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Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome Post author: Post published:April 30, 2026 Post category: Continue ReadingClinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome
TRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies Post author: Post published:April 30, 2026 Post category: Continue ReadingTRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease Post author: Post published:April 30, 2026 Post category: Continue ReadingA Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
A Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus Post author: Post published:April 30, 2026 Post category: Continue ReadingA Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus
A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus Post author: Post published:April 30, 2026 Post category: Continue ReadingA phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A Post author: Post published:April 30, 2026 Post category: Continue ReadingPersonalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1 Post author: Post published:April 30, 2026 Post category: Continue ReadingA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1
