A new CRISPR startup is betting regulators will ease up on gene-editing
Aurora Therapeutics aims to treat an estimated 13,500 U.S. patients with phenylketonuria using a base-editing platform and a new FDA pathway requiring data from few patients.
6 Articles
6 Articles
A new CRISPR startup is betting regulators will ease up on gene-editing
Here at MIT Technology Review we’ve been writing about the gene-editing technology CRISPR since 2013, calling it the biggest biotech breakthrough of the century. Yet so far, there’s been only one gene-editing drug approved. It’s been used commercially on only about 40 patients, all with sickle-cell disease. It’s becoming clear that the impact of CRISPR isn’t as big as we all hoped. In fact, there’s a pall of discouragement over the entire field—…
Aurora: Developing Therapies for the Millions of Patients with Rare Diseases
Johnny began his Ph.D. evolving new Cas9 enzymes in 2012, the same year that Jennifer Doudna published her seminal paper unveiling CRISPR as a revolutionary tool for gene editing and for which she would win her Nobel Prize in 2020. In the time since, we’ve seen immense progress in correcting genetic mutations in humans with remarkable clinical efficacy and the potential for one-time cures. However, the number of patients and diseases treated by …
The "CRISPR gene-editing tool" is a crucial foundation for genome-editing technologies in many fields – from biology and medicine to agriculture and industry. A team from the Helmholtz Institute... has shown that the CRISPR-Cas systems that form its basis are even more versatile than previously thought.
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