Delving into the Latest Updates on Suzhou Nuojiebei Biotechnology Co., Ltd. with Synapse

Overview

Tags

Congenital Disorders

Other Diseases

Endocrinology and Metabolic Disease

AAV based gene therapy

Biological products

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Endocrinology and Metabolic Disease	3
Nervous System Diseases	2
Hemic and Lymphatic Diseases	1
Immune System Diseases	1

Top 5 Drug Type	Count

AAV based gene therapy	6
Biological products	1

Top 5 Target	Count

F8(Coagulation factor VIII)	1
LDLR(LDL receptor)	1
PAH(Phenylalanine hydroxylase)	1
CYP4V2(cytochrome P450 family 4 subfamily V member 2)	1
KLKB1(Plasma kallikrein)	1

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.

Start Date10 Jan 2025

Sponsor / Collaborator

Suzhou Nuojiebei Biotechnology Co., Ltd.

NCT06687733

/ RecruitingPhase 1/2

A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Classic Phenylketonuria

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.

Start Date25 Jul 2024

Sponsor / Collaborator

Suzhou Nuojiebei Biotechnology Co., Ltd.

CTR20241545

/ RecruitingPhase 1/2

NGGT002静脉滴注治疗成人苯丙酮尿症的安全性和有效性的 I/II期临床研究

[Translation] Phase I/II clinical study on the safety and efficacy of intravenous infusion of NGGT002 in the treatment of phenylketonuria in adults

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。

[Translation]

The primary objective is to evaluate the safety and tolerability of NGGT002 injection in adult patients with phenylketonuria (PKU). To evaluate the effectiveness of NGGT002 injection in reducing plasma phenylalanine (Phe) concentrations in adult patients with phenylketonuria. The secondary objectives are to monitor changes in PKU disease in terms of dietary liberalization, neurological and psychological symptoms, and quality of life of patients. To evaluate the pharmacokinetic (PK), carrier shedding, and immunogenicity characteristics of NGGT002 in humans after treatment.

Start Date11 Jul 2024

Sponsor / Collaborator

Suzhou Nuojiebei Biotechnology Co., Ltd.

100 Clinical Results associated with Suzhou Nuojiebei Biotechnology Co., Ltd.

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0 Patents (Medical) associated with Suzhou Nuojiebei Biotechnology Co., Ltd.

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100 Deals associated with Suzhou Nuojiebei Biotechnology Co., Ltd.

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100 Translational Medicine associated with Suzhou Nuojiebei Biotechnology Co., Ltd.

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Pipeline

Pipeline Snapshot as of 21 Mar 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Discovery

2

1

Preclinical

Early Phase 1

1

3

Phase 2

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

NGGT-001 ( CYP4V2 )	Bietti Crystalline Dystrophy More	Phase 1/2
NGGT-002 ( PAH )	Phenylketonurias More	Phase 1/2
NGGT007	Wet age-related macular degeneration More	Phase 1/2
NGGT-003 ( F8 )	Hemophilia A More	Early Phase 1
AAV8-DX88-HSA (NGGT) ( KLKB1 )	Hereditary Angioedema More	Preclinical