Aurora Therapeutics has announced on Aug. 5 that it is discontinuing its lead gene editing program and letting go of several employees. The company, which launched in January with $16 million in seed funding from Menlo Ventures, initially focused on developing therapies for phenylketonuria (PKU), a rare inherited disorder that impairs the body’s ability to break down the amino acid phenylalanine.
A spokesperson for Aurora said competition was a key factor in deprioritizing the PKU program. “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 is among those pursuing similar treatments for PKU, developing BEAM-304, a liver-targeting lipid-nanoparticle formulation designed to correct mutations responsible for the disorder. According to Beam’s website, this therapy is currently undergoing investigational new drug-enabling studies.
Despite these changes, Aurora said it remains committed to advancing gene editing technologies for rare diseases. “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.” Co-founder Jennifer Doudna stated earlier this year that Aurora is leveraging artificial intelligence to design CRISPR-based editors with hopes of treating genetic diseases at their root causes.
The challenges faced by Aurora reflect broader difficulties within biopharma companies attempting personalized N-of-1 therapies—treatments tailored specifically for individual patients with ultra-rare conditions. Previous efforts by other companies such as Grace Therapeutics have also encountered regulatory hurdles when seeking approval for individualized medicines.
“Aurora will provide more information ‘soon,’” according to its spokesperson.
