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Cell therapy weekly: FDA aims to advance gene therapies and T cells reprogrammed without the use of viral vectors

This week: Rubius Therapeutics shows confidence in red blood cell-based immunotherapy with new manufacturing plant.

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Jul 12, 2018
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The news highlights:

FDA unveils framework for the development, review and approval of gene therapies
T cells reprogrammed without viral vectors
New US$155 million cell therapy manufacturing facility for Rubius Therapeutics

FDA unveils framework for the development, review and approval of gene therapies

In a statement, the FDA has announced a framework to advance the development of gene therapies, similar to that for regenerative medicine released last year. The new draft guidances cover development of gene therapies for specific indications – hemophilia, retinal disorders and rare diseases – and three updates to existing guidances on manufacturing of gene therapy, covering chemistry, manufacturing, and control (CMC), testing of retroviral vector-based gene therapy products for replication competent retrovirus (RCR) during product manufacture and patient follow-up and long term follow-up after administration of human gene therapy products.

“When it comes to novel technologies like gene therapy, the FDA is steadfastly committed to a regulatory path that maintains the agency’s gold standard for assuring safety and efficacy. As we develop this evidence-based framework, we’re going to have to modernize how we approach certain aspects of these products in order to make sure our approach is tailored to the unique challenges created by these new platforms,” commented FDA Commissioner Scott Gottlieb in the statement.

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T cells reprogrammed without viral vectors

In new research from the University of California, San Francisco (UCSF; CA, USA), electroporation and CRISPR have been used to insert DNA into T cells, without the use of a viral vector. This new method is fast, easy to use and allows large sections of DNA to be integrated into T cells, with very low levels of off target effects. The method has been demonstrated by use in children with a rare form of autoimmunity and by the creation of engineered T cells that showed anti-cancer activity.

"This is a rapid, flexible method that can be used to alter, enhance, and reprogram T cells so we can give them the specificity we want to destroy cancer, recognize infections, or tamp down the excessive immune response seen in autoimmune disease," said UCSF's Alex Marson, associate professor of microbiology and immunology, and senior author of the new study. "Now we're off to the races on all these fronts."

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New US$155 million cell therapy manufacturing facility for Rubius Therapeutics

A new cell therapy plant in Smithfield (RI, USA) will see Rubius Therapeutics (MA, USA) invest $155 million in a new 135,000-square-foot facility with 160 employees. Rubius is likely use the facility to manufacture its proprietary cell therapy based on utilizing bioreactors to produce enucleated red blood cells that express proteins inside, or on the surface on the cell, to treat cancer or enzyme-related disease.

Commenting earlier in the year, Rubius President Torben Straight Nissen said: “We have been able to express both costimulatory molecules as well as other relevant proteins that are being used in immuno-oncology today on red cells. We don't need to combine those types of therapies because we can express them on red cells.”

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

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