase I Clinical Trial as an adjunct to diet in combination with a statin
New Disease
Disease Phase Change Cancer,
Infection,solid, unspecified
hepatitis-D virus; Phase II Clinical Trial
New Disease
New Approval Infection,
Brazil; Hyperoxaluria virus
hepatitis-D
New
New Launch
Target Germany; Hyperoxaluria
angiopoietin-like 3 (30924)
New Mechanism
Global Status Advance Angiopoietin-like 3 inhibitor
Phase I Clinical Trial
Drug Added
Disease Phase Change Preclinical
Non-alcoholic steatohepatitis; No development reported
Disease
New Phase Change
Licensee Fibrosis, pulmonary,
Alnylam; Worldwide idiopathic; Phase II Clinical Trial
Global Status Advance
Nonproprietary Name Granted Phase III Clinical Trial
AD-01765
Expedited Review
Disease Phase Designation Granted The
Change US; Cardiovascular
Fibrosis, disease,
pulmonary, idiopathic; unspecified;
Macular Fast track
degeneration, age-related,
Global Status
New Approval Advance unspecified; Fibrosis, retinal; No development reported
Taiwan; Amyloidosis, transthyretin-related hereditary
New Launch
Drug Added Bulgaria,
Phase Denmark;
I Clinical TrialAmyloidosis, transthyretin-related hereditary
Development Restarted Phase II/III Clinical Trial
New Licensee
Global Status Advance Suzhou Ribo LifeTrial
Phase I Clinical Science; China (including Hong Kong, Macao, Taiwan)
Lead Identified
New Target retinitis therapy, Sylentis
BMI1 proto-oncogene, polycomb ring finger
New Patent
Global Status Advance US20170283803
Phase I Clinical Trial
Orphan Drug Status
Global Status Granted
Advance The
PhaseUS; Beta-thalassaemia;
I Clinical Trial adults
New
Lead Target
Identified Lipoprotein, Lp(a); 4018AccuRna
PRDM14 siRNA/uPIC,
Global Status Restarted
Development Advance Phase I Clinical Trial
No Development
Nonproprietary Reported
Name Granted Preclinical
QPI-1002
New Disease
Nonproprietary Name Granted Acute
SYL-1001renal failure; Phase III Clinical Trial
Global
ChangeStatus Reversion
in Licensee Status Phase II
Brii Biosciences; Phase II Clinical Trial
New Licensee
Nonproprietary Name Granted China;
ALN-AGT Brii Biosciences
Global Status Advance Phase II Clinical Trial
Therapeutic Class Therapeutic Class Status
RNA interference Phase II Clinical Trial
Antiviral, other
RNA interference Phase
Phase II ClinicalTrial
I Clinical Trial
Hepatoprotective
RNA interference Phase
Phase IIIClinical
ClinicalTrial
Trial
Antianaemic
RNA interference Phase II Clinical Trial
Phase I Clinical Trial
Hepatoprotective
Gene therapy Phase I Clinical Trial
Phase II Clinical Trial
RNA
RNA interference
interference Phase
Phase II ClinicalTrial
I Clinical Trial
Anticancer, immunological
RNA interference Phase
Phase II Clinical
Clinical Trial
Trial
Anticancer, other
RNA interference Phase
Phase IIIIClinical
ClinicalTrial
Trial
Hepatoprotective
RNA interference Phase III ClinicalTrial
Phase II Clinical Trial
Hypolipaemic/Antiatherosclerotic
RNA interference Phase
Phase II
II Clinical
Clinical Trial
Trial
Hypolipaemic/Antiatherosclerotic
RNA interference Phase
Phase II Clinical Trial
II Clinical Trial
Hepatoprotective
RNA interference Phase
Phase II Clinical Trial
II Clinical Trial
Ophthalmological,
RNA interference antiglaucoma Phase
Phase II
II Clinical
Clinical Trial
Trial
Hepatoprotective
RNA interference Phase
Phase II Clinical Trial
II Clinical Trial
Antianaemic
RNA interference Phase
Phase II Clinical Trial
II Clinical Trial
Anticancer,
RNA other
interference Phase IIIClinical
Phase ClinicalTrial
Trial
Metabolic and enzyme disorders
RNA interference Phase
Phase IIIClinical
ClinicalTrial
Trial
Antiviral, other
RNA interference Phase
Phase III ClinicalTrial
II Clinical Trial
Haemostatic
RNA interference Phase III
Launched Clinical Trial
Neurological
RNA interference Launched
Phase I Clinical Trial
Antiviral, other
RNA interference Phase I Clinical Trial
Registered
Hypolipaemic/Antiatherosclerotic
RNA interference Registered
Phase I Clinical Trial
Anticancer, other
RNA interference Phase
Phase IIIClinical
ClinicalTrial
Trial
Antiviral, other
RNA interference Phase II
Launched Clinical Trial
Metabolic and enzyme disorders
RNA interference Launched
Phase I Clinical Trial
Hypolipaemic/Antiatherosclerotic
