Innostellar Biotherapeutics' Gene Therapy LX101 Injection Designated as Breakthrough Therapy for RPE65-Mediated Inherited Retinal Dystrophy
Innostellar Biotherapeutics
Innovative Gene Therapy Drug Research, Development, and Manufacturing
▎WuXi AppTec Content Team Report
On July 22, the latest announcement on the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) revealed,ByUnder Innostellar BiotherapeuticsInnostellar Biotherapeutics Co., Ltd. (Shanghai) submittedLX101 InjectionProposed for inclusion in the breakthrough therapy category,Indications for TreatmentRPE65Patients with biallelic mutation-associated inherited retinal dystrophy (IRD). Public information shows,LX101 InjectionIt is aGene therapy drugs using rAAV as a vector, theirTreatmentRPE65Mutation-Associated IRDHas entered the Phase 3 clinical trial stage.
Source of screenshot:CDE Official Website
LX101 is a product developed by Innostellar Biotherapeutics.Gene Therapy Drugs for Hereditary Retinal Degeneration. The drug is carried by adeno-associated virus type 2 (AAV2) with normalRPE65Genes can specifically recognize and transfect diseased retinal pigment epithelial cells, continuously and efficiently express the RPE65 protein that patients lack, restore normal visual cycle, and achieve the therapeutic goal of improving visual function.In the investigator-initiated clinical studies conducted previously, LX101 demonstrated good safety and has shown efficacy in improving vision in multiple patients.
Public information shows that Innostellar Biotherapeutics was founded in 2020, focusing on the research and manufacturing of innovative gene therapy drugs, with a particular emphasis on gene therapy for hereditary and chronic eye diseases. In June this year, Innostellar Biotherapeutics announced the completion ofSeries B+ Financing. The companyAdvanced 5 candidate molecules into clinical trials, and took the lead in conducting gene therapy clinical research in 3 indications of blinding eye diseases, obtaining 3 INDs. In addition to LX101, LX102 for treating neovascular age-related macular degeneration (nAMD) and LX103 for treating X-linked retinoschisis (XLRS) are also under research.
References:
[1]Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved July 22，2024, From https://www.cde.org.cn/main/xxgk/listpage/da6efd086c099b7fc949121166f0130c
[2] [Highlights] Innostellar Biotherapeutics Completes First Patient Dosing in Phase I Clinical Trial of LX101 Injection. Retrieved July 28，2022, From https://mp.weixin.qq.com/s/iHWqYslzY-N9SsPdEIv8hA
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