NHGRI | Technology Transfer

Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency

Read more about Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency

South Korea
10-2021-7007207
Filed on 2018-10-30

OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

Read more about OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

US Patent 11,802,283
Filed on 2020-09-17

SYNTHETIC GENES FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY
MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA

Read more about SYNTHETIC GENES FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA

US Application 17/620,331
Filed on 2021-12-17

SYNTHETIC GENES FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOLXYLASE ALPHA

Read more about SYNTHETIC GENES FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOLXYLASE ALPHA

European Patent
20743021.6
Filed on 2022-01-18

Novel Adeno-associated Viral (AAV) Vectors To Treat Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MMUT) Deficiency

Read more about Novel Adeno-associated Viral (AAV) Vectors To Treat Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MMUT) Deficiency

Patent Cooperation Treaty
PCT/US2021/050699
Filed on 2021-09-16

HIGH CONCENTRATION METHYLCOBALAMIN OR COMBINATION OF METHYL- AND HYDROXOCOBALAMIN FOR THE TREATMENT OF COBALAMIN C DEFICIENCY AND RELATED DISORDERS

Read more about HIGH CONCENTRATION METHYLCOBALAMIN OR COMBINATION OF METHYL- AND HYDROXOCOBALAMIN FOR THE TREATMENT OF COBALAMIN C DEFICIENCY AND RELATED DISORDERS

Patent Cooperation Treaty
PCT/US2021/054619
Filed on 2021-10-12

METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

Read more about METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

Japan
2021-526207
Filed on 2021-01-18

NON-DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA

Read more about NON-DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA

India
202117007847
Filed on 2018-10-30

METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

Read more about METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

India
202117002972
Filed on 2019-07-16

METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

Read more about METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS

European Patent
19749899.1
Filed on 2019-07-16

Subscribe to NHGRI