Regeneron Pharmaceuticals will share in development of Tessera Therapeutics’ TSRA-196, a Phase 1-ready gene-editing therapy for alpha-1 antitrypsin deficiency (AATD). Other companies developing genetic medicines for this rare disease include Beam Therapeutics, Wave Life Sciences, Korro Bio, and AIRNA.
Beam Therapeutics’ first clinical data in the rare disease alpha-1 antitrypsin deficiency “set a bar for efficacy in the space,” according to one analyst. But the ongoing market slide poses challenges for biotech companies aiming to raise money by selling stock.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
AIRNA’s therapy for alpha-1 antitrypsin deficiency, or AATD, edits RNA to address the underlying cause of this rare inherited disease. Wave Life Sciences and Korro Bio are both further along in the development of RNA-editing therapies for AATD, but AIRNA claims its therapy could be best in class.
The companies will partner on development and commercialization of ARO-AAT, Arrowhead's Phase II RNA-interference drug for alpha-1 antitrypsin-associated liver disease, a rare genetic disorder. An analyst wrote that the deal makes sense given Takeda's position in the AAT-augmentation therapy market.