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

Overview

Tags

Other Diseases

Congenital Disorders

Endocrinology and Metabolic Disease

AAV based gene therapy

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

Top 5 Target	Count

LDLR(LDL receptor)	1
KLKB1(Plasma kallikrein)	1
F8(Coagulation factor VIII)	1
CYP4V2(cytochrome P450 family 4 subfamily V member 2)	1
PAH(Phenylalanine hydroxylase)	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 Date22 Nov 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.

CTR20240103 / RecruitingPhase 1/2

NGGT001视网膜下腔注射治疗结晶样视网膜变性的I/II期临床研究

[Translation] Phase I/II clinical study of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration

主要目的评价NGGT001视网膜下腔注射治疗结晶样视网膜变性的安全性和耐受性，推荐后期临床研究的给药剂量。初步探索NGGT001视网膜下腔注射治疗结晶样视网膜变性的有效性。次要目的评价NGGT001视网膜下腔注射后的药物代谢特性。评价NGGT001视网膜下腔注射后机体的免疫反应。

[Translation]

The primary objective is to evaluate the safety and tolerability of NGGT001 subretinal injection in the treatment of crystalline retinal degeneration and recommend the dosage for later clinical studies. To preliminarily explore the effectiveness of NGGT001 subretinal injection in the treatment of crystalline retinal degeneration. The secondary objective is to evaluate the drug metabolism characteristics of NGGT001 after subretinal injection. To evaluate the body's immune response after subretinal injection of NGGT001.

Start Date21 Mar 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 23 Dec 2024

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

IND Approval

1

Phase 1 Clinical

Phase 2 Clinical

2

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

NGGT-001 ( CYP4V2 )	Retinal Degeneration More	Phase 2 Clinical
NGGT-002 ( PAH )	Phenylketonurias More	Phase 2 Clinical
NGGT-003 ( F8 )	Hemophilia A More	Phase 1 Clinical
NGGT007	Wet age-related macular degeneration More	IND Approval
AAV8-DX88-HSA (NGGT) ( KLKB1 )	Hereditary Angioedema More	Preclinical