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Resamirigene bilparvovec

AT132 is under investigation in clinical trial NCT03199469 (Gene Transfer Clinical Study in X-linked Myotubular Myopathy).

Devafidugene civaparvovec

It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme.

Delandistrogene moxeparvovec

[L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein.

DCVax-L

DCVax-L is a personalized immunotherapy comprising an autologous vaccine with dendritic cells loaded _in vitro_ with autologous tumour lysate.

Etuvetidigene Autotemcel

[L54693] WAS is a rare X-linked disorder caused by a mutation in the gene encoding the WAS protein, leading to a compromised immune system.

CY-1503

1503 is a pentasaccharide ethyl glycoside consisting of one alpha-D-sialylosyl residue as a sodium salt, two beta-D-galactopyranosyl residues, one 2-acetamido-beta-Dglucopyranosyl unit, and one alpha-L-fucopyranosyl

SYNB1020

It was initially designed to convert ammonia to L-arginine [A258838] and granted orphan designation by the FDA for the treatment of urea cycle disorders in August 2016;[L45988] however, the manufacturer

Marstacimab

[A264608] Marstacimab is used to prevent bleeding episodes in patients with hemophilia,[L51803] which is an X‐linked bleeding disorder characterized by coagulation clotting factor deficiency.

Pralsetinib

Pralsetinib, similar to the previously approved selpercatinib, is a kinase inhibitor with enhanced specificity for RET tyrosine kinase receptors (RTKs) over other RTK classes. Enhanced RET (Rearranged during transfection) oncogene expres...

o-Cymen-5-ol