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.

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.

Apollo Therapeutics Group Ltd. and Sunshine Lake Pharma Co. Ltd. inked a potential $938 million licensing deal for APL-18881 (HEC-88473), Sunshine’s dual fibroblast growth factor 21 (FGF21)/glucagon-like peptide-1 (GLP-1) receptor agonist currently in a phase II study for type 2 diabetes.

Gene therapy faces complexities in delivering treatments due to persistent safety concerns and daunting immune responses, but Next Generation Gene Therapeutics Inc. has found a way around this issue using dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function.

Nearly $3.8 billion was earned by Novo Nordisk A/S and Eli Lilly and Co. in the third quarter for their glucagon-like peptide-1 (GLP-1) drugs to treat obesity. Novo’s GLP-1 drug, Wegovy (semaglutide), approved by the U.S. FDA in June 2021, had sales of DKK17.3 billion (US$2.5 billion), while Lilly’s Zepbound (tirzepatide), which is a GLP-1 and glucose-dependent insulinotropic polypeptide dual agonist FDA-approved in November 2023, posted sales of $1.26 billion for the quarter.

Positive findings from a phase III trial of semaglutide in metabolic dysfunction-associated steatohepatitis (MASH) is moving Novo Nordisk A/S to expand the glucagon-like peptide-1 (GLP-1) agonist’s indications in the U.S. and Europe, the Danish pharma said, as the MASH field sights more novel therapies.

Researchers at Harvard Medical School have found that blocking the neuron-released peptide CGRP decreases pain sensitivity and reduces lesion size in endometriosis. Endometriosis is a painful, steroid-dependent inflammatory condition in which tissue similar to that of the endometrial lining grows and establishes outside the uterine mucosa.

Equillium Inc. plans to continue on its own with itolizumab – now the top pipeline priority – as Ono Pharmaceutical Co. Ltd. Is letting expire the option for rights to the monoclonal antibody, designed to target the CD6-ALCAM signaling pathway.