2024年十大畅销基因疗法，仅3款销售额超1亿美元

Core Viewpoint - The current gene therapy market is characterized by both significant investment opportunities and notable challenges, reflecting a duality in its development landscape [2][3]. Investment Highlights - As of mid-April 2023, five gene therapy companies have collectively secured $534.4 million in venture capital funding, indicating strong investor interest in the sector [2]. - Notable funding rounds include Tune Therapeutics raising over $175 million in January, and Atsena Therapeutics completing a $150 million oversubscribed round in April [2]. Market Challenges - AmplifyBio, a CRO/CDMO focused on gene and cell therapies, shut down in April 2023 after four years of operation, highlighting funding shortages faced by early-stage biotech companies [3]. - Despite over ten gene therapies receiving FDA approval, only one is projected to exceed $1 billion in sales in 2024, with only three therapies expected to surpass $100 million in sales [3]. Top Gene Therapies by Projected Sales 1. Zolgensma®: Projected sales of $1.214 billion in 2024, developed by Novartis, for treating spinal muscular atrophy in children under 2 years old [12]. 2. Elevidys®: Projected sales of $820.791 million in 2024, developed by Sarepta Therapeutics, for treating Duchenne muscular dystrophy [13]. 3. Vyjuvek®: Projected sales of $290.5 million in 2024, developed by Krystal Biotech, for treating epidermolysis bullosa [13]. 4. Adstiladrin®: Projected sales of €70 million ($79 million) in 2024, developed by Ferring Pharmaceuticals, for treating high-risk non-muscle invasive bladder cancer [14]. 5. Zynteglo™: Projected sales of $62.273 million in 2024, developed by bluebird bio, for treating beta-thalassemia [14]. 6. Roctavian®: Projected sales of $26 million in 2024, developed by BioMarin Pharmaceutical, for treating severe hemophilia A [14]. 7. Lenmeldy™ / Libmeldy™: Projected sales of $224.54 million in 2024, developed by Orchard Therapeutics, for treating metachromatic leukodystrophy [14]. 8. Luxturna®: Projected sales of CHF 18 million ($20.9 million) in 2024, developed by Spark Therapeutics, for treating RPE65 mutation-associated retinal dystrophy [14]. 9. Lyfgenia®: Projected sales of $11.605 million in 2024, developed by bluebird bio, for treating sickle cell disease [16]. 10. Skysona®: Projected sales of $9.917 million in 2024, developed by bluebird bio, for treating cerebral adrenoleukodystrophy [16].