Prime Medicine Publishes PM359 Clinical Data
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Dec 07 2025
0mins
Should l Buy PRME?
Prime Medicine announced the publication of Phase 1/2 clinical data with PM359, the Company's investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease, CGD, in the New England Journal of Medicine, NEJM. The data will also be presented in a poster session at the 67th American Society of Hematology, ASH, Annual Meeting, December 6-9, 2025 in Orlando, Florida. The publication, titled "Prime Editing for p47-phox Chronic Granulomatous Disease," reports initial data for two patients treated in the Phase 1/2 trial of PM359, which was designed to assess safety, biological activity and preliminary efficacy in adult and pediatric study participants. Results: Both patients enrolled in the study had a history of prior CGD-defining complications; Both patients experienced rapid neutrophil engraftment, achieving 69% and 83% dihydrorhodamine-positive neutrophils by Day 30; Both patients remain free of new CGD-related complications or significant intercurrent illnesses post-infusion; No clinically significant adverse events attributable to PM359 occurred in either patient, and all observed toxicities were consistent with busulfan-based conditioning.
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Analyst Views on PRME
Wall Street analysts forecast PRME stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for PRME is 6.06 USD with a low forecast of 4.25 USD and a high forecast of 9.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
7 Analyst Rating
4 Buy
3 Hold
0 Sell
Moderate Buy
Current: 3.240
Low
4.25
Averages
6.06
High
9.00
Current: 3.240
Low
4.25
Averages
6.06
High
9.00
About PRME
Prime Medicine, Inc. is a biotechnology company engaged in creating and delivering gene editing therapies to patients. It deploys its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. It is advancing a set of high-value programs across its core areas of focus (hematology, immunology and oncology, liver, and lung). These include ex vivo hematopoietic stem cell (HSC) programs for the treatment of p47phox chronic granulomatous disease CGD and X-linked CGD; a lipid nanoparticle (LNP) Prime Editor for the treatment of Wilson’s Disease; a LNP or adeno-associated virus Prime Editors for the treatment of cystic fibrosis; and ex vivo T-cell therapies. PM359, its product candidate within its hematology, immunology and oncology area of focus, targets the p47phox variant of CGD. Its PM577 comprises a Prime Editor encapsulated as RNA cargo in a liver-targeted LNP.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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- Technological Innovation: Avant Technologies is developing a cell encapsulation technology in partnership with SGAustria that protects genetically modified cells from immune rejection, addressing the long-standing issue of diabetes patients relying on immunosuppressive drugs, thereby enhancing treatment safety and efficacy.
- Market Opportunity: With 589 million people globally living with diabetes, Avant Technologies is positioned to capitalize on a substantial market opportunity, projected to grow to 853 million by 2050, highlighting the urgent need for innovative treatment solutions.
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- Strategic Partnerships: Avant's collaboration with SGAustria focuses on developing cell-based therapies for type 1 and insulin-dependent type 2 diabetes using encapsulation technology to address immune rejection, which is expected to significantly enhance patient quality of life.
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- Warrant Details: Each warrant allows the holder to purchase one common share at $0.06 for three years, which will provide the company with additional funding sources to facilitate business expansion.
- Planned Use of Funds: The company intends to use the proceeds for business development and general working capital, aiming to enhance operational efficiency and strengthen market competitiveness.
- Regulatory Approval Requirements: The financing is subject to final approval from the Canadian Securities Exchange and other relevant regulatory bodies, ensuring compliance and reducing future operational risks.
See More
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- Unit Structure: Each unit is priced at $1,000 and consists of 16,667 common shares and 16,667 transferable warrants, with an expected issuance of 5 million to 83.33 million common shares, thereby strengthening the company's capital base.
- Warrant Terms: Each warrant allows the holder to purchase a common share at $0.06 per share for three years, providing potential appreciation opportunities for investors and enhancing market appeal.
- Regulatory Approval: The private placement is subject to final approval from the Canadian Securities Exchange and other relevant regulatory bodies, ensuring compliance and mitigating investment risks.
See More
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- Clinical Data Release: Prime Medicine published Phase 1/2 clinical data for PM359 in the New England Journal of Medicine, showing that two patients achieved 69% and 83% neutrophil engraftment by Day 30, significantly exceeding the 20% minimum threshold for clinical benefit, marking the first demonstration of Prime Editing's safety and efficacy in humans.
- Significant Efficacy: Both patients rapidly restored NADPH oxidase activity post-treatment and remained free of new CGD-related complications, indicating PM359's potential as a precise therapeutic strategy for chronic granulomatous disease, with important clinical implications.
- Safety Confirmation: No clinically significant adverse events attributable to PM359 were observed during treatment, and all noted toxicities were consistent with busulfan-based conditioning, further enhancing confidence in the therapy's safety profile.
- Future Development Potential: This data release not only showcases PM359's early clinical efficacy but also provides critical insights into the broader applications of Prime Editing technology, potentially offering treatment solutions for various genetic diseases and advancing the company's strategic development in the gene editing field.
See More
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- Clinical Data Release: Prime Medicine published Phase 1/2 clinical data for PM359 in the New England Journal of Medicine, demonstrating that two patients achieved 69% and 83% neutrophil engraftment by Day 30, significantly exceeding the 20% minimum threshold for clinical benefit, marking the first human validation of Prime Editing's safety and efficacy.
- Sustained Efficacy: Both patients remained free of new CGD-related complications post-treatment, with Patient 1 stopping mesalamine without CAC flare-ups and Patient 2 showing a substantial decrease in fecal calprotectin levels, indicating PM359's potential to enhance patient quality of life.
- Safety Assessment: No clinically significant adverse events attributable to PM359 were observed during treatment, with all noted toxicities consistent with busulfan-based conditioning, further supporting the safety profile of Prime Editing technology.
- Future Development Potential: This data release not only showcases PM359's early clinical efficacy but also provides critical insights into the broad applicability of Prime Editing technology in gene editing, potentially opening new avenues for treating various genetic and immunological diseases.
See More
