Under the FDA’s new draft guidance, companies can file one application that covers several versions of the same gene-editing treatment, as long as each version aims to make the same kind of genetic correction.
- Companies with modular gene editing platforms stand to benefit most, including Prime Medicine’s prime-editing system and lipid-nanoparticle delivery, if the draft is adopted.
- The draft guidance is now open for public comment, which will later be reviewed by the FDA before it decides whether to adopt it.
- Earlier this month, the company announced it had dosed the first patient in a global Phase 1/2 trial of PM577a for Wilson disease—the first known use of prime editing therapy inside a living patient.
Shares of gene-editing companies, including Prime Medicine (PRME), rallied on Wednesday after the U.S. Food and Drug Administration issued draft guidance designed to speed personalized treatment for rare genetic diseases.
Under the FDA’s new draft guidance, companies can file one application that covers several versions of the same gene-editing treatment, as long as each version aims to make the same kind of genetic correction. Once clinical results support the core approach, new versions that stay within set limits can be added later using mainly lab tests instead of full new patient trials. The guidance applies to gene-editing therapies for rare diseases caused by a single known genetic fault.
Companies with modular gene editing platforms stand to benefit most, including Prime Medicine’s prime-editing system and lipid-nanoparticle delivery, if the draft is adopted. The draft guidance is now open for public comment, which will be later reviewed by the FDA before it decides whether to adopt it.
Shares of Prime Medicine were up 20% at the time of writing, while Beam Therapeutics traded 14% higher and Intellia Therapeutics (NTLA) and Editas Medicine (EDIT) climbed 6-11%. CRISPR Therapeutics (CRSP) posted more modest gains of 4%.
Why PRME Is Outperforming Peers
Prime clocked larger gains than its peers as it builds on company-specific updates. Earlier this month, the company announced it had dosed the first patient in a global Phase 1/2 trial of PM577a for Wilson disease—the first known use of prime editing therapy inside a living patient. The FDA also granted the program Rare Pediatric Disease designation. Initial clinical data from the Wilson disease study are expected in 2027.
The company also recently received the FDA’s clearance to start human trials of an alpha-1 antitrypsin deficiency candidate.
How Did PRME Retail Traders React?
On Stocktwits, retail sentiment around PRME stock jumped from ‘neutral’ to ‘extremely bullish’ territory over the past 24 hours, while message volume increased from ‘low’ to ‘extremely high’ levels.
A Stocktwits user noted that big pharma company Bristol Myers Squibb is invested in Prime. The user is referring to a collaboration deal between BMY and Prime inked in Sept. 2024 under which Prime received $110 million upfront, including a $55 million equity investment. The company is eligible for more than $3.5 billion in potential milestones under the deal.
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Another user, however, warned of upcoming dilution.
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PRME stock has gained 51% year-to-date.
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