Prevail Therapeutics and Lonza Enter Strategic Collaboration

优势治疗与龙沙(Lonza)达成战略合作,专注神经退行性疾病治疗

2019-10-09 13:31:16 Investing News Network

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Through the collaboration, the companies will focus on Prevail’s gene therapy programs for patients with neurodegenerative diseases. Prevail Therapeutics (NASDAQ:PRVL)P and Lonza (SWX:LONN) have announced a strategic collaboration to develop Prevail’s gene therapy program for neurodegenerative diseases. As quoted in the press release: Lonza and Prevail have been working together since 2018, with an initial focus on process development, towards the GMP manufacturing of Prevail’s two lead programs, PR001 and PR006. Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail’s pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases at its gene therapy center of excellence in Houston, TX. “Prevail is a brilliant trailblazer and it is a privilege to partner with them,” said Alberto Santagostino, SVP Head of Cell & Gene Technologies, Lonza Pharma & Biotech. “This strategic collaboration reflects both companies’ commitment to fueling the innovation of novel medicines. The partnership combines Prevail’s frontier science with Lonza’s operational expertise to support the development and commercialization of potentially life-changing treatments. Our cell and gene therapy center of excellence in Houston, TX, will support Prevail in their journey as they develop and potentially bring to market AAV-based gene therapies for patients suffering from debilitating neurodegenerative diseases.” Prevail’s pipeline includes PR001, an AAV9-based gene therapy delivering GBA1, in development for Parkinson’s disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease patients (nGD). Prevail plans to initiate a Phase 1/2 clinical trial of PR001 in PD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN mutation (FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration also has the potential to extend to Prevail’s future pipeline of AAV-based gene therapy programs. Click here to read the full press release.
通过合作,两家公司将专注于 Prefail 的神经退行性疾病患者基因治疗项目。 Preference Therapeutics ( NASDAQ : PRVL ) P 和龙沙(Lonza)( SWX : LONN )宣布了一项战略合作,以开发 Preavi 的神经退行性疾病基因治疗计划。 如新闻稿所述: 龙沙(Lonza)和 Preofi 自2018年开始合作,最初专注于工艺开发,致力于 Prefami 两大主导项目 PR001和 PR006的 GMP 制造。在此合作下,龙沙(Lonza)将致力于基因治疗的 Baculove / Sf9生产系统,在其位于德克萨斯州休斯顿的基因治疗中心为神经退行性疾病患者提供新型 AAV 基因治疗方案。 龙沙(Lonza) Pharma & Biotech 公司 Cell & Gene Technologies 高级副总裁 Alberto Santagonino 表示:“ Prefail 是一款杰出的开拓者,与他们合作是一种荣幸。”“这种战略合作反映出两家公司致力于推动新药创新。这一合作关系将 Preference 的前沿科学与龙沙(Lonza)的运营专长相结合,以支持潜在改变生命的治疗方法的开发和商业化。我们位于德克萨斯州休斯顿的细胞和基因治疗中心,将在他们的发展过程中为 Prefail 提供支持,并可能为患有神经退行性疾病的患者提供基于 AAV 的基因治疗。” Prefail 的管道包括 PR001,一种基于 AAV9的基因疗法,提供 GBA1,用于帕金森病患者的 GBA1突变( PD-GBA )和神经病高歇病患者( nGD )。Preference 计划于2019年在 PD-GBA 启动 PR001的1/2期临床试验。该公司还开发了 PR006,一种基于 AAV9的基因疗法,提供 GRN ,用于额颞痴呆患者的 GRN 突变( FTD-GRN )。普列夫预计 PR006将于2020年进入临床。这一合作也有可能扩展到 Prefei 未来的基于 AAV 的基因治疗项目。 单击此处阅读完整的新闻稿。

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