Sickle Cell Disease

Rare Disease·MeSH: Anemia, Sickle Cell

Prevalence

10.0 / 100K

Incidence

3.0 / 100K/yr

49 drugs in development·19 companies·61 clinical trials

Drug Pipeline by Phase

Approved(7)

Drug	Company
CASGEVY	Editas Medicine, Inc. (EDIT)
CASGEVY	CRISPR Therapeutics AG (CRSP)
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(13)

Drug	Company
20vpcv	Sanofi (SNY)
20vpcv	Sanofi (SNY)
alemtuzumab	GH Research PLC (GHRS)
crizanlizumab	Novartis AG (NVS)
daratumumab	GH Research PLC (GHRS)
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)
filgrastim	GH Research PLC (GHRS)
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(12)

Drug	Company
BIVV003	SANGAMO THERAPEUTICS, INC (SGMO)
Busulfan	SANGAMO THERAPEUTICS, INC (SGMO)
digoxin	Novo Nordisk A/S (NVO)
disc-3405	Disc Medicine, Inc. (IRON)
etavopivat	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 (NVSEF)
itu512	Novartis AG (NVS)
Ristoglogene autogetemcel (risto-cel)	Beam Therapeutics Inc. (BEAM)

Phase 2/Phase 3(2)

Drug	Company
ctx001	VERTEX PHARMACEUTICALS INC / MA (VRTX)
ft-4202	Forma Therapeutics Holdings, Inc. (FMTX)

Phase 4(3)

Drug	Company
Crizanlizumab	Novartis AG (NVS)
Mitapivat	AGIOS PHARMACEUTICALS, INC. (AGIO)
Tebapivat	AGIOS PHARMACEUTICALS, INC. (AGIO)

BioSniper tracks 49 drugs in development for Sickle Cell Disease across 19 companies.

There are 61 clinical trials targeting Sickle Cell Disease tracked by BioSniper.

The estimated prevalence of Sickle Cell Disease is approximately 10.0 per 100,000 population.

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