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HUMx_GATEWAY
THINKING REVOLUTIONARILY FOR THE SOLUTION

​Find the condition you would like to address

Harness the research advancements of in vivo lentiviral system to provide cures for a variety of genetic diseases through HUMx Gateway

HUMx_GATEWAY for IEMs

MUCOPOLYSACCHARIDOSIS_lysosomal-storage diseases

  • Hurler Syndrome (MPSI)_alfa-L-iduronidase

  • Hunter Syndrome (MPSII)_2-iduronate sulfatase

  • Sanfilippo Syndrome (MPSIII A)_N-sulfoglucosamine sulfhydrolase

  • Sanfilippo Syndrome (MPSIII B)_alfa-N-acetylglucosaminidase

  • Sanfilippo Syndrome (MPSIII C)_acetyl-CoA:alfa-glucosamine acetyltransferase

  • Sanfilippo Syndrome (MPSIII D)_N-acetylglucosamine 6-sulfatase

  • Morquio Syndrome (MPSIV A)_N-acetylgalactosaminide 6-sulfatase

  • Morquio Syndrome (MPSIV B)_beta-galactosidase-1

  • Maroteaux Lamy Syndrome (MPSVI)_N-acetylgalactosamine 4-sulfatase

  • Sly Syndrome (MPSVII)_beta-glucuronidase

  • Hyaluronidase Deficiency (MPSIX)_hyaluronidase-1

 

SPHINGOLIPIDOSES_lysosomal-storage diseases

  • Tay-Sachs Disease_beta-hexosaminidase A

  • Sandhoff Disease_beta-hexosaminidase B

  • Niemann-Pick Diseases A, B_acid sphingomyelinase

  • Niemann-Pick Diseases C_NPC1 or NPC2

  • Gaucher's Disease_beta-glucocerebrosidase

  • Fabry's Disease_alfa-galactosidase A

  • Metachromatic Leukodystrophy_arylsulfatase A

  • Krabbe's Disease/Globoid Leukodystrophy_galactocerebrosidase

  • GM1 Gangliosidosis_beta-galactosidase

  • GM2 Gangliosidosis_GM2 ganglioside activator

  • Austin Disease/Multiple Sulfatase Deficiency_multiple sulfatases

 

LIPOFUSCIN_lysosomal-storage diseases

  • Batten Disease_PPT1

  • Ceroid Lipofuscinosis 2_tripeptidyl peptidase

  • Ceroid Lipofuscinosis 3_lysosomal transmembrane protein

  • Parry Disease_CSPa

  • Ceroid Lipofuscinosis 5_CLN-5

  • Ceroid Lipofuscinosis 6_CLN-6

  • Ceroid Lipofuscinosis 7_lysosomal membrane protein

  • Ceroid Lipofuscinosis 8_CLN-8

  • Ceroid Lipofuscinosis 9_unkown

  • Ceroid Lipofuscinosis 10_cathepsin D

  • Ceroid Lipofuscinosis 11_granulin precursor

  • Ceroid Lipofuscinosis 12_cation-transporting ATPase 13A2

  • Kufs Disease Yype B_cathepsin F

  • Progressive Myoclonic Epilepsy_potassium channel tetramerization domain containing

 

MISCELLANEOUS

  • Alfa Mannosidosis_alfa-mannosidase

  • Beta-mannosidosis_beta-mannosidase

  • Schindler Disease_alfa-N-acetylgalactosaminidase

  • Aspartylglucosaminuria_aspartoglucosaminidase

  • Fucosidosis_alfa-l-fucosidase

  • Galactosialidosis_beta-galactosidase and neuraminidase

  • Sialic Acid Storage Disease_sialin

  • Mucolipidosis I_alfa-acetyl neuraminidase

  • I-cell Disease_GlcNAc-1-phosphotransferase​

  • Mucolipidosis IV_cation channel mucolipin 1

  • Pompe Disease_acid maltase or acid alpha-glucosidase 

  • Danon Disease_LAMP-2

  • Cystinosis_CTNS

  • X-linked Adrenoleukodystrophy_ABCD1

  • Action Myoclonus_LIMP-2

  • Cobalamin F-type Disease_LMBD-1

  • Angelman Syndrome_UBE3A

  • Farber Lipogranulomatosis_acid ceramidase

  • Wolman Disease_lysosomal acid lipase​

  • Phenylketonuria_PAH

One-time treatment, Life-long impact
Genetically corrected myeloid cells derived from in vivo lenti-engineered HSCs can migrate into the central nervous system, gradually replacing dysfunctional microglia and restoring function

—offering a durable and potentially curative solution.

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