While women make up half the world’s population and own two out of every five businesses, there are substantial knowledge gaps about conditions affecting their health – mostly due to decades of research excluding women from clinical trials and investment decisions.

PTC Therapeutics Inc.’s accelerated go-ahead from the U.S. FDA for Kebilidi (eladocagene exuparvovec-tneq), a recombinant adeno-associated virus serotype 2-based gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, came with a priority review voucher (PRV) that the company intends to monetize.

At the BioFuture 2024 conference held in New York in November, Seema Kumar, the CEO of Cure, described women’s health as something that has been directed at the “bikini area.” That “bikini” bias extended to both diseases and their causes – women’s health covered the breasts and reproductive system, and its causes were hormonal. Both concepts are far too narrow.

It’s difficult to fathom that the health of half the world’s population is underserved. But it’s a hard truth. There are many conditions that disproportionately impact women. Other conditions and diseases affect women in different ways than men. Decades of research excluding women from clinical trials and investment decisions in male-dominated board rooms have ignored these facts. Though an increasing number of women are now managing investments and driving the research, it’s all still woefully behind. In BioWorld’s new report, Healing the health divide, we’ve highlighted the disparities.

On the heels of U.S. FDA approval of its first T-cell therapy, Adaptimmune Therapeutics plc reported positive results from a pivotal study for its second candidate, letetresgene autoleucel, expected to support a rolling BLA submission in 2025 that could bolster the firm’s offerings as it narrows its focus on the rare sarcoma space.