New Data Boost For Intellia’s Pioneering In Vivo Gene-Editing Therapy
Next Step: Pivot To Cardiomyopathy To Take On Pfizer
New data show that a high dose of Intellia’s in vivo CRISPR-Cas9 based therapy is safe and effective for up to six months, which could unlock a new era in genetic disease treatment.
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Vertex is expanding its investments across advanced therapies again, R&D chief David Altshuler adding a second in vivo gene-editing deal to its pipeline.
Novartis is once again looking to be a pioneer in cell and gene therapy, though this time is selecting a potentially less complex administration method.
AstraZeneca is preparing a US filing for hereditary transthyretin-mediated amyloid polyneuropathy for eplontersen, which it hopes will compete with Pfizer and Alnylam's therapies in that rare disease space.