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Genetix Biotherapeutics Inc. — Rare Disease Pipeline | Kisho | Kisho

Genetix Biotherapeutics Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
B-cell chronic lymphocytic leukemia	Zynteglo	Orphan Designation	-
B-cell neoplasm	Zynteglo	Orphan Designation	-
B-lymphoblastic leukemia/lymphoma, BCR-ABL1–like	Zynteglo	Orphan Designation	-
Beemer-Langer syndrome	Lyfgenia	Orphan Designation	-
Coffin-Siris syndrome 1	Lyfgenia	Orphan Designation	-
Coffin-Siris syndrome 12	Lyfgenia	Orphan Designation	-
DK1-congenital disorder of glycosylation	Skysona	Orphan Designation	-
adrenoleukodystrophy	Skysona	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Zynteglo	Orphan Designation	-
apolipoprotein A-II amyloidosis	Lyfgenia	Orphan Designation	-
cardiofaciocutaneous syndrome 1	Skysona	Orphan Designation	-
developmental and epileptic encephalopathy, 2	Skysona	Orphan Designation	-
hemophilia A	Lyfgenia	Orphan Designation	-
heterotaxy, visceral, 12, autosomal	Lyfgenia	Orphan Designation	-
joint contractures, osteochondromas, and B-cell lymphoma	Zynteglo	Orphan Designation	-
mitochondrial DNA depletion syndrome 6 (hepatocerebral type)	Skysona	Orphan Designation	-
neurodegeneration, childhood-onset, with ataxia, tremor, optic atrophy, and cognitive decline	Skysona	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Zynteglo	Orphan Designation	-
pediatric hepatocellular carcinoma	Zynteglo	Orphan Designation	-
pediatric ovarian dysgerminoma	Zynteglo	Orphan Designation	-
pediatric-onset Graves disease	Zynteglo	Orphan Designation	-
sickle cell disease	Lyfgenia	Orphan Designation	-
tetrasomy 12p	Lyfgenia	Orphan Designation	-
triple-A syndrome	Lyfgenia	Orphan Designation	-