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Reloxiliase

Reloxiliase is a first-in-class, oral crystalline formulation of the oxalate-specific, microbial enzyme oxalate decarboxylase. It is being investigated for the treatment of hyperoxaluria.[A258823]

ABX1100

ABX1100 is an investigational Centyrin-siRNA conjugate that targets the Gys1 gene, which encodes glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in the muscle. … It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease.[L45978]

RP-L301

RP-L301 is a lentiviral vector containing the human liver and erythroid pyruvate kinase gene.

rAAV9-rsATP7A

A recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.

SYNB1353

SYNB1353 is a strain of the probiotic bacteria _Escherichia coli_ (_E. coli_) type Nissle and an engineered probiotic drug. … [A258833] Developed by Synlogic Operating Company, Inc., SYNB1353 is being investigated for the treatment of homocystinuria.

SYNB1020

discontinued further development of SYNB1020 to treat hyperammonemia due to a lack of evidence of ammonia-lowering activities by SYNB1020 in a Phase 1b/2a study. … SYNB1020 is an engineered strain of _Escherichia coli_ Nissle 1917 developed by Synlogic Inc.

PRX-OTC

PRX-OTC is an intracellular enzyme replacement therapy designed to replace missing or defective enzyme in patients with ornithine transcarbamylase deficiency.

RGX-121

RGX-121 is designed to use the NAV AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system.

LB-001

LB-001 is a gene editing therapy designed to incorporate a functioning version of the faulty methylmalonyl-COA mutase (MUT) gene into the genome of patients with methylmalonic acidemia.

Zinc mesoporphyrin