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Precision Biosciences Begins Clinical Trial of CAR T-cell Therapy for Multiple Myeloma.

The first patient has already been dosed for Phase 1/2a trial investigating Precision Biosciences‘ new CAR T-cell therapy candidate, PBCAR269A, for patients with multiple myeloma.

PBCAR269A is an allogeneic CAR T-cell approach that uses T-cells from healthy donors, allowing for the production of an “off-the-shelf” treatment for patients with myeloma.

The clinical trial expects to recruit 48 subjects at five clinical sites in the United States. They will then be receiving a chemotherapy regimen, which eliminates white blood cells to create room for the engineered T-cells and reduce immune reactions against these cells. Then, they will receive a single infusion of increasing PBCAR269A dose, in an attempt to determine the right amount of doses for additional testing.

Chief Medical Officer of Precision Biosciences says that there is a high unmet need for treatment for patients with Multiple Myeloma, and hopes that this treatment will be able to meet that need.

(Source: Myeloma Research News, 2020)

NIH Funds $14.6 Million To A “3-in-1” HIV Treatment.

The National Institutes of Health (NIH) is granting a $14.6 million investment into a 3 for 1 HIV research program by the USC and the Fred Hutchinson Cancer Research Center.

They will study the use of gene editing to remove CCR5 from patients’ stem cells inspired by a patient that has been cured of the virus by receiving blood stem cell transplants from donors who carried a mutation in the CCR5 gene back in 2007.

A five-year grant was given to back the pre-clinical studies, combining gene editing with technology to improve bone marrow transplants. The potential therapy would engineer a patient’s stem cells to fight HIV, and induce them to produce new immune cells once received again by the patient.

(Source: Endpoint News, 2020)

AskBio receives $2.6 million investment to solve gene therapy manufacturing limitations.

AskBio, a dedicated gene therapy company has recently received a $2.6 million grant from Scottish Enterprise to fund research into manufacturing challenges associated with gene therapies.

The company will use the funding to explore the manufacturing challenges related to scaling gene therapies for widespread patient access. AskBio also aims to further develop technologies that can improve the safety and efficacy of current therapies, and enable the treatment of high unmet genetic diseases.

Currently, adeno-associated virus (AAV) manufacturing has high costs and its complexity limits the availability and access to treatments, which poses a challenge for global distribution.

(Source: European Pharmaceutical Manufacturer, 2020)