The Human Cell Atlas project has delivered a fresh tranche of data mapping fibroblasts in healthy and diseased skin and pointing to drug targets with potential in multiple diseases across a range of tissues. Using single cell sequencing and spatial genomics, a technique for showing how gene expression varies at different locations within a tissue, nine different subpopulations of fibroblasts were identified, six in healthy skin and three in disease samples.

Word Sept. 4 from Agios Pharmaceuticals Inc. that the U.S. FDA extended the PDUFA date for the sNDA related to Pyrukynd (mitapivat), after the Cambridge, Mass.-based firm submitted a proposed risk evaluation and mitigation strategy (REMS), drew Wall Street’s attention to the regulatory approach.

After a phase III stumble, Acadia Pharmaceuticals Inc. will drop development of ACP-101, intranasal carbetocin, to treat hyperphagia in patients with the rare genetic disorder Prader-Willi syndrome. Top-line data from the 12-week, double-blind, randomized phase III study missed its primary endpoint by not producing a statistically significant improvement over placebo.

Uniqure NV rang the bell with a best-case scenario in the pivotal phase I/II study with AMT-130 for the treatment of Huntington’s disease, and shares of the Amsterdam-based firm (NASDAQ:QURE) closed Sept. 24 at $47.50, up $33.84, or 248%. The study met its prespecified primary endpoint, with high-dose AMT-130 turning up a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control.

Globally, over half of people living with HIV are women. But in clinical cure trials, they make up only about 20% of participants. And that gender imbalance is causing researchers to miss out on ways to improve cure strategies. Because women’s immune systems appear to be better at controlling HIV infection in a way that silences the reservoir – the provirus integrated into host cells in infected persons.

Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.