Hemophilia CRISPR Gene Editing

Hemophilia Gene Therapy

Hemophilia A gene therapy: current and next-generation approaches

Next-generation gene therapies incorporating chimeric DNA sequences in the transgene can increase clotting factor synthesis and secretion, and advance the efficacy, safety, and durability of gene replacement therapy for hemophilia A as well as other blood clotting disorders.

Clinical applications of gene and cell therapies: case studies for the relevance of precision medicine

With the rapid growth of gene and cell therapies, biotechnology and pharmaceutical companies face a call to action: we must establish proper selection and monitoring protocols to provide patients with the safest and most effective therapeutic options for genetic diseases. This article presents two clinical programs for gene and allogeneic cell therapies and provides a primer for the relevance of precision medicine applications.

MSUD Gene Therapy
Decidua Stromal Cells – Cell Therapy