Top 5 Target	Count

LRRK2(Leucine-rich repeat serine/threonine-protein kinase 2)	1
SOD1(Superoxide dismutase)	1
BDCA2(C-Type lectin domain family 4 member C)	1
SMN2(Survival motor neuron protein)	1

Drugs associated with Biogen Biotechnology (Shanghai) Co. Ltd.

Tofersen

Target

SOD1

Mechanism

SOD1 gene inhibitors

Active Org.

Biogen Japan Ltd. [+5]

Originator Org.

Ionis Pharmaceuticals, Inc.

Active Indication

Amyotrophic Lateral Sclerosis [+1]

Inactive Indication-

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

United States

First Approval Date25 Apr 2023

Nusinersen sodium

Target

SMN2

Mechanism

SMN2 stimulants [+1]

Active Org.

Biogen, Inc. [+6]

Originator Org.

Ionis Pharmaceuticals, Inc.

Active Indication

Neuromuscular Diseases [+1]

Inactive Indication

HMN (Hereditary Motor Neuropathy) Proximal Type I

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

United States

First Approval Date23 Dec 2016

Litifilimab

Target

BDCA2

Mechanism

BDCA2 modulators

Active Org.

Biogen, Inc. [+2]

Originator Org.

Biogen, Inc.

Active Indication

Lupus Erythematosus, Cutaneous [+1]

Inactive Indication-

Drug Highest PhasePhase 3

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Biogen Biotechnology (Shanghai) Co. Ltd.

CTR20234203

/ RecruitingPhase 3

一项在患有活动性亚急性皮肤型红斑狼疮和/或慢性皮肤型红斑狼疮（有或无全身表现）且抗疟药治疗反应不佳和/或不耐受的受试者中评价 BIIB059 疗效和安全性的 2 部分无缝设计（A 部分 [II 期]/B 部分 [III 期]）、随机、双盲、安慰剂对照、多中心临床研究 (AMETHYST)

[Translation] A 2-part seamless design (Part A [Phase II]/Part B [Phase III]), randomized, double-blind, placebo-controlled, multicenter clinical study (AMETHYST) to evaluate the efficacy and safety of BIIB059 in subjects with active subacute cutaneous lupus erythematosus and/or chronic cutaneous lupus erythematosus (with or without systemic manifestations) who have an inadequate response and/or intolerance to antimalarial therapy

本研究的总体目的是在患有活动性 SCLE 和/或 CCLE（有或无全身表现）且抗疟药治疗反应不佳和/或不耐受的受试者中评价 BIIB059 与安慰剂相比的疗效和安全性。

[Translation]

The overall objective of this study was to evaluate the efficacy and safety of BIIB059 compared with placebo in subjects with active SCLE and/or CCLE (with or without systemic manifestations) who had an inadequate response to and/or intolerance to antimalarial therapy.

Start Date26 Jun 2024

Sponsor / Collaborator

Biogen Idec Research Ltd.

[+2]

CTR20223265

/ Active, not recruitingPhase 2

一项确定BIIB122治疗帕金森病受试者的有效性和安全性的IIb期、多中心、随机、双盲、安慰剂对照研究

[Translation] A Phase IIb, multicenter, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of BIIB122 in subjects with Parkinson's disease

主要目的: 评估BIIB122 225 mg与安慰剂相比的有效性次要目的: 评估BIIB122 225 mg与安慰剂相比的安全性和耐受性评估BIIB122 225 mg与安慰剂相比的有效性药代动力学: 表征BIIB122在血浆和CSF中的PK特征

[Translation]

Primary objective: To evaluate the efficacy of BIIB122 225 mg compared to placebo Secondary objectives: To evaluate the safety and tolerability of BIIB122 225 mg compared to placebo Pharmacokinetics: To characterize the PK profile of BIIB122 in plasma and CSF

Start Date27 Feb 2023

Sponsor / Collaborator

Biogen Idec Research Ltd.

