Drug intelligence / Profile preview

TAK-754

Development stage: Phase 2
Lead developer: Takeda
Modality: Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

TAK-754 (also known as BAX 888 and SHP654) is an investigational gene therapy developed for the treatment of hemophilia A. It utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a codon-optimized, B-domain deleted factor VIII (FVIII) transgene specifically to the liver. The goal is for patients' livers to produce functional FVIII protein endogenously, thereby reducing or eliminating the need for exogenous FVIII infusions. The transgene cassette has been optimized by reducing CpG motifs to minimize immunogenicity and enhance durability of expression. Clinical trials have focused on safety, dose determination, and efficacy in adult males with severe hemophilia A[1][3][4][5][6].

Other names: FVIII gene therapy

Targets

F8 (Coagulation Factor VIIIa)

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