Thalassemia

Rare Disease·MeSH: Thalassemia

Prevalence

1.5 / 100K

Incidence

0.6 / 100K/yr

23 drugs in development·13 companies·11 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)
deferasirox	Amneal Pharmaceuticals, Inc. (AMRX)
PYRUKYND®	AGIOS PHARMACEUTICALS, INC. (AGIO)
reblozyl	ACCELERON PHARMA INC (XLRN)
ZYNTEGLO	bluebird bio, Inc. (BLUE)

Phase 3(5)

Drug	Company
AG-348	AGIOS PHARMACEUTICALS, INC. (AGIO)
ctx001	VERTEX PHARMACEUTICALS INC / MA (VRTX)
etavopivat	Novo Nordisk A/S (NVO)
luspatercept	CELGENE CORP /DE/ (CELG)
Luspatercept	BRISTOL MYERS SQUIBB CO (BMY)

Phase 2(1)

Drug	Company
regn7999	REGENERON PHARMACEUTICALS, INC. (REGN)

Phase 1(7)

Drug	Company
digoxin	Novo Nordisk A/S (NVO)
edit-301	Editas Medicine, Inc. (EDIT)
edit-401	Editas Medicine, Inc. (EDIT)
pitavastatin	Novo Nordisk A/S (NVO)
reni-cel	Editas Medicine, Inc. (EDIT)
renizgamglogene autogedtemcel (reni-cel)	Editas Medicine, Inc. (EDIT)
rosuvastatin	Novo Nordisk A/S (NVO)

Preclinical(2)

Drug	Company
In vivo HSC gene editing program for SCD and TDT	Editas Medicine, Inc. (EDIT)
rlyb332	Rallybio Corp (RLYB)

Phase 1/Phase 2(1)

Drug	Company
st-400	Bioverativ Inc. (BIVV)

BioSniper tracks 23 drugs in development for Thalassemia across 13 companies.

There are 11 clinical trials targeting Thalassemia tracked by BioSniper.

The estimated prevalence of Thalassemia is approximately 1.5 per 100,000 population.

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