As CRISPR technology continues to evolve, a new startup, Aurora Therapeutics, is positioning itself to capitalize on potential regulatory changes that could transform the landscape of gene editing. Despite the significant promise of CRISPR—a technology hailed as the most groundbreaking biotech advancement of the century—only one gene-editing drug has received approval to date, and it has seen limited use in treating patients with sickle-cell disease. This reality has dampened expectations within the field, prompting some experts to declare a slowdown in the anticipated gene-editing revolution. Aurora Therapeutics, backed by $45 million in funding from Menlo Ventures and advised by CRISPR co-inventor Jennifer Doudna, is advocating for a more flexible regulatory approach that would allow for the approval of gene-editing treatments that can be customized without necessitating extensive new trials.
Aurora’s inaugural target is phenylketonuria (PKU), a rare inherited disorder where patients cannot metabolize the amino acid phenylalanine, leading to severe health issues if not managed. Current treatments require patients to adhere to a strict diet, but gene editing holds the potential to correct the underlying genetic cause. Aurora’s strategy involves developing a single gene-editing drug that can be slightly modified to address various mutations associated with PKU, which would be more efficient than creating a new drug for each individual mutation. This innovative approach aligns with sentiments expressed by FDA leadership regarding the need for a new regulatory framework supporting personalized therapies that cannot easily be evaluated through traditional clinical trial methods.
The ambition of Aurora Therapeutics stems from a broader recognition of the barriers facing gene-editing technologies, particularly in addressing a wide range of inherited conditions. While many companies focus on a handful of diseases where a single edit can suffice, millions of patients with less common genetic disorders remain without viable treatment options. Aurora’s model aims to bridge this gap by creating a platform that can adapt to various genetic mutations, ultimately enabling personalized treatment solutions. As discussions with the FDA progress, the hope is to pioneer a pathway for bespoke gene therapies that make a meaningful impact on patients’ lives, demonstrating that CRISPR’s capabilities can indeed be harnessed for broader applications in the realm of genetic medicine.
Source: A new CRISPR startup is betting regulators will ease up on gene-editing via MIT Technology Review