Methods of treating ocular diseases using AAV2 variants encoding aflibercept

• Main Authors: WHITEPOOL STEVEN, TEPKU, ADITYA, CLARKE JONATHAN
• Format: Patent
• Language: Chinese; English
• Published: 12.12.2023
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

Provided is a method for treating an ocular disease in a subject, comprising administering to the eye of the subject a unit dose of a recombinant adeno-associated virus (rAAV) particle, where the rAAV particle comprises: a) a nucleic acid encoding a polypeptide and flanking an AAV2 inverted terminal repeat (ITR), the polypeptide comprising an amino acid sequence having at least about 95% identity to the amino acid sequence of SEQ ID NO: 35, and b) an AAV2 capsid protein comprising the amino acid sequence LGETTRP (SEQ ID NO: 14) inserted between positions 587 and 588 of the capsid protein wherein the amino acid residue number corresponds to the AAV2 VP1 capsid protein. 提供用于治疗个体中的眼部疾病的方法，其包括向所述个体的眼睛施用单位剂量的重组腺相关病毒(rAAV)颗粒，其中所述rAAV颗粒包含：a)编码多肽且侧接AAV2反向末端重复序列(ITR)的核酸，所述多肽包含与SEQ ID NO:35的氨基酸序列具有至少约95％同一性的氨基酸序列，和b)AAV2衣壳蛋白，其包含插入所述衣壳蛋白的位置587和588之间的氨基酸序列LGETTRP(SEQ ID NO:14)，其中所述氨基酸残基编号对应于AAV2VP1衣壳蛋白。