RNA interference Phase
Phase II Clinical
Clinical Trial
Trial
Anticancer, other
RNA interference Phase
Phase II ClinicalTrial
I Clinical Trial
Hepatoprotective
RNA interference Phase III
Phase II Clinical
ClinicalTrial
Trial
Metabolic and enzyme disorders
RNA interference Phase
Phase III ClinicalTrial
II Clinical Trial
Cardiovascular
RNA interference Phase
Phase II
II Clinical
Clinical Trial
Trial
Vulnerary
RNA interference Phase II
Launched Clinical Trial
Neurological
RNA interference Launched
Phase I Clinical Trial
Anticancer, other
RNA interference Phase
Phase IIIIClinical
ClinicalTrial
Trial
Ophthalmological,
RNA interference other Phase III Clinical Trial
Phase I Clinical Trial
Ophthalmological, other
Reformulation, implant Phase
Phase IIIClinical
ClinicalTrial
Trial
RNA interference
RNA interference Phase IIIClinical
Phase ClinicalTrial
Trial
Antianaemic
RNA interference Phase I Clinical Trial
Phase I Clinical Trial
Cardiovascular
RNA interference Phase
Phase II Clinical
Clinical Trial
Trial
Anticancer, other
RNA interference Phase
Phase I Clinical Trial
I Clinical Trial
Anticancer,
RNA immunological
interference Phase III
Phase I Clinical
ClinicalTrial
Trial
Immunosuppressant
RNA interference Phase
Phase III Clinical Trial
III Clinical Trial
Ophthalmological,
RNA interference other Phase III Clinical Trial
Phase II Clinical Trial
Antiviral, other
RNA interference Phase
Phase II
II Clinical
Clinical Trial
Trial
Antihypertensive, other Phase II Clinical Trial
Mechanism Of Action Delivery Route Delivery Medium
Gene expression inhibitor Injectable; Injectable,
Hydroxysteroid 17-beta dehydrogenase 13 subcutaneous
Injectable; Injectable,
inhibitor; Gene expression
Gene expression inhibitor inhibitor subcutaneous
Unspecified
Gene expression inhibitor Injectable; Injectable,
Gene expression inhibitor subcutaneous
Injectable
Ubiquitin-specific protease inhibitor; Injectable
Immuno-oncology therapy;
Hypoxia-inducible factor RNA binder;
2 alpha Injectable; Injectable,
antagonist; Gene expression
Alpha 1 antitrypsin inhibitor
inhibitor; Gene intravenous
Injectable; Injectable, Solution
expression inhibitor
Angiopoietin-like 3 inhibitor; Gene subcutaneous
Injectable; Injectable,
expression inhibitor
Apolipoprotein C3 inhibitor; Gene subcutaneous
Injectable; Injectable,
expression inhibitor
Hydroxysteroid 17-beta dehydrogenase 13 subcutaneous
Injectable; Injectable,
inhibitor
Beta 2 adrenoreceptor antagonist; Gene subcutaneous
Ophthalmological Solution
expression inhibitor
Gene expression inhibitor Injectable; Injectable,
C5a inhibitor subcutaneous
Injectable; Injectable,
Gene expression inhibitor subcutaneous
Injectable; Injectable,
Gene expression inhibitor intradermal;Injectable,
Injectable; Injectable,
Gene expression inhibitor subcutaneous
Injectable; Injectable,
Gene expression inhibitor subcutaneous
Injectable; Injectable, Solution
Gene expression inhibitor; Aminolevulinate subcutaneous
Injectable; Injectable,
synthase 1 inhibitor
Gene expression inhibitor subcutaneous
Injectable; Injectable,
PCSK9 inhibitor; Gene expression inhibitor subcutaneous
Injectable; Injectable,
Gene expression inhibitor; Forkhead box subcutaneous
Injectable; Injectable,
P3
Viralinhibitor; Lymphocyte-activation
RNA polymerase inhibitor; HBVgene 3 intravenous
Injectable; Injectable, Solution
antigen release inhibitor;
Gene expression inhibitor;HBV
HAO1capsid
inhibitor subcutaneous
Injectable; Injectable, Solution
Gene expression inhibitor; Angiopoietin- subcutaneous
Injectable; Injectable,
like 3 inhibitor
Glutathione S transferase inhibitor; Gene subcutaneous
Injectable; Injectable,
expression inhibitor
Heat shock protein 47 antagonist; Gene intravenous
Injectable; Injectable, Solution
expression
Lactate inhibitor
dehydrogenase A inhibitor; Gene intravenous
Injectable; Injectable, Solution
expression inhibitor
Apolipoprotein A inhibitor; Gene subcutaneous
Injectable; Injectable,
expression inhibitor
Gene expression inhibitor; Connective subcutaneous
Injectable; Injectable,
tissue growth factor
Transthyretin inhibitor;antagonist;
Gene expression intradermal; Injectable,
Injectable; Injectable, Solution
inhibitor