[+2]

CTR20221451

/ CompletedPhase 3

一项旨在评价Diroximel Fumarate（BIIB098）用于亚太地区成年复发型多发性硬化受试者中的安全性和耐受性以及药代动力学的开放性、单臂、多中心、III期研究

[Translation] An open-label, single-arm, multicenter, Phase III study to evaluate the safety, tolerability, and pharmacokinetics of Diroximel Fumarate (BIIB098) in adult subjects with relapsing forms of multiple sclerosis in the Asia-Pacific region

第一部分：主要目的：旨在确定DRF治疗东亚RMS成人受试者长达24周的安全性和耐受性。次要目的：旨在评价对东亚RMS成人受试者子集多次给予DRF后DRF代谢产物（MMF和HES）的PK。第二部分主要目的：旨在确定DRF治疗东亚RMS成人受试者长达48周的安全性和耐受性。

[Translation]

Part I: Primary objective: To determine the safety and tolerability of DRF in East Asian adult subjects with RMS for up to 24 weeks. Secondary objective: To evaluate the PK of DRF metabolites (MMF and HES) after multiple administrations of DRF in a subset of East Asian adult subjects with RMS. Part II Primary objective: To determine the safety and tolerability of DRF in East Asian adult subjects with RMS for up to 48 weeks.

Start Date22 Jul 2022

Sponsor / Collaborator

Alkermes Pharma Ireland Ltd.

[+2]

100 Clinical Results associated with Biogen Biotechnology (Shanghai) Co. Ltd.

0 Patents (Medical) associated with Biogen Biotechnology (Shanghai) Co. Ltd.

Literatures (Medical) associated with Biogen Biotechnology (Shanghai) Co. Ltd.

28 Mar 2025·Medicine

Research trends on spinal muscular atrophy from 1995 to 2023: A bibliometric analysis

Article

Author: Yu, Hao ; Xia, Yanyan ; Wei, Cuijie ; Sun, Dan ; Zhu, Wenhua ; Zhang, Li

Background:Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive muscle weakness due to motor neuron degeneration. The discovery of the survival motor neuron 1 (SMN1) gene in 1995 revolutionized SMA research, leading to significant therapeutic advancements. This bibliometric analysis aimed to explore global trends in SMA research and therapy, with a particular focus on China.Methods:A comprehensive database search identified 4506 relevant publications (3812 articles, 694 reviews) published between 1995 and 2023. Bibliometric tools were used to analyze publication trends, collaborations, and research topics.Results:SMA research has experienced substantial growth, with the United States leading in publications followed by the United Kingdom and Germany. China has shown increasing engagement in this field. Key research areas include genetic and molecular mechanisms, survival motor neuron gene therapy, antisense oligonucleotides, and muscle strength-promoting factors. Chinese researchers have contributed significantly to these areas, with a higher reporting frequency of SMA-related topics compared to other countries.Conclusion:This bibliometric analysis provides a comprehensive overview of global SMA research, highlighting significant advancements, and identifying future directions. The findings offer valuable insights for researchers, clinicians, and policymakers in China to ensure alignment with global medical advancements and improve the lives of individuals affected by SMA.

01 Aug 2024·Journal of Neurology

Nusinersen effectiveness and safety in pediatric patients with 5q-spinal muscular atrophy: a multi-center disease registry in China

Article

Author: Mao, Shanshan ; Zhang, Li ; Wang, Xiuxia ; Sun, Dan ; Xia, Yanyan ; Peng, Jing ; Wang, Hua ; Jin, Ruifeng ; Hu, Jun ; Hong, Siqi ; Chen, Ken ; Tao, Zhe ; Zhang, Xiaoli ; Luo, Rong ; Lu, Xinguo ; Wang, Yi ; Yang, Lin ; Wu, Liwen ; Liang, Jianmin ; Yao, Xiaoli ; Zhong, Jianmin

