Kisho

Ionis Pharmaceuticals, Inc. — Rare Disease Pipeline | Kisho | Kisho

Ionis Pharmaceuticals, Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
ATTRV30M amyloidosis	Wainua	Orphan Designation	-
Angelman syndrome	2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA	Orphan Designation	-
HSD10 disease, neonatal type	2-O-(2-methoxyethyl)/DNA modified antisense drug that selectively targets MECP2 pre-mRNA	Orphan Designation	-
Lafora disease	2¿-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 (GYS1) pre mRNA	Orphan Designation	-
Pelizeaus-Merzbacher spectrum disorder	2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA	Orphan Designation	-
acquired polycythemia vera	Sapablursen	Orphan Designation	-
agenesis of the corpus callosum with peripheral neuropathy	Wainua	Orphan Designation	-
amyotrophic lateral sclerosis	Ulefnersenphosphorothioate oligonucleotide	Orphan Designation	-
beta-thalassemia HBB/LCRB	synthetic 2?-O-(2-methoxyethyl)-modified antisense oligonucleotide linked to a tri-antennary cluster of N-acetyl galactosamine (GalNAc3) sugars targeting transmembrane protease, serine 6 messenger ribonucleic acid	Orphan Designation	-
familial amyloid neuropathy	Wainua	Orphan Designation	-
familial chylomicronemia syndrome	Tryngolza	Orphan Designation	-
familial partial lipodystrophy, Dunnigan type	synthetic oligomer of 16 nucleotides	Orphan Designation	-
fetal methylmercury syndrome	2-O-(2-methoxyethyl)/DNA modified antisense drug that selectively targets MECP2 pre-mRNA	Orphan Designation	-
hereditary angioedema	Dawnzera	Orphan Designation	-
hereditary spastic paraplegia 11	Wainua	Orphan Designation	-
hereditary spastic paraplegia 61	Wainua	Orphan Designation	-
inborn disorder of methionine cycle and sulfur amino acid metabolism	2-O-(2-methoxyethyl)/DNA modified antisense drug that selectively targets MECP2 pre-mRNA	Orphan Designation	-
malignant carotid body paraganglioma	zilganersen	Orphan Designation	-
polyneuropathy-hand defect syndrome	Wainua	Orphan Designation	-
post-treatment Lyme disease syndrome	zilganersen	Orphan Designation	-
treatment-refractory schizophrenia	zilganersen	Orphan Designation	-

← Back to Companies