Unidentified pharmacological activity intravenous
Injectable; Injectable,
Gene expression inhibitor; Caspase 2 intravenous
Injectable; Injectable,
inhibitor
Gene expression inhibitor intravitreal
Ophthalmological
K-Ras inhibitor Injectable; Injectable, Pellet
Gene expression inhibitor; intratumoralInjectable,
Injectable;
Transmembrane
Gene expression protease,
inhibitor serine 6 subcutaneous
Injectable; Injectable,
Gene expression inhibitor subcutaneous
Unspecified
Stathmin antagonist; Gene expression Injectable; Injectable,
inhibitor
p53 inhibitor intratumoral
Injectable; Injectable, Solution
Vanilloid receptor 1 antagonist; Gene intravenous
Ophthalmological Solution
expression inhibitor
Gene expression inhibitor Injectable; Injectable,
Gene expression inhibitor; Renin inhibitor subcutaneous
Injectable; Injectable,
subcutaneous
Delivery Technology Target Target Entrez
Liposomes; Nanoparticles Unspecified Gene ID
Not Applicable
hydroxysteroid 17-beta dehydrogenase 345275
13
Unspecified Not Applicable
patatin like phospholipase domain 80339
containing
Unspecified3 Not Applicable
Cbl proto-oncogene B 868
endothelial PAS domain protein 1 2034
serpin family A member 1 5265
angiopoietin like 3 27329
apolipoprotein C3 345
hydroxysteroid 17-beta dehydrogenase 345275
13
adrenoceptor beta 2 154
serpin family A member 1 5265
complement C5 727
Nanoparticles transforming growth factor beta 1 7040
prostaglandin-endoperoxide
lipoprotein(a) synthase 5743
4018
Unspecified Not Applicable
serpin family C member 1 462
5'-aminolevulinate synthase 1 211
large S protein, hepatitis-B virus 944569
proprotein convertase subtilisin/kexin 255738
type 9 box P3
forkhead 50943
lymphocyte
polymerase,activating
hepatitis-B 3 virus 3902
944565
precore/core protein, 1hepatitis-B virus
hydroxyacid oxidase 944568
54363
angiopoietin like 3 27329
Nanoparticles glutathione S-transferase pi 1 2950
Nanoparticles serpin family H member 1 871
lactate dehydrogenase A 3939
lipoprotein(a) 4018
cellular communication network factor 1490
Liposomes; Nanoparticles 2
transthyretin 7276
EWS RNA binding protein 1 2130
Fli-1 proto-oncogene,
caspase 2 ETS transcription 2313
835
Unspecified Not Applicable
Depot KRAS proto-oncogene, GTPase 3845
BMI1 proto-oncogene,
transmembrane serine polycomb
protease 6ring 648164656
lipoprotein(a) 4018
PR/SET domain 14 63978
stathmin 1 3925
tumor protein p53 7157
transient receptor potential cation 7442
channel subfamily V member 1
Unspecified Not Applicable
angiotensinogen 183
Target Family Target EC Number Trialtrove Trial Count
7
Enzyme > Dehydrogenase 3
1
Enzyme > Lipase 1
2
Enzyme 3
1
2
2
5
Enzyme > Dehydrogenase 1
Receptor > GPCR > Adrenoceptor 4
2
Cytokine/Growth factor > Cytokine > 10
Chemokine
Cytokine/Growth factor > Cytokine > 8
Growth factor 1.14.99.1 1
2
7
Enzyme > Synthase 2.3.1.37 5
2
Enzyme > Protease/peptidase 20
1
Enzyme > Polymerase 12
1.1.3.15 6
1
Enzyme > Transferase 2.5.1.18 1
9
Enzyme > Dehydrogenase 1.1.1.27 7
4
Cytokine/Growth factor > Growth 4
factor, non-cytokine 7
0
Enzyme > Protease/peptidase 3.4.22.55 2
1
2
Enzyme > Protease/peptidase 3
1
1
1
6
Ion channel > Transient receptor 5
potential channel 9
Peptide hormone 3
Origin NCE Molecular Weight
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid, viral No
vector
Biological, cellular, autologous No
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid No
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid, non- No
viral vectorsynthetic, nucleic
Chemical, No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid, non- No
viral vectornucleic acid, non-
Biological, No
viral vectorsynthetic, nucleic
Chemical, No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid No
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid, non- No
viral vectorsynthetic, nucleic
Chemical, No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid, non- No
viral vectorsynthetic, nucleic
Chemical, No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Biological, nucleic acid No
Biological, nucleic acid, non- No
viral vectorsynthetic, nucleic
Chemical, No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical, synthetic, nucleic No
acid
Chemical Name

