NHGRI | Technology Transfer

IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA

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European Patent
22856962.0
Filed on 2024-06-27

SYNTHETIC MMAB GENES AND AAV VECTORS TO TREAT COBALAMIN B DEFICIENCY

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Patent Cooperation Treaty
PCT/US2024/023432
Filed on 2024-04-05

NON-DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA

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Japan
2024-000692
Filed on 2024-01-05

OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

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Australia
2023274161
Filed on 2023-11-30

OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

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Singapore
10202303471P
Filed on 2023-12-11

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

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US Application 18/032,132
Filed on 2023-04-14

NOVEL ADENO-ASSOCIATED VIRAL (AAV) VECTORS TO TREAT HEREDITARY METHYLMALONIC ACIDEMIA (MMA) CAUSED BY METHYLMALONYL-COA MUTASE
(MMUT) DEFICIENCY

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US Application 18/026,421
Filed on 2023-03-15

OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

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Macao
J/6735
Filed on 2022-12-23

GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA

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Patent Cooperation Treaty
PCT/US2022/082527
Filed on 2022-12-29

GENE THERAPY FOR COMBINED METHYLMALONIC ACIDEMIA/ACIDURIA AND HYPERHOMOCYSTEINEMIA/HOMOCYSTINURIA, COBALAMIN C TYPE, AND DEFICIENCY OF MMACHC

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US Application 18/056,356
Filed on 2022-11-17

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