Cell therapy weekly: UltraCAR-T given boost in fight against acute myeloid leukemia

Also this week: gene therapy trial for cocain addiction proposed by Mayo Clinic and the cell and Ori Biotech's gene therapy platform receives US$9.4 million.

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Jan 09, 2020
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The news highlights:

UltraCAR-T, PRGN-3006, given orphan drug designation to treat acute myeloid leukemia
Gene therapy trial for cocaine addiction proposed by Mayo Clinic
Ori Biotech's cell and gene therapy platform receives US$9.4 million

UltraCAR-T, PRGN-3006, given orphan drug designation to treat acute myeloid leukemia

Precigen (MD, USA) obtain orphan drug designation status for their non-viral ultraCAR-T therapy, PRGN-3006. The drug, designed to target relapsed or refractory acute myeloid leukemia, will now qualify for various incentives following the US FDA’s (MD, USA) decision to approve the designation, helping to bring the innovative treatment to patients quicker.

"This regulatory designation underscores the critical medical need for new therapies to treat AML patients. AML is a progressive, debilitating and often fatal disease with limited treatment options," explained Helen Sabzevari, President and CEO of Precigen. "As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease."

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Gene therapy trial for cocaine addiction proposed by Mayo Clinic

Researchers from the Mayo Clinic (MN, USA) are seeking approval to test a new gene therapy for cocaine addiction. Having recently published positive results with mice, within the Human Gene Therapy journal, the team hope to progress to human trials. The therapy involves inserting a gene into the liver, which enables the cocaine to be metabolized into a harmless byproduct.

“Substance use disorders present an immense public health problem in the US and other industrialized countries,” commented Terence R. Flotte (University of Massachusetts Medical School, MA, USA). “Putting the power of an innovative gene therapy to work on this problem presents an exciting new approach.”

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Ori Biotech's cell and gene therapy platform receives US$9.4 million

Ori Biotech (London, UK) has obtained US$9.4 million in seed funding to further their work in cell and gene therapy manufacturing. The innovative platform, designed by Ori Biotech, aims to produce scalable solutions to address the critical issues facing clinical and commercial manufacturing through automation and standardization.

"The successful financing underscores the potential of the Ori platform to fully automate cell and gene therapy manufacturing to increase throughput, improve quality and decrease costs,” commented Jason Foster, CEO of Ori Biotech. “We look forward to collaborating with best-in-class suppliers, service providers and therapeutics developers to create next generation manufacturing solutions. We appreciate the support from our investors, and I am honored to join a company that has the potential to positively impact millions of lives by enabling patient access to these lifesaving treatments."

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

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