NHGRI | Technology Transfer

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/006735
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

ABERRANT POST-TRANSLATIONAL MODIFICATIONS(PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS

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US Application 17/923,394
Filed on 2022-11-04

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

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US Application 17/766,067
Filed on 2022-04-01

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

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European Patent
20804709.2
Filed on 2022-04-26

OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

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Japan
2022-000125
Filed on 2022-01-04

CANCER DETECTION AND CLASSIFICATION

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US Application 17/632,783
Filed on 2022-02-03

NON-DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA

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Hong Kong
62021044683.2
Filed on 2021-12-16

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