Sickle Cell Disease (SCD) Approved Therapies & Global Pipeline

Last Update: Aug 5, 2023

Approved Therapies

Company Product MOA Indication Approval
NovartisAdakveo (crizanlizumab)Anti-P-selectin mAbAdd-on or monotherapy to reduce the frequency of VOCs in SCD patients aged 16 and older.2019 (US), 2020 (EU, revoked 2023), 2021 (UK)
Global Blood TherapeuticsOxbryta (voxelotor)HbS stabiliser/polymerisation inhibitorTreatment of SCD in adults and pediatric patients 4 years of age and older. Ages 12+ in UK.2019 (US), 2022 (EU), 2022 (UK)
Emmaus Life SciencesEndari (L-glutamine)Antioxidant/unclearReduce acute complications of SCD2017 (US)
MultipleHydroxyurea/ HydroxycarbamideIncrease HbF production, immunomodulationReduce painful crises and blood transfusions in pts w/ SCA w/recurrent mod-severe painful crises1998 (US)
-Allogeneic HSCTReplace beta globin-defective stem cellsSevere SCD - eligible patients-
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Global Pipeline, Disease-modifying Agents

Company Candidate MOA Indications Phase
Global Blood TherapeuticsInclacumabAnti P-selectin mAbSCD - reduce frequency of VOCsIII
Forma TherapeuticsEtavopivat (FT-4202)PKR activatorSCD - Hb+VOCsII/III
Agios PharmaMitapivatPKR activatorSCD, thalessemiasII/III
Global Blood TherapeuticsGBT601Next gen HbS polymerisation inhibitorSCD - disease modifyingII/III
EpiDestiny/Novo NordiskEPI01DNMT1 inhibitor / increase expression of HbFSCDII
Fulcrum TherapeuticsFTX-6058EED inhibitor / increase expression of HbFSCD Ib
Agios PharmaAG-946PKR activatorSCD, hemolytic anemiasI
CSLCSL889Plasma-derived hemopexinTreatment of VOC in SCDI
GlycomimeticsGMI-1687E-selectin antagonistSCD - reduce VOCIND-C

Gene therapies/Curative Therapies

Company Candidate MOA Indications Phase
Vertex/CRISPR TherapeuticsCTX001 (Exa-cel)Ex vivo autologous CRISPR/Cas9 BCL11A edited HSCSevere hemoglobinopathies (SCD) cureBLA
Bluebird bioLentiGlobin BB305 (bb1111)Ex vivo autologous HSC lentiviral therapy delivering functional HBBSCD cureIII, I/II
Sangamo/Bioverativ (Sanofi)BIVV-003ZFN-modified autologous stem cells w/ disrupted BCL11A geneSCD cure, B-thalessemiaI/II
Intellia TherapeuticsOTQ923Ex vivo HSC gene therapy to increase HbF expressionSevere SCDI/II
Editas MedicineEDIT-301Ex vivo HSC gene therapy to increase HbF expressionSevere SCDI/II
Aruvant SciencesRVT-1801Ex vivo autologous HSC lentiviral therapy delivering gamma-globin geneSCD cure, beta-thalessemiaI
Bluebird bioBCH-BB694shRNA miR lentiviral vector to knockdown BCL11A expressionSevere SCDI
CSL/CalimmuneCSL200 (CAL-H)Ex vivo HSC gene therapy delivering gamma-globin geneSCD cure, B-thalessemiaI
NovartisADPT03Ex vivo CRISPRSCD cureI
Beam TherapeuticsBEAM-101Ex vivo base-edited HSC cell therapy (corrected HbS)SCD, Beta ThalassemiaI/II
Prime Medicine -Ex vivo base-edited HSC cell therapy (corrected HbS)SCDPreclin

Discontinued Programs

Company Candidate MOA Indications Phase
Pfizer/GlycomimeticsRivipanselPan-selectin antagonistSCD - reduce VOCsIII
ImaraTovinontrine (IMR-687)PDE9 inhibitorSCDIIb
Modus TherapeuticsSevuparinPolysaccharide, anti-inflammatorySCD - reduce VOCsII
MerckMontelukastCysLT1 receptor inhibitorSCDII
Graphite BioGPH101Autologous HbA CRISPR-corrected HSCTSevere SCD cureI/II
Beam TherapeuticsBEAM-102Ex vivo base-edited HSC cell therapy (corrected HbS)SCDIND-enabling