Couple's Blindness Gene Therapy Earns $3M Breakthrough Prize

Molecular biologist Jean Bennett and ophthalmologist Albert Maguire have been awarded the $3 million Breakthrough Prize in Life Sciences for their development of Luxturna, the first approved gene therapy for inherited blindness. The therapy targets Leber congenital amaurosis (LCA), a genetic disorder causing blindness. Its success was evidenced in clinical trials where patients described profound visual improvements, including seeing their children's faces for the first time.

Bennett and Maguire, who met during their studies and later adopted two dogs treated with the therapy, faced significant challenges in developing the treatment. Their work, alongside physician Katherine High, involved introducing a working copy of the faulty RPE65 gene into retinal cells, restoring lost vision. This achievement represents a major milestone in genetic medicine.

The prize ceremony also recognized advancements in gene therapy for sickle cell anemia and beta thalassaemia, as well as discoveries in frontotemporal dementia and ALS. However, Bennett also voiced concerns about the current US administration's impact on scientific research, warning of potential damage to the field and a brain drain.

Both Bennett and another prize recipient, Stuart Orkin, expressed dismay over political attacks on academic institutions and scientific infrastructure. They emphasized the importance of sustained investment and support for scientific endeavors to prevent future losses and ensure continued progress in medical breakthroughs.