Cell therapy weekly: Positive results from first liver disease cell therapy trial

This week: The FDA approves a decentralized clinical trial for a spinal cord cell therapy and a pharma collaboration seeks to progress next generation cancer cell therapies.

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Oct 10, 2019
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The news highlights:

Positive results from first liver disease cell therapy trial
Strategic collaboration will manufacture AAV9 gene therapy for neurodegenerative disease
FDA-approved, decentralized clinical trial to study spinal cord injury cell therapy
Pharma collaboration to progress next generation cancer cell therapies

Positive results from first liver disease cell therapy trial

In a Nature Medicine publication, researchers from the University of Edinburgh’s MRC Centre for Regenerative Medicine (CRM; Edinburgh, Scotland) have reported promising results from the first clinical trial assessing the safety of a liver cirrhosis cell therapy. The treatment involves the reprograming of autologous blood cells into macrophages, which are then re-administered to patients. No adverse treatment effects were observed in the preliminary investigation and planning for the next stage of the trial is now underway.

Stuart Forbes, Professor and Chair of Transplantation and Regenerative Medicine at the CRM, commented: “Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.” 

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Strategic collaboration will manufacture AAV9 gene therapy for neurodegenerative disease

Prevail Therapeutics (NY, USA) and Lonza Pharma & Biotech (Basel, Switzerland) have announced a strategic collaboration to manufacture AAV gene therapies for patients with neurodegenerative disorders. PR001, an AAV9-based gene therapy delivering GBA1 indicated for Parkinson’s disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease patients (nGD), and PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN mutation (FTD-GRN), will be manufactured at Lonza's gene therapy center of excellence in Houston (TX).

Asa Abeliovich, Founder and Chief Executive Officer, Prevail Therapeutics, commented, "We are pleased to partner with Lonza, a leader in the manufacturing of AAV gene therapy vectors, to enable large-scale production of our novel gene therapies that we believe hold potential to halt the course of neurodegenerative diseases for patient populations with urgent unmet needs.  Lonza and Prevail will work together closely on process development and scaling up production of PR001, our gene therapy for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease, to supply late-stage clinical trials and for commercial production."

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FDA-approved, decentralized clinical trial to study spinal cord injury cell therapy

The US FDA has approved VirTrial (AZ, USA) and Hope Biosciences (TX, USA) to carry out a hybrid, decentralized clinical trial to investigate Hope Biosciences’ autologous, adipose-derived, culture-expanded mesenchymal stem cell therapy (HB-adMSCs) for the treatment of spinal cord injury. The trial design will allow for more eligible patients to participate in the trial by providing patients with remote, nurse-led visits, allowing patients who cannot travel far to participate.

Donna Chang, President and CEO of Hope Biosciences, explained: “…we found that VirTrial met our needs as a sponsor and most importantly, patient needs. VirTrial’s partnership in our trial has opened doors for patients who would otherwise be unable to participate due to health and lack of mobility. Together, we are able to safely deliver and monitor our therapies for patients in the comfort of their homes. What this means for the world of cell therapy is nothing short of groundbreaking!”

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Pharma collaboration to progress next generation cancer cell therapies

GSK (London, UK) and Lyell Immunopharma (CA, USA) have announced a 5-year partnership to advance the development and marketing of cell therapies for various cancers. The collaboration will leverage Lyell’s biotechnology expertise and GSK’s extensive product pipeline and cell and gene therapy programs to improve the specificity of CAR-T cell therapies for the treatment of solid tumor cancers, which currently represents un unmet clinical need.

Hal Barron, Chief Scientific Officer and President of Research and Development at GSK, commented: “We are witnessing significant scientific innovation in cell and gene therapies, transforming the treatment of some blood-borne cancers, but patients with solid tumors are in need of equally effective treatments. Applying Lyell’s novel approach to counter T cell exhaustion and working with world class scientists…increases our probability of delivering the next generation of cancer cell therapies for patients with solid tumors.”

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.

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