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Oral Decitabine/Tetrahydrouridine

Sickle Cell Disease

Phase 2Active

Key Facts

Indication

Sickle Cell Disease

Phase

Phase 2

Status

Active

Company

Novo Nordisk

About Novo Nordisk

Novo Nordisk's mission is to defeat diabetes and other serious chronic diseases through innovative biopharmaceutical solutions. The company has achieved transformative success with its GLP-1-based therapies, Ozempic® and Wegovy®, catalyzing a new era in metabolic care and generating massive commercial growth. Its strategy is built on expanding the clinical utility of its core platforms, advancing a deep pipeline of next-generation multi-agonists and novel modalities, and scaling manufacturing to meet global demand. Recent milestones include the US approval of the first once-weekly basal insulin, Awiqli®, and promising Phase 2 data for a triple agonist.

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Therapeutic Areas

Diabetes Obesity Rare Blood & Endocrine Disorders Cardiovascular Disease Chronic Kidney Disease

Other Sickle Cell Disease Drugs

Drug	Company	Phase	Status
Oxbryta® (voxelotor)	Cleo Life Sciences	Approved	Supported late-stage development & NDA
CT-101	Cetya Therapeutics	Pre-clinical	Active - Lead candidate nominated
In Vivo Cell Therapy	GigaMune	Pre-clinical	Active
Ramatroban (KAR101)	KARE Biosciences	Preclinical	Active
Endari (L-glutamine)	Emmaus Life Sciences	Approved	Active
Digital Model for Sickle Cell Disease	Koneksa Health	Development/Validation	Active
ILX-002	Illexcor Therapeutics	Pre-clinical	Active
Epeleuton	Afimmune	Phase 2	Active
Undisclosed	Hillhurst Biopharmaceuticals	Preclinical or Phase 1	Active
CLY-124	Cellarity	Phase 1	Active
AB1	AkiraBio	Pre-clinical	Active
Exagamglogene Autotemcel (CASGEVY)	Vertex Pharmaceuticals	Approved	Commercial