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DURHAM, N.C. - Precision BioSciences (NASDAQ:DTIL), a biotechnology company currently valued at approximately $51 million and trading near $4.57, announced Wednesday that its gene editing therapy PBGENE-DMD for Duchenne muscular dystrophy (DMD) has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. According to InvestingPro analysis, the company appears undervalued based on its Fair Value metrics.
The designation applies to therapies targeting serious diseases affecting fewer than 200,000 people in the U.S., primarily under age 18. Approximately 15,000 Americans live with DMD, a rare genetic disorder.
With this designation, Precision may become eligible for a Priority Review Voucher upon potential FDA approval of PBGENE-DMD. Such vouchers can be used to expedite review of another product or be sold to another company.
PBGENE-DMD utilizes Precision’s ARCUS gene editing platform to excise exons 45-55 of the dystrophin gene through a one-time administration. According to the company, this approach could potentially benefit up to 60% of DMD patients by enabling production of near full-length dystrophin protein.
In preclinical studies, the therapy demonstrated ability to target key muscle types involved in DMD progression and produced functional improvements in a humanized mouse model, according to the press release.
The company is currently completing final IND-enabling toxicology studies and expects to generate initial clinical data in 2026.
Precision BioSciences focuses on developing in vivo gene editing therapies using its proprietary ARCUS platform for genetic and infectious diseases where limited treatment options exist.
The information in this article is based on a company press release statement.
In other recent news, Precision BioSciences has been granted a Rare Pediatric Disease Designation by the FDA for its PBGENE-DMD treatment aimed at Duchenne muscular dystrophy. This designation may allow the company to receive a Priority Review Voucher upon FDA approval, which could be used for another product or sold for non-dilutive capital. Additionally, Precision BioSciences received a Fast Track designation from the FDA for its PBGENE-HBV therapy targeting chronic hepatitis B. This designation is intended to expedite the development and review process for treatments addressing serious conditions. The company is conducting a global Phase 1 trial, known as ELIMINATE-B, to evaluate the safety and efficacy of PBGENE-HBV. In governance news, Precision BioSciences held its annual shareholder meeting, where Kevin J. Buehler and Shari Lisa Piré were elected as Class III directors. Shareholders also ratified Deloitte & Touche LLP as the independent registered public accounting firm for the fiscal year ending December 31, 2025. Meanwhile, the biotech sector, including Precision BioSciences, experienced a downturn following the resignation of FDA’s top vaccine official, Peter Marks, causing concern over potential changes in the regulatory landscape.
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