Prime Medicine, Inc. (PRME) Presents At Citi's Biopharma Back To School Conference Transcript
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Sep 02 2025
0mins
Should l Buy PRME?
Source: SeekingAlpha
Upfront payment from BMS collaboration $110 million upfront, with an additional $185 million in potential preclinical milestones. This collaboration focuses on ex vivo CAR-T therapies for immunology, hematology, and oncology.
Cash runway $260 million pro forma cash balance as of June 2025, extended into 2027 due to recent financing in August 2025. This extension is attributed to the recent financing and potential non-equity sources.
Market opportunity for AATD Estimated $20 billion to $40 billion market opportunity globally, with 100,000+ patients having the mutation and 10,000 to 15,000 diagnosed in the U.S. The market is limited to the U.S. and Europe due to the absence of the disease in the Asian population.
Market opportunity for Wilson's disease Estimated $20 billion to $40 billion global market opportunity, with 10,000 to 11,000 patients in the U.S. and similar numbers in Europe. Japan and Asian countries have higher prevalence rates, potentially making Japan a market as large as the U.S. and Europe combined.
Preclinical data for Wilson's disease Demonstrated high levels of editing at relevant dose levels, strong phenotypic data showing reduction in liver copper, urinary copper, and corresponding increase in fecal copper. This data supports confidence in clinical translation.
Preclinical data for AATD Strong preclinical profile with high editing levels, favorable Z versus M protein levels, and total AAT levels. The data suggests potential for a best-in-class therapy.
Proof of concept for Prime Editing in CGD Achieved proof of concept with 2 patients showing rapid engraftment and normalization of biomarkers indicative of inflammatory bowel disease. However, the program is being discontinued due to a limited patient population in the U.S. (30-50 patients).
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Analyst Views on PRME
Wall Street analysts forecast PRME stock price to rise
7 Analyst Rating
4 Buy
3 Hold
0 Sell
Moderate Buy
Current: 3.320
Low
4.25
Averages
6.06
High
9.00
Current: 3.320
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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- 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.
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- 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
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- FDA Approval of New Therapies: The FDA's approval of three transformative cell therapies in December marks a pivotal shift from experimental concepts to commercial reality, further propelling the strategic development of companies like Avant Technologies.
- 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.
See More
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- Market Growth Potential: The gene therapy market is projected to reach $36.55 billion by 2032, growing at a compound annual rate of 17.98%, reflecting a surge in demand for curative treatments that drives investor confidence in functional cures.
- FDA Approval of New Therapies: The FDA's approval of three transformative cell therapies in December marks a shift from experimental concepts to commercial reality, providing new market opportunities for companies like Avant Technologies.
- 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.
- Anti-Aging Therapy Development: Avant's second joint venture, Klothonova, partners with Austrianova to develop anti-aging therapies that restore circulating α-Klotho levels using genetically modified human cells, with potential markets spanning Alzheimer's disease and cardiovascular conditions.
See More
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- Successful Financing: Prime Drink Group has completed a non-brokered private placement raising $450,000, issuing 7,500,015 common shares and the same number of warrants, thereby strengthening its capital base to support future growth.
- 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
PrimeDrink Group Plans Non-Brokered Private Placement to Raise Up to $5 Million
- Funding Objective: PrimeDrink Group Corp. is proceeding with a non-brokered private placement aiming to raise a minimum of $300,000 and a maximum of $5 million to support business development and general working capital needs.
- 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
Prime Medicine Publishes Phase 1/2 Data for PM359, Demonstrating Significant Efficacy
- 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
Prime Medicine Publishes Phase 1/2 Data for PM359, Demonstrating Significant Efficacy
- 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
