Investor excitement over the development of products that can be injected directly into patients to correct inherited diseases, or “in vivo gene editing,” has largely been fueled over the past few months by Intellia Therapeutics and its experimental therapy NTLA-2001 to address the rare liver disease transthyretin (ATTR) amyloidosis. But Intellia’s huge run—its stock is up 70% year to date—has overshadowed some of the players in the field, including LogicBio Therapeutics, which has seen its shares lose half their value this year.
California biotech Inhibrx released interim results today from a Phase I trial looking at a recombinant fusion protein program, INBRX-101. The study looked at patients with alpha-1 antitrypsin deficiency, or AATD.