small interfering RNA (siRNA) inhibiting antithrombin liver

production: duplex of [(2S,4R)-1-{30-(2-acetamido-2-deoxy-
Givosiran [INN]

Olpasiran [INN]

small interfering ARN (siRNA); RNA duplex of guanylyl-(3'?

5')-2'-O-methyluridylyl-(3'?5')-adenylyl-(3'?5')-adenylyl-(3'?

Rna, (g-am-g-am-a-um-a-um-u-um-c-am-c-cm-c-um-u-cm-
a), complex with rna (um-g-am-a-gm-g-gm-u-gm-a-am-a-um-
Chemical structure (SMILES format) Molecular Formula
CAS Number LogP H Bond Donors H Bond Acceptors

1499251-18-1
1639325-43-1

2225856-03-9

1420706-45-1

1231737-88-4
Rotatable Bonds Patent Number Patent Priority
Country & Date

US8168775 US 2008/10/20

US20170283803 US 2014/07/14
Marketing

Orphan Drug Status

________________
Orphan Drug Status
________________

Orphan Drug Designation

________________
Orphan drug status
________________
Orphan Drug Status
________________

Orphan Drug Status

________________
Orphan Drug Status
________________
Orphan Drug Status
________________
Filings
________________
Approvals
________________
Approvals
________________
Approvals
________________

