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Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

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  • Post published:March 25, 2026
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Continue ReadingClinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Stem Cell Therapy for Limbal Stem Cell Deficiency

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  • Post published:March 25, 2026
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Continue ReadingStem Cell Therapy for Limbal Stem Cell Deficiency

Stem Cell Gene Therapy for Sickle Cell Disease

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  • Post published:March 25, 2026
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Continue ReadingStem Cell Gene Therapy for Sickle Cell Disease

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

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  • Post published:March 25, 2026
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Continue ReadingA Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

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  • Post published:March 25, 2026
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Continue ReadingOpen-Label Study of mRNA-3927 in Participants With Propionic Acidemia

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

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  • Post published:March 25, 2026
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Continue ReadingAn Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration

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  • Post published:March 25, 2026
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Continue ReadingA Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration

CCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer

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  • Post published:March 25, 2026
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Continue ReadingCCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

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  • Post published:March 25, 2026
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Continue ReadingGene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

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  • Post published:March 25, 2026
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Continue ReadingPhase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
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