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<p>The FDA’s recent approval of gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), for sickle cell disease (SCD) marks a significant advance, offering hope for transformative treatments. However, this milestone underscores the urgent need for therapeutic equity to ensure access for underserved populations. India bears a significant global SCD burden, particularly among Scheduled Tribes (STs), with carrier prevalence ranging from 8 to 40% and homozygous cases at 0.4–1.5%. India’s National Sickle Cell Anemia Elimination Mission aims to address this through community screening, genetic counseling, and CRISPR-CAS technology. Challenges include limited stem-cell transplantation infrastructure, inconsistent screening protocols, and societal stigma affecting diagnosis and treatment. Comprehensive strategies combining hydroxyurea, penicillin prophylaxis, and emerging gene therapies with patient-centric and collaborative approaches are essential to ensure equitable care and alleviate SCD burdens globally. This commentary aims to provide a comprehensive overview of current incidence and existing strategies for marginalized community with integration of gene therapies for better outcomes.</p>

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Sickle Cell Anemia Elimination in India: From Inclusive Care for the Marginalized Communities to Gene Therapies

  • 期刊论文

  • 发表日期：

    2025年9月5日

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  • Hrishikesh Munshi,
  • Suchitra Surve,
  • Yogeshwar Kalkonde,
  • Nilesh Wasekar,
  • Rahul K. Gajbhiye

摘要

The FDA’s recent approval of gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), for sickle cell disease (SCD) marks a significant advance, offering hope for transformative treatments. However, this milestone underscores the urgent need for therapeutic equity to ensure access for underserved populations. India bears a significant global SCD burden, particularly among Scheduled Tribes (STs), with carrier prevalence ranging from 8 to 40% and homozygous cases at 0.4–1.5%. India’s National Sickle Cell Anemia Elimination Mission aims to address this through community screening, genetic counseling, and CRISPR-CAS technology. Challenges include limited stem-cell transplantation infrastructure, inconsistent screening protocols, and societal stigma affecting diagnosis and treatment. Comprehensive strategies combining hydroxyurea, penicillin prophylaxis, and emerging gene therapies with patient-centric and collaborative approaches are essential to ensure equitable care and alleviate SCD burdens globally. This commentary aims to provide a comprehensive overview of current incidence and existing strategies for marginalized community with integration of gene therapies for better outcomes.