The field of gene therapy has seen unprecedented growth in recent years, with 20 approved indications in the U.S., a record number of approvals in 2023, and a rapid expansion of investigational trials across various diseases. Great strides have been made in potential treatments for inherited and acquired genetic diseases, including hemophilia B, sickle cell…
Unicorn gene-delivery firm VectorBuilder eyes future breakthroughs
Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current…
Viral vector analysis for successful targeting in gene therapy
April 25, 2023 is an important anniversary in the field of genomics. It marks the seventieth anniversary of four scientists — James Watson, Francis Crick, Maurice Wilkins, and Rosalind Franklin — publishing a paper in Nature and proposing the double helix structure of DNA1. The discovery of the double helix with complementary strands helped scientists…