🌟 Some Newly Launched / Emerging Genetic Medicines (Global)
- Zolgensma (Onasemnogene abeparvovec)
- For Spinal Muscular Atrophy (SMA).
- A one-time gene therapy that replaces the faulty gene causing muscle weakness.
- Casgevy (Exa-cel – CRISPR-based gene editing, by Vertex/CRISPR Therapeutics)
- Approved in 2023 (US & UK).
- Treats Sickle Cell Disease and Beta-Thalassemia by editing bone marrow stem cells.
- Hemgenix (Etranacogene dezaparvovec)
- For Hemophilia B.
- Increases body’s ability to make clotting factor IX.
- Luxturna (Voretigene neparvovec)
- For a rare inherited retinal disease (causes blindness).
- Helps restore vision by delivering a normal copy of the faulty gene.
- Roctavian (Valoctocogene roxaparvovec)
- For Hemophilia A.
- Gene therapy that helps the body produce factor VIII (blood clotting).
🔮 Future Trends in Genetic Medicines
- CRISPR-based therapies → rewriting faulty genes.
- mRNA therapies → beyond vaccines, used for cancer and rare diseases.
- Personalized Medicine → treatments based on your unique DNA.