AbstractObjectiveTo evaluate the effectiveness and safety of nusinersen for the treatment of 5q-spinal muscular atrophy (SMA) among Chinese pediatric patients.MethodsUsing a longitudinal, multi-center registry, both prospective and retrospective data were collected from pediatric patients with 5q-SMA receiving nusinersen treatment across 18 centers in China. All patients fulfilling the eligibility criteria were included consecutively. Motor function outcomes were assessed post-treatment by SMA type. Safety profile was evaluated among patients starting nusinersen treatment post-enrollment. Descriptive analyses were used to report baseline characteristics, effectiveness, and safety results.ResultsAs of March 2nd, 2023, 385 patients were included. Most patients demonstrated improvements or stability in motor function across all SMA types. Type II patients demonstrated mean changes [95% confidence interval (CI)] of 4.4 (3.4–5.4) and 4.1 (2.8–5.4) in Hammersmith Functional Motor Scale-Expanded (HFMSE), and 2.4 (1.7–3.1) and 2.3 (1.2–3.4) in Revised Upper Limb Module (RULM) scores at months 6 and 10. Type III patients exhibited mean changes (95% CI) of 3.9 (2.5–5.3) and 4.3 (2.6–6.0) in HFMSE, and 2.1 (1.2–3.0) and 1.5 (0.0–3.0) in RULM scores at months 6 and 10. Of the 132 patients, 62.9% experienced adverse events (AEs). Two patients experienced mild AEs (aseptic meningitis and myalgia) considered to be related to nusinersen by the investigator, with no sequelae.ConclusionsThese data underscore the significance of nusinersen in Chinese pediatric patients with SMA regarding motor function improvement or stability, and support recommendations on nusinersen treatment by Chinese SMA guidelines and continuous coverage of nusinersen by basic medical insurance.

BMC Pediatrics

Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China

Article

Author: Mao, Shanshan ; Zhang, Li ; Sun, Dan ; Wang, Xiuxia ; Peng, Jing ; Xia, Yanyan ; Tao, Zhe ; Wang, Hua ; Jin, Ruifeng ; Zhong, Jianmin ; Hong, Siqi ; Chen, Ken ; Hu, Jun ; Zhang, Xiaoli ; Luo, Rong ; Wang, Yi ; Yang, Lin ; Lu, Xinguo ; Yao, Xiaoli ; Liang, Jianmin ; Wu, Liwen

AbstractBackgroundNusinersen was the first approved disease modifying therapy (DMT) for spinal muscular atrophy (SMA). Intrathecal administration of nusinersen enables drug delivery directly to the central nervous system, where the motor neurons are located. Per the package insert, individuals with SMA receive 4 loading doses of nusinersen followed by maintenance doses every 4 months thereafter. The aim of this analysis was to investigate the administration practices of and adherence to nusinersen in Chinese children with SMA.MethodsData were analyzed from a longitudinal, multicenter registry enrolling children with 5q-SMA in China. Information on nusinersen administration, including administration date, care setting, use of sedation and general anesthesia, method of administration, and use of imaging guidance before administration, was collected both retrospectively and prospectively. Adherence rate was calculated at dose and participant level. A dose was considered adherent if the inter-dose interval (for dose-level) and interval from the first dose (for participant-level) followed the standard dosing regimen, with a grace period of ± 7 days for Dose 2 to 4 and ± 28 days thereafter.ResultsA total of 385 participants receiving nusinersen with a total of 2,415 doses were included in the study. The median (interquartile range) number of doses administered per participant was 6 (5–7). Over 99% of intrathecal injections were given in an inpatient setting. Only a few (n = 3, 0.1%) required general anesthesia, while 9% (n = 217) of doses were administered under the use of sedation. Interlaminar lumbar puncture (n = 2,407, 99.7%) was the most common method of administration, followed by cervical puncture (n = 5, 0.2%) and transforaminal lumbar puncture (n = 3, 0.1%). Over 90% of injections did not utilize any imaging guidance prior to administration, with ultrasound (n = 142, 5.9%) being the most commonly used imaging guidance. The adherence rate was 95.7% (1,943/2,030) at dose level and 81.0% (312/385) at participant level. The median inter-dose intervals aligned well with the dosing schedule, with 14 days for Doses 2 and 3, 35 days for Dose 4, and 114–124 days for maintenance doses thereafter.ConclusionsFindings from the analysis demonstrated high real-world adherence to nusinersen in Chinese children with SMA.

100 Deals associated with Biogen Biotechnology (Shanghai) Co. Ltd.

100 Translational Medicine associated with Biogen Biotechnology (Shanghai) Co. Ltd.

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Pipeline

Pipeline Snapshot as of 11 May 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

Phase 2 Clinical

Phase 3 Clinical

Approved

Current Projects

Drug(Targets)	Indications	Global Highest Phase

Tofersen ( SOD1 )	Amyotrophic Lateral Sclerosis More	Approved
Litifilimab ( BDCA2 )	Lupus Erythematosus, Cutaneous More	Phase 3
Nusinersen sodium ( SMN2 )	Spinal Muscular Atrophy More	Phase 3
DNL-151 ( LRRK2 )	Parkinson Disease More	Phase 2

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