Approvals
________________

Approvals
________________
Approval
_________________
Expedited Review Designation
_________________
Approvals
________________
Approvals
________________
Orphan Drug Status
________________
Orphan Drug Status
________________
Orphan Drug Status
________________

Orphan Drug Status

________________
Filings
_______________
Licensing
Agreements
________________
Agreements
________________
Agreements
________________
Availability
________________
Agreements
________________
Agreements
________________

Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreement
__________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
________________
Agreements
_______________

Agreements
________________________
Agreements
________________
Agreements
________________
Phase III

Hepatic dysfunction; Non-alcoholic steatohepatitis

A Phase
Ocular II trial isunspecified
disease, expected in the 2nd half of 2022 (R&D Day
A Phase III trial is expected in late 2024 (R&D Day

Hepatic dysfunction, alpha-1 antitrypsin deficiency related

A
A randomized,
pivotal Phaseplacebo-controlled Phase
III trial was expected III (AROAAT3001)
in 2020 (Company is
presentation, Arrowhead
Lipoprotein lipase Pharmaceuticals, 18 Oct 2019, page
deficiency
A Phase III clinical study in patients with familial

Paroxysmal nocturnal haemoglobinuria

A Phase III trial in combination with pozelimab is expected in

Haemophilia A; Haemophilia B
Alnylam;
PorphyriaSanofi
A randomized, double-blind, placebo-controlled Phase III
Atherosclerosis
Novartis

Hyperoxaluria
A randomized, double-blind, placebo-controlled, parallel

Hyperoxaluria
It is in an open-label roll-over extension Phase III trial (2018-

Amyloidosis, transthyretin-related hereditary

Alnylam
Neuropathy, ischaemic optic
Quark Pharmaceuticals; Biocon

Acute renal failure

Quark
Dry eyePharmaceuticals
syndrome
It
Infection,randomized,
is in a double-blind, placebo-controlled,
hepatitis-B virus
Phase III trials are planned (Form S-1, Vir Biotechnology, 3
Phase II
An authorization has been received from the US FDA to
proceed with its IIND Non-alcoholic
Hepatic dysfunction; application in 40 stably NA-suppressed,
steatohepatitis
A proof-of-concept
Ocular Phase II trial is expected in 2022 (R&D
disease, unspecified
A Phase II trial is expected in mid-2023 (R&D Day
Huntington's disease
A CTA has
Cancer, been filed in the UK for an open-label Phase Ib/II
colorectal
Phase II trial was planned (Company presentation, Apeiron
Hepatic dysfunction, alpha-1 antitrypsin deficiency related
Arrowhead
Hyperlipidaemia,Pharmaceuticals
unspecified; Hypertriglyceridaemia
It
Hypertriglyceridaemia;sequential
is in a randomized, assignment,
Lipoprotein quadruple single
lipase deficiency
A first-in-human,
Non-alcoholic single and multiple dose-escalating Phase
steatohepatitis
It is in a randomized, dose escalation, sequential assignment
Glaucoma
It is in Phase
Hepatic II trials (Company
dysfunction, pipeline,deficiency
alpha-1 antitrypsin Sylentis, 1related
Mar 2017
It is in a randomized,
Berger's disease placebo-controlled, double-blind,
It is in a randomized,
Cancer, basal cell double-blind, placebo-controlled,
Sirnaomics
Infection, hepatitis-B virus
Dicerna;
HaemophiliaRoche
A; Haemophilia B
Alnylam;
Porphyria Sanofi
It is in an open-label extension Phase I/II trial (ALN-AS1-002)
Heterozygous familial hypercholesterolaemia;
Hypercholesterolemia
Infection, hepatitis-B virus
Janssen
Hyperoxaluria
A Phase II trial in oxalate nephrolithiasis patients is expected

It is in Phase II trials (Company pipeline, BMS, 31 Jan 2019,

https://www.bms.com/researchers-and-partners/in-the-
Hyperoxaluria
Dicerna
Cardiovascular disease, unspecified
It is in ascarring
Keloid randomized, double-blind, parallel-assignment,
OliX Pharmaceuticals
Amyloidosis, transthyretin-related hereditary
An open-label extension, single-group-assignment Phase II
Glaucoma
Quark Pharmaceuticals
Cancer, pancreatic
It is in an open-label, single-arm, randomized, parallel-

