Noga Therapeutics, a private, pre-clinical-stage biopharmaceutical company developing novel lentiviral gene therapies for primary immune deficiencies, and Lonza, a leading cell and gene therapy manufacturer, announced 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.

“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,” said Eytan Abraham, Head of Personalized Medicine at Lonza.

Noam Diamant, PhD, Chief Executive Officer of Noga Therapeutics commented, “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.”

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