DTIL News & Events

Precision BioSciences announced presentation of new preclinical study data supporting the potential long-term efficacy of PBGENE-DMD. The data presentation took place during a poster session at the Muscular Dystrophy Association Clinical & Scientific Conference 2026 currently taking place in Orlando, Florida. In new data from a GLP study conducted in a humanized DMD mouse model which replicates muscle degeneration observed in patients with DMD, PBGENE-DMD treatment led to improvements in numerous markers of muscle damage, including Alanine Aminotransferase, Aspartate Aminotransferase, and Creatine Kinase. At 90 days after administration of PBGENE-DMD, treatment resulted in a approximately 50%-65% reduction in CK levels, indicating substantial muscle integrity improvements. Furthermore, results showed improvements in muscle pathology assessments with lower composite injury scores across multiple muscle tissues compared to control vehicle-treated mice. Together, these data support PBGENE-DMD's differentiated profile as a gene editing therapy designed to potentially treat up to 60% of DMD patients by restoring production of a near full-length, functional dystrophin protein through permanent gene correction.

Precision BioSciences announced that the FDA has granted fast track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy. The company also announced that it will host a virtual key opinion leader event on Tuesday, March 17 at 9:00 AM ET to discuss PBGENE-DMD and the planned Phase 1/2 FUNCTION-DMD clinical study.

Precision BioSciences (DTIL) announced the achievement of a clinical milestone under its license agreement with TG Therapeutics (TGTX). The milestone payment for azercabtagene zapreleucel was triggered by progress of a Phase 1 clinical trial of azer-cel in progressive forms of multiple sclerosis. As a result of this milestone event, Precision will receive $7.5M in proceeds, inclusive of $5.25M cash and $2.25M for the purchase of 201,504 shares of Precision common stock by TG Therapeutics at $11.17 per share, pursuant to the terms of the companies' license agreement. Existing cash and cash equivalents, inclusive of the azer-cel milestone proceeds, continued fiscal and operating discipline, and availability of the ATM facility, are expected to provide sufficient cash runway through 2028. In January 2024, Precision BioSciences announced a licensing deal, granting TG Therapeutics exclusive worldwide rights to develop and commercialize azer-cel in autoimmune diseases and other non-oncology indications. Under the agreement, Precision received upfront and potential near-term economics and remains eligible for additional development, regulatory, and commercial milestone payments, as well as royalties on net sales. Precision is eligible to receive up to $288M in additional milestone payments as well as high-single-digit to low-double-digit royalties on net sales.

Precision BioSciences announced that the company received a study may proceed notification from the FDA. This allows Precision BioSciences to initiate IRB activities and clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical trial for PBGENE-DMD for the treatment of ambulatory Duchenne muscular dystrophy patients at highly specialized clinical trial sites. The objective of the study is to evaluate safety, tolerability and efficacy, including dystrophin protein expression and functional outcomes in patients afflicted with DMD.