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Certainly! **Pfizer** has been actively advancing its gene therapy pipeline, particularly in the field of hemophilia. Here are some key developments:

1. **Giroctocogene fitelparvovec**:
- Pfizer announced positive topline results from the **Phase 3 AFFINE study** evaluating giroctocogene fitelparvovec. This investigational gene therapy targets **hemophilia A** in adults with moderately severe to severe forms of the condition.
- Key findings:
- Achieved its primary objective of **non-inferiority** and **superiority** compared to routine Factor VIII (FVIII) replacement prophylaxis treatment.
- Demonstrated a statistically significant reduction in mean total annualized bleeding rate (ABR) compared to the pre-infusion period.
- Most participants maintained FVIII activity >5% at 15 months post-infusion.
- Generally well-tolerated, with no impact on efficacy and safety results¹.
- Notably, Pfizer also received FDA approval for **Beqvez**, a gene therapy for **hemophilia B**.
- Additionally, regulatory submissions for **marstacimab** as a potential treatment for both hemophilia A and B are underway in the U.S. and Europe².

2. **Other Gene Therapy Programs**:
- Pfizer currently has three Phase 3 programs investigating gene therapy in populations with high unmet needs:
- **Hemophilia B**
- **Hemophilia A**
- **Duchenne muscular dystrophy**³.

Pfizer's commitment to advancing gene therapies holds great promise for patients worldwide. 🌟🧬⁴⁵ 🚀

Source: Conversation with Copilot, 7/30/2024
(1) Pfizer Announces Positive Topline Results From Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-results-phase-3-study.
(2) Pfizer Hemophilia Gene Therapy Meets Phase 3 Goals, But Its Success .... medcitynews.com/2024/07/pfizer-gene-therapy-hemophilia-a-sangamo-pfe-sgmo/.
(3) Pfizer Announces Positive Top-Line Results from Phase 3 Study of .... pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-top-line-results-phase-3-study.
(4) Pfizer Pipeline. cdn.pfizer.com/pfizercom/product-pipeline/Pipeline_Update_02NOV2021.pdf?xv5ox6MFA62A.LANHa95hryf4ouB.n0s_slide_15.
(5) New Drug Development Pipeline: Pfizer's Medicine, Vaccine Discovery .... pfizer.com/science/drug-product-pipeline.

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