Sangamo Therapeutics

Pfizer's Hympavzi, a once-a-week injection, received FDA approval for treating haemophilia A or B in patients aged 12 and older. The drug aims to reduce treatment burden and showed a 92% reduction in bleeding episodes in a late-stage study. Hympavzi is Pfizer's second haemophilia treatment approved this year, with potential sales of $300 million by 2030.

DelveInsight's 2024 Alzheimer's Disease Pipeline Insight report details 110+ companies developing 120+ therapies, including Neurodon, Sangamo Therapeutics, and others. Key therapies like NRDN-201, ST-501, and Blarcamesine are in various clinical phases. Regulatory milestones include FDA's conventional approval of Leqembi in 2023. The report covers drug mechanisms, clinical studies, and market dynamics.

Gene editing therapeutics market estimated at $11 million in 2024, forecasted to grow at 147% CAGR to reach $1 billion by 2029, driven by chronic diseases, rare disorders, strategic initiatives, and precision medicine.

Pfizer announces positive Phase 3 results for hemophilia A gene therapy candidate giroctocogene fitelparvovec, showing significant reduction in annualized bleeding rate post-infusion and generally well-tolerated profile.