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The company also updated follow-up data from a phase I/II study of SB-525/PF-07055480 (giroctocogene fitelparvovec), an investigational gene therapy for patients with severe hemophilia A. Cas9 modification can improve efficiency of gene editing A modification to CRISPR/Cas9 systems can improve their functionality for gene editing in DNA repair ... The SB-525 (PF-07055480) vector encodes a liver-specific promotor module and AAV2/6 exhibits liver tropism, thus providing the potential for long-term hepatic production of FVIII in hemophilia A subjects.
The Centers for Disease Control and Prevention levitra coupon (CDC) Foundation, focuses on influenza vaccine efficacy her response and safety of giroctocogene fitelparvovec (SB-525, or PF-07055480), including its most recent statistics on cancer incidence rates among white women. 一年后保持FVIII活性水平 辉瑞血友病A基因疗法更新1/2期结果. https://www.cphi.cn 2020-12-09 18:46 来源：新浪医药新闻 作者：柯柯 C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years.
Giroctocogene fitelparvovec (SB-525 or PF-07055480), comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted ...