Crispr Therapeutics AG (CRSP)

[WebSlinger]

This is from today's ARK Investments weekly newsletter:

At the Peripheral Nerve Society Conference this weekend, Intellia Therapeutics made history by presenting the first data of a CRISPR Cas9 based in vivo gene editing therapy in patients with Hereditary Transthyretin Amyloidosis (hATTR). Please find the results of this milestone trial in this New England Journal of Medicine publication.

hATTR is a disease in which amyloid proteins build up and, if untreated, can cause multiple organ failures. Typically, the primary impact is on the heart and nerves.

Until now, patients with hATTR faced limited curative treatment options. Alnylam’s Patisiran knocks down serum TTR by 80%, but greater knock down rates could enhance the clinical benefits.

Intellia Therapeutic’s presentation highlighted that editing the TTR gene could result in a one-time treatment, eliminating the need for chronic therapy. In a small six-person dose escalation study, the company showed that, in the second cohort at 0.3 mg/kg, three patients achieved an 87% average mean reduction in TTR and a max reduction of 96%, surpassing that of Patrisiran and other therapies. In our view, as Intellia escalates the therapeutic dose, higher reductions in TTR will lead to better patient outcomes.

We believe this data will enter the history books, signifying that in vivo gene editing and one-time cures are possible. The only question remaining is will it be durable?