Avrobio is sidestepping market and regulatory issues in Fabry disease by suspending development of AVR-RD-01, but says it remains focused on advancing treatment of lysosomal storage disorders with gene therapies.

Analysts’ fears have been confirmed as a US FDA demand for clinical outcomes data has forced Applied Therapeutics to delay a planned filing for its lead product candidate, AT-007, in the rare disease galactosemia based on biomarker data alone.

In a wilderness period for biotech stocks, platform company revenues may offer investors less volatile biotech exposure. They are unlikely to shepherd an exit from the current doldrums, though.