Acute renal failure

Quark Pharmaceuticals
Pain, nociceptive, general; Dry eye syndrome
A randomized, parallel-assignment,
Infection, hepatitis-B virus double-blind, pilot,
Alnylam; Vir Biotechnology
Hypertension, unspecified
A Phase II trial (KARDIA-2) in 800 patients with primary
Phase I
Arbutus Biopharma
Infection,
Non-alcoholic hepatitis-B virus
steatohepatitis
It
AnisIND
in a filing
randomized, double-blind,
is expected in early 2022,placebo-controlled, 2-part,
with Phase I trial is
expected in mid-2022 (R&D
Non-alcoholic steatohepatitis Day presentation, Alnylam, 22
It
AnisIND
in a filing
randomized, double-blind,
for a Phase placebo-controlled,
I trial for the treatment of single-
Huntington’s
Cancer, solid,disease has been submitted, with intiation was
unspecified
A US open-label,
Cancer, renal single group assignment, dose-ranging
An IND application
A randomized, for an open-label
double-blind, parallel, has been
single andsubmitted
multiple- to
ascending dose, Phase I trial (AROAAT1001) in 45 healthy

In a Phase Ib trial, bamosiran eye drops were well tolerated

and resulted locally
A randomized, and systemically
double-blind, safe after single
placebo-controlled, Phase and
I trial
to evaluate the safety, tolerability, pharmacokinetics
It is in an open-label, non-randomized, ascending-dose, and
parallel-assignment
A CFDA approval forPhase I trial
a 2-part, (R3918-HV-1982;
randomized, 2020-
pharmacokinetics,
pharmacodynamics,
Eli Lilly double-blind, placebo-controlled Phase I
It
Theis in a randomized,
approval of CTA double-blind,
in Hong Kong,single-ascending
South Korea, anddose
Thailand
Haemophilia has A;
been filed (FormB10-K, Dicerna, 13 Mar 2019,
Haemophilia
Alnylam;
PorphyriaSanofi
A randomized,
Infection, parallel
hepatitis-B assignment, single-ascending dose,
virus
It is in a randomized,
The Medicines Company double-blind, placebo-controlled, single
An open-label,
Cancer, parallel-group, single-dose Phase I study
solid, unspecified
It is in an open-label, multiple ascending doses, first-in-
Janssen
An open-label, randomized, parallel-assignment, single-dose
Hyperlipidaemia, unspecified
It is in a randomized,
Cancer, double,lung,
colorectal; Cancer, single-ascending
non-small cell;and repeat-
Cancer,
pancreatic
Bristol-Myers Squibb
It is in an open-label, non-randomized, 2-part, single-dose,
Hyperoxaluria
Dicerna
Cardiovascular disease, unspecified
Amgen
A randomized, single-blind, parallel Phase I trial (HG-BMT-PI-
01) in 32 healthy
A randomized, male subjects
double-blind, in S. Korea, to evaluate
placebo-controlled, single- the
ascending-dose,
Cancer, sarcoma,parallel-assignment
Ewing's Phase I trial (ALN-
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trialUS
is complete
It is and well-tolerated
in a randomized, (Press release,
observer-masked, Quark, 19 Nov
parallel-group,
sequential-assignment Phase I trial (SYL1801_I) in Spain in
It is in a randomized, double-blind, placebo-controlled,
parallel-assignment,
Cardiovascular disease, single-ascending
unspecified dose Phase I trial
It is in a randomized,
Cancer, breast double-blind, placebo-controlled, first-in-
It is in ansolid,
Cancer, open-label, investigator-initiated, first-in-human,
unspecified
An open-label,
Acute single-group-assignment, dose-escalation
Renal failure
Silence Therapeutics
A single-blind, Phase I study (SYL1001_I) to evaluate the
ocular
Alnylam; tolerance of SYL-1001 topical eye drops in 30 healthy
Vir Biotechnology
In 6 single ascending
Hypertension, dose cohorts of 8 healthy volunteers,
unspecified
It is in a randomized, double-blind, placebo-controlled, single-
Preclinical Record URL
In vivo https://citeline.informa.com/drugs/details/142511?qId=dc1d03bc
In preclinical models, AB-729 exhibited potent and durable
It was at the lead optimization stage (Company presentation, https://citeline.informa.com/drugs/details/150947?qId=dc1d03bc
