Precision BioSciences Receives Study May Proceed Notification From FDA, Allowing Co To Initiate IRB Activities And Clinical Trial Site Activation For FUNCTION-DMD Phase 1/2 Clinical Trial For PBGENE-DMD

– U.S. FDA Study May Proceed notification enables initiation of clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical study in patients with Duchenne muscular dystrophy (DMD) –

– Institutional Review Board (IRB) process is underway at multiple world-class DMD clinical trial sites –

– PBGENE-DMD is an in vivo gene editing investigational product designed to correct the underlying genetic cause of DMD through a novel one-time gene editing therapy –

– Company plans to host a virtual event in March after the Muscular Dystrophy Association conference to discuss PBGENE-DMD and the FUNCTION-DMD clinical study with participation from key opinion leaders including patient advocacy –

Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (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 (DMD) 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.

PBGENE-DMD is Precision's wholly-owned, first-in-class in vivo gene editing program utilizing a gene excision approach intended to permanently correct the dystrophin gene in patients with mutations between exons 45 and 55, the "hot-spot" region, which accounts for the largest molecular subset of patients with DMD at approximately 60%. The therapeutic goal is to restore the ability of the dystrophin gene to produce a near full-length functional dystrophin protein. This protein retains 80% of full-length dystrophin, substantially larger than synthetic micro-dystrophin constructs approved or in development today (which are approximately 34% of the size of full-length dystrophin).