[細胞・遺伝子治療] LonzaがNoga Therapeutics と、Cocoonシステムによる自家レンチウイルス遺伝子治療開発を提携

・Noga TherapeuticsとLonzaのコラボレーションでは、新たな一次免疫不全（PID）治療プログラムに革新的なCocoon^® システムを利用
・Noga Therapeutics の治療薬は、PID患者を対象としたヒト初回投与試験(FIH 試験)に向け前臨床モデルで使用される
・Cocoon^® システムー個別患者用治療薬の自動製造プラットフォームは、前臨床規模の細胞治療製品から商業生産へのスケールアップまでエンド・ツー・エンドの製造が可能

Quote from Eytan Abraham, Head of Personalized Medicine at Lonza:
“We are thrilled to be collaborating with Noga to accelerate and enable bringing a novel autologous gene therapy for PIDs to the clinic and patients. This collaboration will be the first instance of harnessing the Cocoon platform to manufacture an autologous gene therapy, thus showcasing the flexibility of the Cocoon system. We believe that by pairing Lonza’s Cocoon Platform, coupled with the array of R&D and manufacturing capabilities and expertise with Noga’s gene therapy technology and skills, we will reinforce our commitment to allow novel therapies to be manufactured reliably, reproducibly, at the needed scale and a sustainable cost.”

Quote from Noam Diamant, PhD., Chief Executive Officer of Noga Therapeutics:
“The technological gap that delays the development of lentiviral gene therapies for hundreds of Primary Immune Deficiencies (PIDs) must be addressed. We are excited to be working with Lonza to combine the strengths of our SyndogenousTM promoter design platform with those of the Cocoon. By designing an optimal lentivector and by automation of the entire cell therapy process, we will be able to significantly improve the control of this intricate therapy. I hope these advantages will enable us to accelerate the development of a much-needed therapy and to reach patients sooner.”

Ness-Ziona, Israel and Basel, Switzerland, 12 May 2020 – Noga Therapeutics, a private, pre-clinical-stage biopharmaceutical company developing novel lentiviral gene therapies for primary immune deficiencies, and Lonza, a leading cell & gene therapy manufacturer, announced today that the companies have a research collaboration and manufacturing agreement in place. This collaboration, aimed at the development of an autologous hematopoietic stem cell-based gene therapy for a primary immune deficiency, will leverage Lonza's expertise in process development and the usage of its proprietary Cocoon^® Platform.
The Cocoon^® Platform is a closed, highly customizable and scalable platform with minimal touchpoints, which will help Noga Therapeutics to accelerate the development of lentiviral gene therapies. The decentralized setting of the Cocoon^® Platform allows for great flexibility, as protocols are performed within a single single-use highly customizable cassette-system.
Lentiviral Autologous Gene Therapy makes use of the clinically efficacious and safe lentiviral gene delivery platform. In this treatment, hematopoietic stem cells are collected from the patient and transduced by lentiviruses that deliver a functional copy of a defective gene. The repaired stem cells are administered back to the patient and give rise to a functional immune system. Over the past two decades, this methodology has proven to be highly efficacious and safe in several primary immune deficiencies. However, some primary immune deficiencies with promising proof-of-concept studies are yet to enter the clinic due to efficacy and safety concerns. To this end, Noga harnesses the power of synthetic biology and bioinformatics to design optimized lentiviral vectors.

本ニュース・リリースは、Lonza Group Ltd が、2020 年5月12 日（スイス現地時間）に発表したニュース・リリースを日本語に翻訳再編集したものです。本資料の正式言語は英語であり、その内容・解釈については英語が優先します。
英文ニュースリリース：Noga Therapeutics and Lonza Announce Collaboration to Develop Autologous Lentiviral Gene Therapy on the Cocoon Platform
(https://www.lonza.com/news/2020-05-12-13-00)
（リンク先より全文をご覧いただけます/英語）