New R&D Center Will Focus on Cell-based Therapies

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by Margarida Maia |

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cell-based therapies/sicklecellanemianews.com/Brooklyn ImmunoTherapeutics opens R&D center

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Biopharmaceutical company Brooklyn ImmunoTherapeutics has launched a new center focused on research and development (R&D) of gene editing and cell-based therapies to treat cancer and rare blood disorders, including sickle cell disease.

“Our new R&D facility will provide us with the ability to advance our research and development efforts,” Howard J. Federoff, MD, PhD, Brooklyn’s CEO and president, said in a press release.

The center, based in Cambridge, Massachusetts, shares a location with biotechnology developers Factor Bioscience and Novellus Therapeutics.

In April, Brooklyn bought the global rights to develop and market certain cell-based therapies based on the patented technology and practical knowledge of these two companies.

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Factor is developing a portfolio of cell-based technologies to drive the study and treatment of a range of diseases. Its partner, Novellus, is a clinical-stage company that advances the use of cell-based technologies for therapeutic applications.

“We believe co-locating with Factor and Novellus will promote our capability to leverage our collaboration and further our objectives in developing a diversified portfolio of new, advanced therapeutics,” Federoff said.

“Recent presentations by Factor and Novellus … have reinforced our conviction that our development pathways can be broadly applicable to real world disease indications for which no effective therapies currently exist,” Federoff added.

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Brooklyn’s recently acquired licensed platform from Factor and Novellus includes three original technologies that the company expects to develop further at its new R&D center.

The first is a highly efficient technology for reprogramming cells, or converting cells from one particular type to another. It uses messenger RNA, known as mRNA, a molecule with the genetic instructions needed to make proteins.

The second is a gene-editing technology that allows the generation of personalized cell-based therapies in which a disease-causing defective gene may be corrected. Used in combination with mRNA-based cell reprogramming, the technology may be used for the development of therapies involving cells from an individual’s own body or from another individual.

The third is a proprietary delivery system that uses tiny particles of fat (nanolipids) to deliver mRNA molecules into the skin, brain, eye, and lung tissue.

“We are delighted to advance our R&D efforts to commence translation of gene editing, cellular therapy and development for our emerging clinical programs with a focus on orphan diseases such as sickle cell anemia, familial amyloidosis and cell therapies for cancer,” Federoff concluded.