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BIVV003

Sickle Cell Disease

Phase 1/2Active

Key Facts

Indication

Sickle Cell Disease

Phase

Phase 1/2

Status

Active

Company

Sangamo Therapeutics

About Sangamo Therapeutics

Sangamo Therapeutics is a genomic medicine company with a mission to develop lasting, potentially curative therapies for severe diseases using its foundational zinc finger protein (ZFP) technology. The company has achieved clinical proof-of-concept across multiple modalities, most notably with its lead gene therapy candidate, isaralgagene civaparvovec (ST-920), for Fabry disease, for which it is preparing a Biologics License Application (BLA) submission. Its strategy combines advancing a wholly-owned neurology pipeline with leveraging its platform through strategic collaborations with major pharmaceutical firms like Pfizer, Novartis, Takeda, and Lilly to expand into other therapeutic areas and share development costs.

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Therapeutic Areas

Neurology Metabolic Hematology Immunology

Other Sickle Cell Disease Drugs

Drug	Company	Phase	Status
ANXV	Annexin Pharmaceuticals	Research	Identified
Oxbryta® (voxelotor)	Cleo Life Sciences	Approved	Supported late-stage development & NDA
CT-101	Cetya Therapeutics	Pre-clinical	Active - Lead candidate nominated
Endari (L-glutamine oral powder)	Emmaus Life Sciences	Approved	Commercial/Marketed
Undisclosed	Biossil	Phase 2/3	Active
In Vivo Cell Therapy	GigaMune	Pre-clinical	Active
Ramatroban (KAR101)	KARE Biosciences	Preclinical	Active
Digital Model for Sickle Cell Disease	Koneksa Health	Development/Validation	Active
ILX-002	Illexcor Therapeutics	Pre-clinical	Active
Epeleuton	Afimmune	Phase 2	Active
CLY-124	Cellarity	Phase 1	Active
AB1	AkiraBio	Pre-clinical	Active