Seven months after launching with $16 million in seed financing, Jennifer Doudna’s Aurora Therapeutics is pivoting away from its lead indication, phenylketonuria, owing to growing competition in the field.
Aurora Therapeutics has joined the growing list of companies struggling to create N-of-1 therapies for ultrarare genetic diseases. The fledgling biotech is parting ways with both its lead gene editing program and “several” staffers.
Aurora launched in January with $16 million in seed capital from Menlo Ventures and an initial focus on phenylketonuria (PKU), a rare, inherited disorder in which the body is unable to break down the amino acid phenylalanine. Toxins can then build up, leading to neurological complications.
However, Aurora has since encountered competition in the PKU space, a company spokesperson told BioSpace on Tuesday.
“We have decided to deprioritize the PKU program as others in the field have started their own programs,” the spokesperson said in an email. “As part of that, several team members were unfortunately let go.”
Beam Therapeutics, for one, is also taking a gene-editing approach to PKU with BEAM-304, a liver-targeting lipid-nanoparticle (LNP) formulation of base-editing reagents designed to correct mutations in the phenylalanine hydroxylase gene that causes the disorder. The therapy is in investigational new drug (IND)-enabling studies, according to Beam’s website.
But Aurora is not giving up.
“Aurora remains committed to scaling gene editing for rare diseases,” the spokesperson added. “We are focusing on bringing novel technology to address the areas of highest unmet need.”
Aurora is leveraging AI to help design its CRISPR-based editors, leveraging the modality’s “promise of treating the root causes of genetic disease,” co-founder and CRISPR pioneer Jennifer Doudna said in a statement on Jan. 9.
N-of-1 therapies caught the attention of many within biopharma—and outside of—in May 2025 when a 9-month-old baby named KJ suffering from CSP1 deficiency, a rare and potentially fatal genetic disorder, received a gene therapy designed just for him. Reports indicate that KJ is thriving.
Aurora’s mission is to scale up the impact of these personalized gene editing therapies to make them available to more patients.
Herein lies the challenge that has felled multiple companies attempting to develop personalized medicines.
Grace Therapeutics, founded by Matt Wilsey in an attempt to create a cure for his daughter, Grace, who has an ultra-rare disease called NGLY1 deficiency, is struggling to win approval for the gene therapy it has developed. While the company has treated 10 patients with GTx-104 and Wilsey claims it has led to improvements in some patients, including his daughter, according to STAT News, the FDA rejected the therapy in April. The regulator’s issues appear to be related to manufacturing.
Aurora, for its part, is pressing on. “We believe that the need for scalable solutions has only grown and that recent technological advances and regulatory changes will enable better treatments for patients,” the company spokesperson said on Tuesday. Aurora will provide more information “soon.”
