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RNAi Therapeutic Top 20 2024

Top 20 RNAi Therapeutic Companies of 2024

Shots:  RNAi therapeutics are innovative medications that enable rapid identification of specific inhibitors of disease targets across a wide range of molecule classes. Several biopharma companies are investing in RNAi therapeutics extravagantly to maintain a healthy foothold in innovative medicines  In 2023, the global RNAi drug delivery market size was valued at $84.1B and is…

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Novartis

Novartis Reports the CHMP’s Positive Opinion for Fabhalta (iptacopan) to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH)

Shots:  The opinion was based on the P-III (APPLY-PNH) & (APPOINT-PNH) trials of Fabhalta (200mg, oral, BID) in PNH patients with residual anemia who switched from Anti-C5 and complement inhibitor naïve (incl. anti-C5 therapies) respectively for 24wks.  In the APPLY-PNH study, 82.3% vs 2.0% had a ≥2 g/dL increase in Hb levels without transfusions while…

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