CRISPR Therapeutics specializes in developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. This technology enables precise, directed changes to genomic DNA for treating serious human diseases. The company focuses on various disease areas including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.
Exsilio Therapeutics develops tools that use naturally occurring genetic elements to insert therapeutic genes into cells via RNA intermediates. The company combines computational modeling with laboratory experiments to design elements that target safe harbor sites in disease‑relevant cells, enabling precise and stable gene integration. Its peer, Averna Therapeutics, also employs similar genetic elements but focuses on delivering medicines in lipid nanoparticles to treat a broad range of diseases, including genetic disorders, cancer, and autoimmune conditions.
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