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DURHAM, N.C. - Precision BioSciences (NASDAQ:DTIL), a small-cap biotechnology company currently valued at $49.34 million, announced new preclinical data for its PBGENE-DMD gene editing candidate for Duchenne muscular dystrophy (DMD) showing increased dystrophin-positive muscle cells across multiple muscle types. According to InvestingPro analysis, the company appears undervalued based on its Fair Value estimates.
The company reported observing up to a three-fold increase in dystrophin-positive muscle cells between three and nine months in a DMD mouse model, with up to 85% of cells in the gastrocnemius (calf muscle) showing dystrophin positivity. The study also examined effects in the quadricep, heart, and diaphragm. With analyst price targets ranging from $13 to $60, significantly above the current trading price, market participants appear optimistic about the company’s potential. Investors can access more detailed analysis and 8 additional key insights through InvestingPro.
"These new data build upon the preclinical data shared at ASGCT in May 2025, demonstrating that PBGENE-DMD treatment resulted in significant and sustained improvement of maximum force output," said Cassie Gorsuch, Chief Scientific Officer at Precision BioSciences.
PBGENE-DMD uses two complementary ARCUS nucleases delivered via a single AAV administration to excise exons 45-55 of the dystrophin gene. The approach aims to restore near full-length dystrophin protein production and could potentially address up to 60% of DMD patients.
The company indicated that final IND-enabling toxicology studies are currently underway, with plans to submit an Investigational New Drug application and/or Clinical Trial Application in 2025. Initial clinical data is expected in 2026.
Precision BioSciences stated that its current cash runway should be sufficient to progress both PBGENE-HBV, its Phase 1 hepatitis B asset, and PBGENE-DMD through Phase 1 clinical readouts. Financial data from InvestingPro shows the company maintains a strong liquidity position with a current ratio of 6.62, though it’s worth noting the company is currently burning through cash at a significant rate. The next earnings update is scheduled for August 14, 2025.
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness due to alterations in the dystrophin protein. Currently, there are no approved treatments that significantly improve muscle function over time.
The information in this article is based on a press release statement from Precision BioSciences.
In other recent news, Precision BioSciences announced that its gene editing therapy, PBGENE-DMD, has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. This designation is significant as it targets Duchenne muscular dystrophy (DMD), a rare genetic disorder affecting approximately 15,000 individuals in the United States, primarily under age 18. With this designation, Precision BioSciences may become eligible for a Priority Review Voucher, which can expedite the review of another product or be sold to another company. The company is currently completing final IND-enabling toxicology studies, with initial clinical data expected in 2026.
Additionally, Precision BioSciences held its annual shareholder meeting, where Kevin J. Buehler and Shari Lisa Piré were elected as Class III directors to serve until 2028. Shareholders also ratified the appointment of Deloitte & Touche LLP as the independent registered public accounting firm for the fiscal year ending December 31, 2025. An advisory vote on executive compensation passed, while a proposal to amend the Certificate of Incorporation did not achieve the necessary majority. These developments reflect ongoing governance and strategic decisions within the company.
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