Alnylam, 6 Dec
Development 2018, Slide
candidate 212, https://www.alnylam.com/wp-
selection was expected in early 2021 https://citeline.informa.com/drugs/details/168355?qId=dc1d03bc
(R&D Day presentation, Alnylam, 22 Nov 2019, Slide 183, https://citeline.informa.com/drugs/details/197840?qId=dc1d03bc
In preclinical study, pre-existing serum antibodies to AAV5 https://citeline.informa.com/drugs/details/71131?qId=dc1d03bc-4
neutralizing antibodies (NABs) are not found in the https://citeline.informa.com/drugs/details/71748?qId=dc1d03bc-4
GLP toxicology studies are complete (Press release, https://citeline.informa.com/drugs/details/117164?qId=dc1d03bc
Arrowhead
In vivo Pharmaceuticals, 18 Oct 2019, https://citeline.informa.com/drugs/details/134469?qId=dc1d03bc
In PiZ
In vivo mouse model, ARC-AAT has deeply reduced https://citeline.informa.com/drugs/details/144959?qId=dc1d03bc
In
In LDL
vivo receptor knockout mice, ARO-ANG3 reduced https://citeline.informa.com/drugs/details/144958?qId=dc1d03bc
In ApoC3
In-life transgenic
phase of the GLP mice, a single studies
toxicology 2mg/kgisdose of ARO-
complete (Press https://citeline.informa.com/drugs/details/170030?qId=dc1d03bc
release, Arrowhead Pharmaceuticals, 18 Oct 2019,
Topically administered SYL-040012 was effective against https://citeline.informa.com/drugs/details/41612?qId=dc1d03bc-4
ocular
In vivo hypertension associated with OAG (Press release, https://citeline.informa.com/drugs/details/128189?qId=dc1d03bc
In
In non-human
vivo primates, single dose studies showed that 63% https://citeline.informa.com/drugs/details/82653?qId=dc1d03bc-4
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preclinical Heymann
studies, STP-705nephritis model an
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increase of https://citeline.informa.com/drugs/details/40196?qId=dc1d03bc-4
active
It T cell
was in infiltration
preclinical into the tumour
development microenvironment
(Company pipeline, 27 Dec https://citeline.informa.com/drugs/details/161518?qId=dc1d03bc
2018,
In vivohttps://dicerna.com/wp- https://citeline.informa.com/drugs/details/131679?qId=dc1d03bc
In
In non-human
vivo primates, the non-optimized GalXC molecule https://citeline.informa.com/drugs/details/73087?qId=dc1d03bc-4
Chronic GLP
Preclinical toxicology
results showedstudies
that itof fitusiran, including
completely a 9mth
blocked the https://citeline.informa.com/drugs/details/80245?qId=dc1d03bc-4
production
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toxic haeme stage
biosynthesis
(Company intermediates that
pipeline, Suzhou https://citeline.informa.com/drugs/details/82624?qId=dc1d03bc-4
Ribo, Jul 2016,
Addtional preclinical studies are planned (Press release, https://citeline.informa.com/drugs/details/119878?qId=dc1d03bc
Alnylam,
In vivo 11 Jan 2016, https://citeline.informa.com/drugs/details/135209?qId=dc1d03bc
In tumor-bearing
It was mouse
at the pre-IND model,
stage INT-1B3,
(Company demonstrated
pipeline, Arrowhead https://citeline.informa.com/drugs/details/134467?qId=dc1d03bc
Pharmaceuticals,
In vivo 15 Sep 2017, https://citeline.informa.com/drugs/details/90855?qId=dc1d03bc-4
In wild-type
It was at theanimals,
researchHAO1
stage silencing
(Company resulted in dose-
pipeline, 27 Dec 2018, https://citeline.informa.com/drugs/details/161517?qId=dc1d03bc
https://dicerna.com/wp-content/uploads/2018/11/Dicerna-
In vivo https://citeline.informa.com/drugs/details/160766?qId=dc1d03bc
In animal
In vivo model studies, NBF-006 demonstrated significant https://citeline.informa.com/drugs/details/67955?qId=dc1d03bc-4
Preliminary
In vivo proof-of-concept and a feasibility study confirming https://citeline.informa.com/drugs/details/113336?qId=dc1d03bc
In
In animal
vivo models, DCR-PHXC inhibits the lactate https://citeline.informa.com/drugs/details/118311?qId=dc1d03bc
