Aurora joins the clutch of companies linked to Nobel Prize winner and CRISPR trailblazer Jennifer Doudna.
Last May, a nine-month-old baby named KJ received a bespoke gene therapy designed specifically for him. KJ suffered from CSP1 deficiency, a rare and potentially fatal genetic disorder. Now, a group of CRISPR pioneers, including Nobel laureate Jennifer Doudna, is looking to make personalized genetic medicines a reality for millions more patients.
Friday, Aurora Therapeutics debuted with $16 million in seed capital from Menlo Ventures. Its goal: To develop the industry’s first platform technology that can reliably and repeatedly produce gene therapies to address rare disease-causing mutations that would otherwise be implausible to treat at scale.
Aurora will use AI to help design its CRISPR-based editors, leveraging the modality’s “promise of treating the root causes of genetic disease,” Doudna said in a statement on Friday.
Alongside technological advancements, Aurora in its news release also pointed to recent regulatory changes as a key factor in its development model. In particular, the biotech noted changes in the review framework “that support grouping multiple mutations within a disease into unified development paths,” which it said could “make personalized therapies economically and operationally viable.”
Last November, the FDA rolled out its plausible mechanism pathway, specifically targeting investigational gene therapies that address rare diseases with patient populations small enough to render randomized trials implausible.
In an article for The New England Journal of Medicine, Commissioner Marty Makary and Center for Biologics Evaluation and Research Director Vinay Prasad specifically cited the success of Baby KJ’s treatment, noting that this case demonstrated the value of these personalized therapies. Under the new review scheme, the FDA will take into consideration a disease’s underlying pathways, as well as a therapy’s mechanism of action.
“Aurora is uniquely positioned to execute this model by pairing deep gene-editing expertise and hands-on clinical experience with purpose-built clinical, manufacturing, and quality systems designed for rapid, parallel development of mutation-specific therapies,” the startup said on Friday.
Aurora is initially setting its sights on phenylketonuria, a rare metabolic disease linked to mutations in the PAH gene, which leads to a toxic excess of phenylalanine in the blood. If not identified early and treated with a special diet, patients with this condition suffer from cognitive issues. The disease can also compromise brain development in kids and teens. While beginning with PAH, the new biotech plans to expand its coverage to other mutations over time.
