ASH 2022 | Treatment of severe SCD with OTQ923 CRISPR/Cas9 gene therapy
Akshay Sharma, MBBS, St. Jude Children’s Research Hospital, Memphis, TN, provides insight into the mechanism of action and efficacy of OTQ923, an autologous, CRISPR/Cas9-edited hematopoietic stem cell product in patients with severe sickle cell disease (SCD). The therapy targets the globin locus in hematopoietic stem cells, helps increase fetal hemoglobin levels, and removes sickle cell-associated complications and side effects in patients. The therapeutic benefits of this treatment have persisted in follow-ups six and 12 months after the treatment. This interview took place at the 64th ASH Annual Meeting and Exposition congress held in New Orleans, LA.
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Disclosures
Spotlight Therapeutics: Consultancy; CRISPR Therapeutics: Other: Clinical Trial Site PI, Research Funding; Novartis: Other: Clinical Trial Site PI; Magenta Therapeutics: Other: Clinical Trial Site PI; Vindico Medical Education: Honoraria; Medexus Inc: Consultancy; Vertex Pharmaceuticals/CRISPR Therapeutics: Consultancy, Membership on an entity’s Board of Directors or advisory committees, Other: Clinical Trial Site PI.
