New platform aims for personalized gene editing for rare diseases
Aurora, founded by Nobel laureate behind CRISPR tech, nets $16M funding
Written by |
Aurora Therapeutics has launched a personalized gene-editing platform to develop CRISPR-based therapies — ones that precisely correct disease-causing genetic mutations — for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency.
The launch was supported by $16 million in seed funding from Menlo Ventures, a California-based company that works with early-stage founders.
The tech company was founded by Jennifer Doudna, PhD, a CRISPR pioneer and Nobel laureate, and Fyodor Urnov, PhD. It aims to build on recent advances in gene-editing technology that allow the development of therapies targeting different mutations on the same gene in parallel.
“Aurora’s launch signals a turning point for personalized gene editing,” Edward M. Kaye, MD, Aurora’s CEO, said in a company press release. “We now have the science, tools and regulatory tailwinds needed to move from isolated success stories to a sustainable way of developing many therapies in parallel.”
Johnny Hu, PhD, principal at Menlo Ventures, said Aurora “is opening a new frontier in genetic medicine” that will increase patient access to new technology.
“We believe that the pairing of recent advances in gene editing and [artificial intellingence] with a scalable development and regulatory strategy will dramatically expand the number of people who can benefit from gene editing,” Hu said.
AADC is caused by mutations in the DDC gene, which provides instructions for making the AADC enzyme. This enzyme is involved in the production of dopamine and serotonin, two chemical messengers nerve cells use to communicate.
Kebilidi approved in late 2024 as gene therapy for AADC
As with other rare genetic diseases, gene therapies, which typically deliver a functional version of the mutated gene to cells, may be an effective treatment strategy. Kebilidi (eladocagene exuparvovec), a one-time treatment delivered directly into the brain, has been approved since 2024 as a gene therapy for AADC.
Gene-editing technologies aim to make precise changes to a gene’s DNA sequence, potentially correcting the underlying mutation in a particular disease. If successful in target cells, these changes can be permanent.
CRISPR is a gene technology that works like a pair of molecular scissors to cut DNA at specific locations, allowing researchers to add, delete, or replace genetic material. It includes two essential components: an enzyme that cuts the DNA, and a guide RNA, a small piece of RNA that guides the enzyme to the exact gene location that needs to be changed.
By innovating in both clinical development and approval pathways, Aurora is showing the true promise of gene editing for patients who were previously out of reach.
“Since its discovery, CRISPR has offered the promise of treating the root causes of genetic disease, but we lacked a scalable way to bring those therapies to patients with rare mutations,” said Doudna, who published a paper on CRISPR in 2012 with a research partner. The pair went on to win the Nobel Prize in chemistry in 2020.
“By innovating in both clinical development and approval pathways, Aurora is showing the true promise of gene editing for patients who were previously out of reach,” Doudna said.
Aurora platform uses umbrella approach to gene editing
Aurora’s platform will leverage modular gene editors and new regulatory pathways to develop CRISPR gene-editing therapies able to correct disease-causing mutations. The treatments can be customized for other variants of the same gene by using different guide RNAs, allowing the combination of multiple rare mutations within the same gene into a unified development pathway.
This umbrella approach may help advance several mutation-specific gene-editing treatments in parallel, establishing an economically and operationally viable process.
The U.S. Food and Drug Administration is also establishing a regulatory pathway that enables the approval of a therapeutic platform that can be adapted for different mutations, allowing personalized therapies to move to market faster. The plausible mechanism pathway is designed to accelerate the approval of personalized therapies by relying on a small number of successful cases with meaningful clinical improvement rather than large clinical trials.
Aurora’s initial program focuses on phenylketonuria, a metabolic disorder caused by mutations in the PAH gene that lead to toxic elevations of phenylalanine, a protein building block, in the blood.
