Sickle Cell

Rare Disease·MeSH: Anemia, Sickle Cell

Prevalence

30.0 / 100K

48 drugs in development·20 companies·1 clinical trial

Drug Pipeline by Phase

Approved(7)

Drug	Company
CASGEVY	CRISPR Therapeutics AG (CRSP)
CASGEVY	Editas Medicine, Inc. (EDIT)
CASGEVY (exagamglogene autotemcel)	Editas Medicine, Inc. (EDIT)
endari	Emmaus Life Sciences, Inc. (EMMA)
LYFGENIA	bluebird bio, Inc. (BLUE)
oxbryta	Global Blood Therapeutics, Inc. (GBT)
PYRUKYND®	AGIOS PHARMACEUTICALS, INC. (AGIO)

Phase 3(15)

Drug	Company
20vpcv	Sanofi (SNY)
20vpcv	Sanofi (SNY)
alemtuzumab	GH Research PLC (GHRS)
crizanlizumab	Novartis AG (NVS)
Crizanlizumab	Novartis AG (NVS)
ctx001	VERTEX PHARMACEUTICALS INC / MA (VRTX)
disc-3405	Disc Medicine, Inc. (IRON)
etavopivat	Novo Nordisk A/S (NVO)
etavopivat a	Novo Nordisk A/S (NVO)
etavopivat b	Novo Nordisk A/S (NVO)
etavopivat c	Novo Nordisk A/S (NVO)
exa-cel	VERTEX PHARMACEUTICALS INC / MA (VRTX)
inclacumab	Global Blood Therapeutics, Inc. (GBT)
pcv21	Sanofi (SNY)
pcv21	Sanofi (SNY)

Phase 2(5)

Drug	Company
abatacept	GH Research PLC (GHRS)
disc-0998	Disc Medicine, Inc. (IRON)
etavopivat	Forma Therapeutics Holdings, Inc. (FMTX)
hu - hydroxyurea	Novo Nordisk A/S (NVO)
pociredir	Fulcrum Therapeutics, Inc. (FULC)

Phase 1(10)

Drug	Company
BIVV003	SANGAMO THERAPEUTICS, INC (SGMO)
Busulfan	SANGAMO THERAPEUTICS, INC (SGMO)
digoxin	Novo Nordisk A/S (NVO)
gbt601	Global Blood Therapeutics, Inc. (GBT)
motixafortide	GH Research PLC (GHRS)
pitavastatin	Novo Nordisk A/S (NVO)
Plerixafor	SANGAMO THERAPEUTICS, INC (SGMO)
reni-cel	Editas Medicine, Inc. (EDIT)
renizgamglogene autogedtemcel (reni-cel)	Editas Medicine, Inc. (EDIT)
rosuvastatin	Novo Nordisk A/S (NVO)

Preclinical(3)

Drug	Company
In vivo HSC gene editing program for SCD and TDT	Editas Medicine, Inc. (EDIT)
sickle cell disease product	GreenLight Biosciences Holdings, PBC (GRNA)
vadc45	Vor Biopharma Inc. (VOR)

Phase 1/Phase 2(4)

Drug	Company
BEAM-101	Beam Therapeutics Inc. (BEAM)
itu512	Novartis AG (NVS)
itu512	NOVARTIS AG (NVSEF)
Ristoglogene autogetemcel (risto-cel)	Beam Therapeutics Inc. (BEAM)

Phase 2/Phase 3(1)

Drug	Company
ft-4202	Forma Therapeutics Holdings, Inc. (FMTX)

Phase 4(3)

Drug	Company
bms-986470	BRISTOL MYERS SQUIBB CO (BMY)
Mitapivat	AGIOS PHARMACEUTICALS, INC. (AGIO)
Tebapivat	AGIOS PHARMACEUTICALS, INC. (AGIO)

BioSniper tracks 48 drugs in development for Sickle Cell across 20 companies.

There are 1 clinical trial targeting Sickle Cell tracked by BioSniper.

The estimated prevalence of Sickle Cell is approximately 30.0 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.