First in-vivo gene therapy administered in ‘historic event’

In a landmark development for gene therapy, the first patient to be administered with an in vivo ​CRISPR therapy was announced today.

The Phase I/II trial is being conduct by Allergan and Editas to trial the partners’ AGN-151587 sub-retinal injection for the treatment of Leber congenital amaurosis 10 (LCA10).

After dosing, the patient will be monitored to determine the safety, tolerability and efficacy of the treatment. A further 17 patients will be recruited to the trial.

The condition is a rare inherited form of blindness caused by mutations in the centrosomal protein 290 gene. At present, there are no approved treatment options for those living with the condition.

LCA10 is the most common cause of inherited childhood blinds, with approximately two to three children born with the condition per 100,000 people born worldwide. The condition is monogenic, which means that the replacement of one gene could be enough to treat the condition.

“This dosing is a truly historic event – for science, for medicine, and most importantly for people living with this eye disease,”​ said Cynthia Collins, CEO of Editas Medicine.

Editas and Allergan entered their current partnership in 2017, with the latter company receiving the option to license up to five treatment candidates, including the gene therapy candidate being tested in this trial.

Editas received $90m (€80m) upfront in the initial part of the deal, with further undisclosed payments dependent on developmental milestones.

The partnership will focus on ocular programs, which is a therapeutic area where gene therapies have already achieved success, after Spark’s Luxturna (voretigene neparvovec) received US Food and Drug Administration approval​ for an inherited form of vision loss in 2017.

For Allergan, the news that the trial is progressing is a positive for of its purchaser, AbbVie, which expects to conclude a takeover deal for $63bn​, with the European Union approving the deal yesterday, leaving the transaction waiting on US approval.