Last May, a nine-month-old baby named KJ received a personalized gene therapy for CSP1 deficiency, a rare and potentially fatal genetic disorder. The treatment was designed specifically for him. Now, Aurora Therapeutics, which includes CRISPR pioneer and Nobel laureate Jennifer Doudna among its founders, is seeking to expand the reach of such individualized therapies.
On Friday, Aurora Therapeutics announced it has secured $16 million in seed funding from Menlo Ventures. The company aims to create what it describes as the industry’s first platform technology capable of reliably producing gene therapies for rare disease-causing mutations that are otherwise difficult to address at scale.
Aurora plans to use artificial intelligence to design its CRISPR-based gene editors. “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.
The company also cited recent regulatory changes as important for its business model. Specifically, Aurora highlighted updates in review frameworks that allow grouping multiple mutations within a disease into unified development paths. According to the company, these changes could make personalized therapies more viable both economically and operationally.
In November 2025, the U.S. Food and Drug Administration (FDA) introduced a new pathway called the plausible mechanism pathway. This approach targets investigational gene therapies intended for rare diseases with patient populations too small for randomized trials. Under this framework, the FDA considers both the underlying pathways of diseases and how proposed therapies work.
An article in The New England Journal of Medicine by Commissioner Marty Makary and Center for Biologics Evaluation and Research Director Vinay Prasad referenced Baby KJ’s case as evidence supporting personalized treatments under this new review process.
“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,” Aurora stated on Friday.
Initially, Aurora will focus on phenylketonuria (PKU), a rare metabolic disorder caused by mutations in the PAH gene that leads to high levels of phenylalanine in the blood. If not treated early through dietary intervention, PKU can result in cognitive problems and hinder brain development in children and teenagers. While starting with PAH-related conditions, Aurora intends to expand its efforts to cover additional mutations over time.
