更新于：2024-11-21

OTOF-GT gene therapy(Sensorion SAS)

更新于：2024-11-21

概要

研发状态

概要

基本信息

药物类型

腺相关病毒基因治疗

别名

A dual vector gene therapy product containing AAV8 vectors expressing the 5¿ and 3¿ portion of human otoferlin (OTOF) gene、Adeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence、OTOF-GT

+ [1]

靶点

OTOF

作用机制

OTOF调节剂(otoferlin modulators)、基因转移

治疗领域

神经系统疾病耳鼻咽喉疾病

在研适应症

耳聋神经性听力损失听力障碍+ [1]

非在研适应症-

原研机构

Sensorion SA

在研机构

Sensorion SA

非在研机构-

最高研发阶段临床1/2期

首次获批日期-

最高研发阶段(中国)-

特殊审评罕见儿科疾病 (美国)、孤儿药 (欧盟)

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关联

项与 OTOF-GT gene therapy(Sensorion SAS) 相关的临床试验

NCT06370351 / Recruiting临床1/2期

A Phase I/II, Open-ended, Adaptative, Open Label Dose Escalation and Expansion Clinical Trial to Evaluate the Efficacy and Safety of Unilateral Intracochlear Injection of SENS-501 Using an Injection System in Children with Severe to Profound Hearing Loss Due to Otoferlin Gene Mutations

This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.

开始日期2024-06-21

申办/合作机构

Sensorion SA

100 项与 OTOF-GT gene therapy(Sensorion SAS) 相关的临床结果

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100 项与 OTOF-GT gene therapy(Sensorion SAS) 相关的转化医学

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100 项与 OTOF-GT gene therapy(Sensorion SAS) 相关的专利（医药）

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项与 OTOF-GT gene therapy(Sensorion SAS) 相关的文献（医药）

2024-03-01·Advanced Science

AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness

Article

作者: Tan, Chang ; Zhou, Yinyi ; Zhang, Ziyu ; Chai, Renjie ; Liu, Jiancheng ; Shu, Yilai ; Qi, Jieyu ; Yang, Xuehan ; Zhang, Shanzhong ; Chen, Tian ; Tan, Fangzhi ; Li, Huawei ; Zhang, Liyan ; Lu, Yicheng ; Gao, Xia ; Jiang, Lulu ; Yu, Chaorong ; Xu, Lei ; Sun, Sijie ; Song, Huaien ; Dong, WenXiu ; Wu, Lianqiu ; Lu, Ling ; Qian, Xiaoyun ; Zhai, Yabo

AbstractMutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is extensively validated in Otof‐deficient mice. However, the clinical safety and efficacy of AAV‐OTOF is not reported. Here, AAV‐OTOF is generated using good manufacturing practice and validated its efficacy and safety in mouse and non‐human primates in order to determine the optimal injection dose, volume, and administration route for clinical trials. Subsequently, AAV‐OTOF is delivered into one cochlea of a 5‐year‐old deaf patient and into the bilateral cochleae of an 8‐year‐old deaf patient with OTOF mutations. Obvious hearing improvement is detected by the auditory brainstem response (ABR) and the pure‐tone audiometry (PTA) in these two patients. Hearing in the injected ear of the 5‐year‐old patient can be restored to the normal range at 1 month after AAV‐OTOF injection, while the 8‐year‐old patient can hear the conversational sounds. Most importantly, the 5‐year‐old patient can hear and recognize speech only through the AAV‐OTOF‐injected ear. This study is the first to demonstrate the safety and efficacy of AAV‐OTOF in patients, expands and optimizes current OTOF‐related gene therapy and provides valuable information for further application of gene therapies for deafness.

2024-01-01·Advanced Science

Preclinical Efficacy And Safety Evaluation of AAV‐OTOF in DFNB9 Mouse Model And Nonhuman Primate

Article

作者: Zhang, Yang ; Tan, Fangzhi ; Yu, Chaorong ; Sun, Shan ; Liu, Jiancheng ; Qi, Jieyu ; Zhang, Shanzhong ; Zhang, Liyan ; Chen, Tian ; Zhou, Yinyi ; Lu, Ling ; Zhang, Ziyu ; Sun, Sijie ; Jiang, Lulu ; Wang, Qiuju ; Wang, Hongyang ; Wu, Lianqiu ; Chai, Renjie

AbstractOTOF mutations are the principal causes of auditory neuropathy. There are reports on Otof‐related gene therapy in mice, but there is no preclinical research on the drug evaluations. Here, Anc80L65 and the mouse hair cell‐specific Myo15 promoter (mMyo15) are used to selectively and effectively deliver human OTOF to hair cells in mice and nonhuman primates to evaluate the efficacy and safety of OTOF gene therapy drugs. A new dual‐AAV‐OTOF‐hybrid strategy to transfer full‐length OTOF is generated, which can stably restore hearing in adult OTOFp.Q939*/Q939* mice with profound deafness, with the longest duration being at least 150 days, and the best therapeutic effect without difference in hearing from wild‐type mice. An AAV microinjection method into the cochlea of cynomolgus monkeys without hearing impairment is further established and found the OTOF can be safely and effectively driven by the mMyo15 promoter in hair cells. In addition, the therapeutic dose of AAV drugs has no impact on normal hearing and does not cause significant systemic toxicity both in mouse and nonhuman primates. In summary, this study develops a potential gene therapy strategy for DFNB9 patients in the clinic and provides complete, standardized, and systematic research data for clinical research and application.

