A new CRISPR startup is betting regulators will ease up on gene-editing

A New CRISPR Startup is Betting Regulators Will Ease Up on Gene-Editing

The gene-editing technology CRISPR has been hailed as one of the biggest biotech breakthroughs of the century, but so far, it has only been used commercially on about 40 patients, all with sickle-cell disease. The impact of CRISPR hasn't lived up to the hype, and some are saying the gene-editing revolution has "lost its mojo." A new startup, Aurora Therapeutics, is hoping to change that by developing an "umbrella approach" to testing and commercializing treatments.

The Problem with Gene-Editing

The need to change regulations around gene-editing treatments was endorsed in November by the head of the US Food and Drug Administration, Martin Makary, who said the agency would open a "new" regulatory pathway for "bespoke, personalized therapies" that can't easily be tested in conventional ways. The problem is that most gene-editing companies are chasing the same few conditions, such as sickle-cell, where a single edit works for all patients. But that leaves around 400 million people who have 7,000 other inherited conditions without much hope to get their DNA fixed.

Aurora's Approach

Aurora's first target is the rare inherited disease phenylketonuria (PKU), which affects about 20,000 people in the US. People with PKU lack a working version of an enzyme needed to use up the amino acid phenylalanine, a component of pretty much all meat and protein. If the amino acid builds up, it causes brain damage. In theory, gene editing can fix PKU. In mice, scientists have already restored the gene for the enzyme by rewriting DNA in liver cells, which both make the enzyme and are some of the easiest to reach with a gene-editing drug.

The Challenge of Personalized Gene-Editing

The problem is that in human patients, many different mutations can affect the critical gene. According to Cory Harding, a researcher at Oregon Health Sciences University, scientists know about 1,600 different DNA mutations that cause PKU. There's no way anyone will develop 1,600 different gene-editing drugs. Instead, Aurora's goal is to eventually win approval for a single gene editor that, with minor adjustments, could be used to correct several of the most common mutations, including one that's responsible for about 10% of the estimated 20,000 PKU cases in the US.

The FDA's Role

Edward Kaye, the CEO of Aurora, says that the way the FDA approves gene editing has to change. "We can't have a separate clinical trial for each mutation," he says. The FDA has been very understanding of this issue, and Kaye is optimistic that they will open a new regulatory pathway for bespoke, personalized therapies.

The Future of Gene-Editing

The future of gene-editing is looking bright, but it's not without its challenges. The technology is still in its early stages, and there are many regulatory hurdles to overcome. However, with companies like Aurora pushing the boundaries of what's possible, we can expect to see significant advancements in the years to come.

Why This Matters

The implications of gene-editing are far-reaching and profound. If we can develop a way to edit genes with precision and accuracy, we could potentially cure some of the world's most devastating diseases. We could also use gene-editing to improve crop yields, increase food security, and even enhance human performance.

Deep Dive

Biotechnology and health are rapidly evolving fields, and gene-editing is at the forefront of this revolution. With the help of companies like Aurora, we can expect to see significant advancements in the years to come.

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Source: https://www.technologyreview.com/2026/01/09/1130945/crispr-startup-aurora-betting-regulation-pku/