Precision Bio's DMD Therapy Gets Rare Pediatric Status, But Stock Slides; Retail Crowd Sees ‘Good Risk-Reward’

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:

• Significant, durable functional improvements in a humanized DMD mouse model
• Restoration of dystrophin production in key muscle types, including cardiac and skeletal muscle
• Editing of satellite muscle stem cells, believed to be important for long-term durability

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.

For updates and corrections, email newsroom[at]stocktwits[dot]com.