Suzhou Nuojiebei Biotechnology Co., Ltd.

This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

主要目的评估NGGT002注射液在成人苯丙酮尿症（PKU）患者中的安全性和耐受性。评估NGGT002注射液在成人苯丙酮尿症患者中降低血浆苯丙氨酸（Phe）浓度的有效性。 次要目的监测PKU疾病在饮食自由化、神经和心理症状以及患者生活质量方面的变化。评价治疗后，NGGT002在人体内的药代动力学(PK)、载体脱落和免疫原性特征。