项与 OTOF-GT gene therapy(Sensorion SAS) 相关的新闻（医药）

2024-11-06

·Business Wire

Sensorion Announces Its Participation in Upcoming Investor Conferences to be Held in November 2024

MONTPELLIER, France--( BUSINESS WIRE )--Regulatory News: Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announced its participation in upcoming investor conferences to take place in November 2024. Sensorion will participate in the Stifel Healthcare Conference , which will be held in New York, USA, on November 18-19, 2024. Sensorion's management team will be present for one-to-one meetings on November 18, 2024. Sensorion will also participate in the LifeSci Capital and Sofinnova Partners Growth & Innovation Summit, to take place in London, UK, on November 18, 2024. Sensorion's management team will be present for one-to-one meetings throughout the conference. Sensorion will also be attending the Jefferies London Healthcare Conference, in London, UK, on November 19-21, 2024. Sensorion's management team will be present for one-to-one meetings throughout the conference. If you would like to meet Sensorion's management team, please contact us by email: ir.contact@sensorion-pharma.com About Sensorion Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion’s portfolio also comprises clinical-stage small molecule programs for the treatment and prevention of hearing loss disorders. Sensorion’s clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, has completed in a study of SENS-401 in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022. www.sensorion.com Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN Disclaimer This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2023 full year report published on March 14, 2024, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

临床2期

2024-09-26

·Business Wire

Sensorion Announces Webconference for Retail Shareholders on October 10, 2024

MONTPELLIER, France--( BUSINESS WIRE )--Regulatory News: Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announces it is hosting a webconference for its retail shareholders on October 10, 2024. The webconference will be held in French. Sensorion’s Chief Executive Officer, Nawal Ouzren, and management team, will host a webconference dedicated to its retail shareholders, in French, on October 10, 2024, at 6pm CET (12pm ET), to comment on the Company’s latest announcements and outlook. Click here to join the meeting Meeting ID: 312 282 544 045 Secret Code: bfjH3J You will also be able to join the webconference from Sensorion’s website . About Sensorion Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT, in preclinical stage, targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. Sensorion’s portfolio also comprises clinical-stage small molecule programs for the treatment and prevention of hearing loss disorders. Sensorion’s clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, has completed in a study of SENS-401 in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022. www.sensorion.com Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN Disclaimer This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2023 full year report published on March 14, 2024, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.

临床2期基因疗法

2024-09-23

·Endpoints

Context makes a T cell engager deal; Ginkgo Bioworks backers have a new SPAC

Plus, news about Longevity Biomedical, Sensorion, Teva and Bristol Myers Squibb: Context Therapeutics buys a T cell engager: The biotech will pay $15 million upfront and up to $118.5 million in biobucks for BioAtla’s Nectin-4 x CD3 T cell engager for solid tumors. Companies developing T cell engagers, or TCEs, have received more interest in recent months , particularly as they have started to test out the therapies in autoimmune diseases . — Kyle LaHucik Ginkgo Bioworks creators are back with another SPAC: The executives behind the blank check company that took Ginkgo Bioworks public have returned with plans for a new $250 million SPAC called Bold Eagle Acquisition. Ginkgo $DNA was valued at about $15 billion when it went public in September 2021. It’s now valued at $445 million. Bold Eagle’s leaders are serial SPAC creators and have been involved in several blank check combinations outside of biopharma including with Lionsgate Studios, Skillz, DraftKings and Target Hospitality. — Kyle LaHucik Longevity Biomedical also inks SPAC deal: The therapeutics and digital health company plans to combine with FutureTech II Acquisition and trade on the Nasdaq as $LBIO . Longevity’s pipeline includes a device for ischemic stroke, tissue biomaterial for soft-tissue reconstruction and other programs. — Kyle LaHucik Sensorion touts data from hearing loss pipeline: The French biotech said the first child given its gene therapy, SENS-501, in a Phase 1/2 trial tolerated the treatment well and had no adverse events. Meanwhile, data from a Phase 2 test of its small molecule drug, SENS-401, after seven weeks of treatment showed that 40% of patients had complete hearing preservation. — Ayisha Sharma Teva unveils Phase 3 schizophrenia results: The Tel Aviv-based company’s long-acting injectable, olanzapine, achieved “significant improvements” in the primary endpoint of the Positive and Negative Syndrome Scale (PANSS) total score at eight weeks. Teva is testing a subcutaneous form of its intramuscular injection. The safety profile was also consistent with oral formulations of olanzapine. — Ayisha Sharma Bristol Myers Squibb to lay off 79 workers in New Jersey: According to a Worker Adjustment and Retraining Notification notice , BMS plans to let go 79 employees in Lawrenceville, NJ, between December and January. BMS did not respond to a request for comment. The drugmaker has conducted several rounds of layoffs this year, including previous layoffs in Lawrenceville and letting go of 252 employees from a Mirati Therapeutics site in San Diego in March. — Katherine Lewin

临床2期并购临床3期临床结果基因疗法

100 项与 OTOF-GT gene therapy(Sensorion SAS) 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
耳聋	临床2期	法国	Sensorion SA	2024-06-21
耳聋	临床2期	澳大利亚	Sensorion SA	2024-06-21
神经性听力损失	临床2期	法国	Sensorion SA	2024-06-21
神经性听力损失	临床2期	澳大利亚	Sensorion SA	2024-06-21
听力障碍	临床1期	-	Sensorion SA	-
听力损失	临床申请	德国	Sensorion SA	2023-07-19
听力损失	临床申请	意大利	Sensorion SA	2023-07-19

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06370351 (BusinessWire) 人工标引	临床1/2期	耳聋	1	SENS-501	(鹹繭積構衊醖廠窪衊願) = The surgery was well tolerated by the patient and no safety signals were reported. 衊鏇艱積築範遞醖獵顧 (築廠淵構獵網醖鹽遞夢 )	积极	2024-09-20