Phase 1/ 2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients with FOXG1 Syndrome (FS) Post author: Post published: November 7, 2026 Post category: Continue ReadingPhase 1/ 2 Study of FRF-001, an AAV-9 Gene Therapy, in Patients with FOXG1 Syndrome (FS)
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A Post author: Post published: November 7, 2026 Post category: Continue ReadingPersonalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Temporal AI models for programming therapeutic cell state transitions Post author: Post published: November 7, 2026 Post category: Continue ReadingTemporal AI models for programming therapeutic cell state transitions
A precision organoid platform to investigate APOE4 dependent cerebrovascular impairments Post author: Post published: November 7, 2026 Post category: Continue ReadingA precision organoid platform to investigate APOE4 dependent cerebrovascular impairments
Leveraging lessons from normal development to overcome barriers to efficient transplantation of stem cell derived cortical neurons. Post author: Post published: November 7, 2026 Post category: Continue ReadingLeveraging lessons from normal development to overcome barriers to efficient transplantation of stem cell derived cortical neurons.
Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy for Multiple Sclerosis Post author: Post published: November 7, 2026 Post category: Continue ReadingStem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy for Multiple Sclerosis
Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies Post author: Post published: November 7, 2026 Post category: Continue ReadingLate-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies
Microglia replacement therapy for CSF1R-related Leukoencephalopathy Post author: Post published: November 7, 2026 Post category: Continue ReadingMicroglia replacement therapy for CSF1R-related Leukoencephalopathy
CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia Post author: Post published: November 7, 2026 Post category: Continue ReadingCRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia
Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders Post author: Post published: November 7, 2026 Post category: Continue ReadingDevelopment of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders
