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Prime Medicine Inc. (PRME) is pioneering next-generation genetic therapies through its Prime Editing platform, developing potential cures for previously untreatable diseases. This news hub provides investors and industry observers with essential updates on clinical advancements, strategic collaborations, and regulatory developments.
Access real-time updates on PRME's progress across therapeutic areas including hematology, immunology, and neuromuscular disorders. Our curated collection features official press releases, earnings reports, and analysis of key milestones in gene editing innovation.
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Prime Medicine (Nasdaq: PRME) has unveiled a preclinical program for treating alpha-1 antitrypsin deficiency (AATD), marking their second liver franchise program. The company plans to file an IND/CTA in mid-2026.
The program utilizes Prime's proprietary universal liver lipid nanoparticle (LNP) to edit the E342K (Pi*Z) mutation in the SERPINA1 gene. Initial in vivo data showed up to 72% precise correction of the SERPINA1 gene in hepatocytes of humanized mice, restoring over 95% of serum AAT to the corrected isoform with healthy AAT protein levels above 20µM.
Preclinical studies using unoptimized surrogate Prime Editors in non-human primate models demonstrated greater than 50% editing with an excellent safety profile and no detectable off-target edits. The company expects to advance both AATD and Wilson's Disease programs toward clinical data in 2027.
Prime Medicine (NASDAQ: PRME) has reported its full year 2024 financial results and provided key business updates. The company is progressing with its Phase 1/2 clinical trial of PM359 for p47phox CGD, with initial data expected in 2025. Their Wilson's Disease program (PM577) is advancing through IND-enabling studies, targeting IND/CTA filing in 1H 2026.
Financial highlights for 2024 include:
- R&D expenses increased to $155.3 million (from $147.9M in 2023)
- G&A expenses rose to $50.2 million (from $43.4M in 2023)
- Net loss was $195.9 million (improved from $198.1M in 2023)
- Cash position strengthened to $204.5 million (up from $135.2M in 2023)
The company expects its current cash position to fund operations into the first half of 2026.
Prime Medicine (Nasdaq: PRME), a biotechnology company focused on developing one-time curative genetic therapies, has announced its participation in the TD Cowen 45th Annual Health Care Conference. The company's President and CEO, Keith Gottesdiener, M.D., will engage in a fireside chat on Monday, March 3, 2025, at 11:10 a.m. ET in Boston, MA.
The presentation will be accessible through a live audio webcast in the 'Events & Presentations' section of Prime Medicine's website. Interested parties can access the replay of the webcast on the company's website for 30 days after the presentation.
Prime Medicine, a biotechnology company specializing in one-time curative genetic therapies, has announced that its President and CEO, Keith Gottesdiener, M.D., will present at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Tuesday, January 14, 2025, at 3:00 p.m. PT (6:00 p.m. ET) in San Francisco, CA.
A live audio webcast of the presentation will be accessible under the “Events & Presentations” section on the company’s website, www.primemedicine.com. Additionally, a replay of the webcast will be available on the website for 30 days after the presentation.
Prime Medicine reported Q3 2024 financial results and business updates. The company presented initial in vivo data for its Wilson's Disease program and universal liver-targeted LNP at ESGCT, showing up to 80% precise mutation correction. A strategic collaboration with Bristol Myers Squibb brought $110 million upfront with potential for $3.5 billion in milestone payments. The company reported a net loss of $52.5 million and pro-forma cash position of $244.6 million, expected to fund operations into 1H 2026. Prime Medicine remains on track to file IND/CTA for Wilson's Disease in 1H 2026 and report initial data from Phase 1/2 trial of PM359 for CGD in 2025.
Prime Medicine (Nasdaq: PRME) has announced its participation in three upcoming investor conferences in November 2024. The company will engage in fireside chats at Guggenheim's Inaugural Healthcare Innovation Conference in Boston on November 13 at 3:00 p.m. ET, the Stifel 2024 Healthcare Conference in New York on November 18 at 1:15 p.m. ET, and the Jefferies London Healthcare Conference in London on November 19 at 2:30 p.m. GMT. Live audio webcasts will be available on Prime Medicine's website, with replays accessible for 90 days after each event.
Prime Medicine (Nasdaq: PRME) presented promising preclinical data for its Wilson's Disease program and universal liver-targeted LNP platform at the ESGCT 31st Annual Congress. The company demonstrated successful correction of disease-causing mutations in both mouse and non-human primate models, achieving up to 80% precise correction of the H1069Q mutation in humanized mice and up to 51% editing in NHPs. The proprietary GalNAc-LNP delivery system showed improved safety, potency, and biodistribution compared to other LNPs.
The company plans to initiate IND-enabling activities for the Wilson's Disease program in Q4 2024, with IND/CTA filing expected in 1H 2026. Additional data from their Glycogen Storage Disease Type 1b (GSD1b) program showed up to 85% precise liver cell editing, with effects lasting up to 44 weeks in NHPs, validating their universal LNP approach.
Prime Medicine (Nasdaq: PRME) announced it will present new preclinical data from its Prime Editing pipeline at two upcoming scientific meetings: the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress in Rome (October 22-25, 2024) and the American Association for the Study of Liver Diseases (AASLD) in San Diego (November 15-19, 2024).
The company will showcase in vivo data from its Wilson's Disease program and its universal liver-directed lipid nanoparticle (LNP) platform. Prime Medicine is on track to initiate IND-enabling activities for the Wilson's Disease program in Q4 2024, with an IND or CTA filing expected in H1 2026.
Presentations will cover various topics, including glycemic control restoration in Glycogen Storage Disease Type 1b, Prime Edited cell products for Chronic Granulomatous Disease, and advancements in Prime Editing for Wilson's Disease and Retinitis Pigmentosa.
Prime Medicine (Nasdaq: PRME) has announced a strategic focus on high-value programs in its pipeline. The company is prioritizing:
- Two programs for chronic granulomatous disease (CGD), including PM359 for p47phox CGD and a new program for X-linked CGD
- A collaboration with Bristol Myers Squibb for ex vivo T-cell therapies
- A liver lipid nanoparticle program for Wilson's Disease
- Two strategies for Cystic Fibrosis treatment
Prime Medicine expects to share initial clinical data from its CGD trial in 2025 and new preclinical data for Wilson's Disease in Q4 2024. The company aims to file an IND/CTA for Wilson's Disease in 1H 2026. With the Bristol Myers Squibb collaboration and cost savings, Prime Medicine anticipates extending its cash runway into 1H 2026.
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