Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein Post author: Post published:April 20, 2026 Post category: Continue ReadingStudy to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease Post author: Post published:April 20, 2026 Post category: Continue ReadingTransplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia Post author: Post published:April 20, 2026 Post category: Continue ReadingOpen-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Stem Cell Gene Therapy for Sickle Cell Disease Post author: Post published:April 20, 2026 Post category: Continue ReadingStem Cell Gene Therapy for Sickle Cell Disease
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Post author: Post published:April 20, 2026 Post category: Continue ReadingStudy of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations Post author: Post published:April 20, 2026 Post category: Continue ReadingA Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency Post author: Post published:April 20, 2026 Post category: Continue ReadingClinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Stem Cell Therapy for Limbal Stem Cell Deficiency Post author: Post published:April 20, 2026 Post category: Continue ReadingStem Cell Therapy for Limbal Stem Cell 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 Post author: Post published:April 20, 2026 Post category: 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
A Vaccine (Neoantigen-Targeted ppDC) for the Treatment of H3 G34-mutant Diffuse Hemispheric Glioma Post author: Post published:April 20, 2026 Post category: Continue ReadingA Vaccine (Neoantigen-Targeted ppDC) for the Treatment of H3 G34-mutant Diffuse Hemispheric Glioma
