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Aurora Therapeutics Launches to Scale Bespoke Gene Editing for PKU

Co-founded by Jennifer Doudna with Fyodor Urnov, the startup plans to use a base-editing platform under the FDA’s “plausible mechanism” pathway.

Overview

  • Aurora unveiled a $16 million seed round from Menlo Ventures and named biotech veteran Ed Kaye as CEO.
  • The company intends to develop multiple mutation-specific therapies in parallel for phenylketonuria, starting with versions for the three most common PAH mutations.
  • Its approach centers on a reusable base-editing platform that swaps guide RNAs to retarget the same core editor for different mutations.
  • Aurora aims to leverage the FDA’s newly described pathway that could allow approvals for rare diseases using small patient datasets tied to a consistent technology platform.
  • Academic efforts will continue separately, with the Innovative Genomics Institute pursuing bespoke editors for ultra-rare cases and a CHOP/Penn trial preparing to test similar editors across related disorders.