The FDA designation could make Precision eligible for a valuable Priority Review Voucher, while preclinical data showed strong dystrophin restoration across key muscle groups.

Precision BioSciences drew heightened retail investor chatter on Wednesday after the FDA awarded Rare Pediatric Disease designation to its gene editing therapy PBGENE-DMD, which targets duchenne muscular dystrophy (DMD) treatment.

The stock surged as much as 9% during regular trading but reversed course to close down 7.2% at $4.24, and extended losses in after-hours trading, slipping 3.5%.

The designation, which is intended to provide incentives for the development of drugs and biologics for serious and life-threatening conditions that predominantly affect individuals under age 18, could provide Precision with a priority review voucher (PRV) upon approval of the drug.

PRVs can be redeemed for accelerated FDA review of a different drug or sold to other companies, creating potential for non-dilutive capital.

Precision’s chief development and business officer, Cindy Atwell, said the designation supports the potential of PBGENE-DMD to deliver a “meaningful improvement and durable functional benefit” for up to 60% of patients.

PBGENE-DMD is a first-in-class in vivo gene editing therapy that uses the company’s proprietary Arcus platform, designed to excise exons 45–55 of the dystrophin gene using two Arcus nucleases delivered in a single Adeno-associated virus (AAV). 

The one-time treatment aims to restore a near full-length, naturally produced dystrophin protein, considered functional in humans.

In preclinical studies, PBGENE-DMD showed:

 

The company is currently completing final IND-enabling toxicology studies and said it expects to begin clinical testing in 2026.

Precision also said that its Arcus platform enables precise, defined therapeutic gene edits using a small, structurally distinct nuclease, supporting gene insertion, elimination, and excision. 

On Stocktwits, retail sentiment was ‘extremely bullish’ amid a 4,025% surge in 24-hour message volume.

One user pointed to Precision’s cash reserves of over $80 million and described the current setup as a “good risk-reward,” suggesting the downside appears limited while the potential upside could be unlimited.

Another user, reflecting on a much higher cost basis from early 2021, assessed whether the current dip might offer a fresh buying opportunity.

The stock has declined 9.6% so far in 2025.

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