Lep001 – gene therapy for the treatment of congenital lipodystrophy

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingLep001 – gene therapy for the treatment of congenital lipodystrophy

Development of an off-the-shelf iPSC derived CAR T cell therapy for the treatment of solid tumors

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingDevelopment of an off-the-shelf iPSC derived CAR T cell therapy for the treatment of solid tumors

Development of an AAV Epigenetic Gene Therapy for Gain-of-Function SCN9A Disorders and Chronic Pain

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingDevelopment of an AAV Epigenetic Gene Therapy for Gain-of-Function SCN9A Disorders and Chronic Pain

Evaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingEvaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome

Genetic Therapy Targeting mHTT mRNA and Somatic Expansion to Treat Huntington’s Disease

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingGenetic Therapy Targeting mHTT mRNA and Somatic Expansion to Treat Huntington’s Disease

A Novel Gene Therapy to Target Glioblastoma via Custom-Engineered Adenovirus-Associated Viral Vectors

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingA Novel Gene Therapy to Target Glioblastoma via Custom-Engineered Adenovirus-Associated Viral Vectors

Parkin Gene Therapy for Parkinson’s Disease

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingParkin Gene Therapy for Parkinson’s Disease

A novel non-genotoxic antibody conditioning therapeutic to expand access to and safety of HSCT and CD34+ gene therapies

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingA novel non-genotoxic antibody conditioning therapeutic to expand access to and safety of HSCT and CD34+ gene therapies

Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia

• Post author:
• Post published:December 28, 2025
• Post category:

Continue ReadingHematopoietic Stem Cell Gene Therapy for Alpha Thalassemia

Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.

• Post author:
• Post published:December 28, 2025
• Post category:

Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy…

Continue ReadingDevelopment of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.