In SAR studies in Tg mice, assessed structure and chemical https://citeline.informa.com/drugs/details/91919?qId=dc1d03bc-4
In vivo https://citeline.informa.com/drugs/details/64853?qId=dc1d03bc-4
A non-human
In vivo primate study in 6 doses was ongoing. It https://citeline.informa.com/drugs/details/133971?qId=dc1d03bc
In preclinical
In vivo studies, pbi-shRNA EWS/FLI1 type 1 LPX https://citeline.informa.com/drugs/details/42022?qId=dc1d03bc-4
It
It was in toxicology
is in preclinical studies for acute
development glaucoma
(Company to enable
pipeline, Sylentis, 6 https://citeline.informa.com/drugs/details/128349?qId=dc1d03bc
Jul 2016 & 5 Mar 2018,
In pre-clinical studies, siG12D showed that programmed cell https://citeline.informa.com/drugs/details/71740?qId=dc1d03bc-4
death
In vivo(apoptosis) effectively as well as halting of tumour https://citeline.informa.com/drugs/details/143816?qId=dc1d03bc
In ß-Thalassemic
A pre-IND meetingmice, SLN124
has been ameliorated
completed therelease,
(Press https://citeline.informa.com/drugs/details/143817?qId=dc1d03bc
Silence
In murineTherapeutics, 7 Jan 2020,
models, treatment https://www.silence-
with PRDM14-specific chimera https://citeline.informa.com/drugs/details/160122?qId=dc1d03bc
siRNA
In vivo mixed with a PIC nanocarrier caused 50.3% reduction https://citeline.informa.com/drugs/details/41131?qId=dc1d03bc-4
In previous
A an orthotopic pancreatic cancer
lead compound, mouse
PFT-101B, wasmodel,
undercompouds https://citeline.informa.com/drugs/details/30445?qId=dc1d03bc-4
investigation for the same indication (Direct communication,
In preclinical studies SYL1001 has showed its high ability to https://citeline.informa.com/drugs/details/66785?qId=dc1d03bc-4
inhibit
In vivoTRPV1 receptor and block the perception of ocular https://citeline.informa.com/drugs/details/146103?qId=dc1d03bc
In
In an
vivoadeno-associated virus-HBV mouse model, VIR-2218 in https://citeline.informa.com/drugs/details/90304?qId=dc1d03bc-4
In a transgenic rat model of preeclampsia, sc administration
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gs/details/73087?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
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gs/details/119878?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/135209?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/134467?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/90855?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/161517?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/160766?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/67955?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
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gs/details/64853?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/133971?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/42022?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/128349?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/71740?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/143816?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
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gs/details/160122?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/41131?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/30445?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
gs/details/66785?qId=dc1d03bc-4a38-4238-98e8-88bfffbebe1f
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(Therapeutic Class is RNA interference) AND [(Most Advanced Current Status is
Launched) OR (Most Advanced Current Status is Phase III Clinical Trial) OR (Most
Advanced Current Status is Phase II Clinical Trial) OR (Most Advanced Current
Status is Phase I Clinical Trial) OR (Most Advanced Current Status is Registered)]

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