Companies in #Alpha-1 Antitrypsin Deficiency
Found 3 companies operating in this sector.
Intellia Therapeutics is a clinical-stage biotechnology company developing in vivo and ex vivo CRISPR/Cas9-based gene-editing therapies. Its lead program NTLA-2001 is being studied for transthyretin (ATTR) amyloidosis, with additional programs in hereditary angioedema, hemophilia A/B, and other genetic diseases. Intellia has also invested heavily in commercial-launch infrastructure, including a dedicated team working on cell-and-gene therapy readiness in advance of its first potential approvals.
Korro Bio is a clinical-stage biopharmaceutical company developing genetic medicines based on RNA editing for rare and highly prevalent diseases. Its proprietary OPERA (Oligonucleotide Promoted Editing of RNA) platform uses GalNAc-conjugated oligonucleotides to recruit endogenous adenosine deaminase acting on RNA for precise, transient single-base edits, an approach designed to avoid the permanent DNA cuts associated with gene editing. The company's lead programme, KRRO-121, is in the Phase 1/2 ANCHOR trial in healthy volunteers for hyperammonemia associated with urea cycle disorders and hepatic encephalopathy; KRRO-111 is in late preclinical development for alpha-1 antitrypsin deficiency, targeting both the lung and liver manifestations of the disease, with a regulatory submission and first-in-human entry guided for the first half of 2027. Korro also has discovery programmes in neurologic and pain indications and a collaboration with Novo Nordisk covering cardiometabolic disease. The company is headquartered in Cambridge, Massachusetts and trades on the Nasdaq Global Market under the ticker KRRO.
Sangamo Therapeutics (NASDAQ: SGMO) is a genomic medicine company headquartered in Richmond, California, focused on translating ground-breaking science into genomic medicines using zinc finger nucleases (ZFNs), zinc finger protein transcription factors (ZFP-TFs), and adeno-associated virus (AAV) delivery. Sangamo's clinical-stage pipeline includes giroctocogene fitelparvovec (hemophilia A gene therapy, partnered with Pfizer) and isavufermin or SAR445136 (alpha-1 antitrypsin deficiency, partnered with Sanofi). The company is also developing preclinical in vivo genome-editing programs and ex vivo gene-edited cell therapies.