TG Therapeutics Clears Investigational Drug Azer-Cel for Multiple Sclerosis: Precision BioSciences

DURHAM, NC, USA – August 9, 2024 – Precision BioSciences, a leading gene editing company listed on Nasdaq (DTIL), has announced the U.S. Food and Drug Administration (FDA) clearance for an Investigational New Drug (IND) application submitted by TG Therapeutics. This clearance allows TG Therapeutics to initiate clinical trials to evaluate Azercabtagene Zapreleucel (azer-cel), an experimental allogeneic CAR T therapy, in human patients with progressive forms of multiple sclerosis.

Azer-cel is an innovative therapeutic approach designed to treat autoimmune diseases. It was originally discovered by Precision BioSciences and later licensed to TG Therapeutics specifically for use in autoimmune disease treatments. With this regulatory clearance, TG Therapeutics plans to commence phase 1 clinical trials in 2024, marking a significant step forward in exploring new therapeutic options for patients suffering from chronic autoimmune conditions.

Michael Amoroso, Chief Executive Officer of Precision BioSciences, expressed his enthusiasm for the IND clearance, congratulating TG Therapeutics on reaching this milestone. He highlighted the potential of allogeneic CAR T therapies to revolutionize treatments for patients with chronic diseases. Furthermore, Amoroso emphasized Precision BioSciences' commitment to advancing its own gene editing pipeline, with plans to submit an IND and/or Clinical Trial Application for PBGENE-HBV, a treatment targeting hepatitis B, by the end of the year.

In January 2024, a strategic partnership was established between Precision BioSciences and TG Therapeutics concerning azer-cel. Under this agreement, Precision BioSciences granted global rights to TG Therapeutics for the development of azer-cel in treating autoimmune diseases and non-cancer indications. In return, Precision BioSciences received upfront and near-term payments totaling $17.5 million, with the potential to earn up to $288 million based on the achievement of specific clinical, regulatory, and commercial milestones. Additionally, Precision BioSciences is eligible for royalties ranging from high-single-digit to low-double-digit percentages on net sales of azer-cel.

Precision BioSciences, Inc. is renowned for its advanced gene editing capabilities, primarily driven by its proprietary ARCUS® platform. The ARCUS® technology is distinguished by its unique method of cutting DNA, smaller size, and simpler structure compared to other gene editing technologies. These features of ARCUS nucleases contribute to more precise and defined therapeutic outcomes.

The company’s pipeline is focused on in vivo gene editing candidates aimed at providing lasting cures for a broad spectrum of genetic and infectious diseases, particularly those with inadequate current treatments. The ARCUS® platform enables sophisticated gene edits, including gene insertion, elimination, and excision. Gene insertion involves adding DNA to a gene to induce function or expression, elimination refers to removing unwanted genomic elements such as viral DNA, and excision entails removing large portions of defective genes by delivering two ARCUS nucleases simultaneously.

Precision BioSciences remains dedicated to leveraging its advanced genome editing platform to develop novel therapies that can significantly improve the quality of life for patients worldwide.

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