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On Thursday, Chardan Capital Markets adjusted its price target for Prime Medicine (NASDAQ:PRME) shares, reducing it to $16.00 from $17.00, while reaffirming a Buy rating for the stock. Currently trading at $2.00 with a market capitalization of $255 million, the stock sits within a broader analyst target range of $6 to $19. According to InvestingPro data, Prime Medicine’s shares have seen significant volatility, with a beta of 1.85. The revision follows Prime Medicine’s recent disclosure of progress in a prime editing program aimed at treating alpha-1 antitrypsin deficiency (AATD), a condition that is not considered rare. The company is on track to file an Investigational New Drug (IND) application or Clinical Trial Application (CTA) by mid-2026.
Prime Medicine, selected as a Chardan Biotech Top Pick for 2025, has separated this specific indication from its broader early-stage in vivo editing efforts within its financial modeling. The program, already accounted for in the company’s cash guidance, could position Prime as a strong candidate in the competitive AATD market. InvestingPro analysis shows the company maintains a healthy current ratio of 5.58, though its overall financial health score remains weak. Investors can access detailed financial metrics and 12+ additional ProTips with an InvestingPro subscription. AATD is linked to mutations in the SERPINA1 gene, leading to lung disease from a lack of circulating AAT and liver complications due to the accumulation of abnormal AAT in hepatocytes.
The company’s approach involves prime editing of hepatocytes to revert the E342K (PiZ) mutation, which is present in over 95% of patients with severe AATD, to the normal M form. This strategy aims to address both lung and liver pathologies. Prime Medicine employs its universal GalNAc-LNP delivery system, also used in its PM577 asset for Wilson’s disease, to administer the treatment. Preliminary data shows that the treatment corrected the mutation in approximately 50 to 70% of hepatocytes in mice, leading to up to 95% restoration of serum AAT to the M-AAT form, achieving levels within the normal human range.
The company’s success in mutation correction and restoration of serum AAT levels to nearly normal ranges is significant, as the normal range in healthy humans is 20-48µM, and 11µM is considered the protective threshold. Prime Medicine’s update indicates a potential breakthrough in the treatment of AATD, with the company’s prime editing technology showing promise in preclinical studies. With the next earnings report due on May 9, 2025, investors seeking comprehensive analysis can access Prime Medicine’s detailed Pro Research Report, available exclusively on InvestingPro, along with in-depth coverage of 1,400+ other US stocks.
In other recent news, Prime Medicine has announced progress in its preclinical program for a gene editing therapy aimed at treating alpha-1 antitrypsin deficiency (AATD), a genetic disorder with no current curative treatments. The company plans to file for regulatory approval to begin human trials by mid-2026, utilizing its proprietary lipid nanoparticle delivery system to correct the E342K mutation in the SERPINA1 gene. Prime Medicine’s preclinical studies showed promising results, with up to 72% correction of the mutation in hepatocytes and restoration of normal protein levels in a humanized mouse model.
In addition, Prime Medicine is advancing PM359, a therapy targeting chronic granulomatous disease (CGD), with its Phase 1/2 clinical trial already underway in the United States. The therapy has received rare pediatric and orphan drug designations, highlighting its potential in treating this rare disease. Analyst firms such as H.C. Wainwright and JMP Securities have maintained their positive outlooks on Prime Medicine, both setting a price target of $10.00 and emphasizing the company’s innovative Prime Editing technology.
The analysts underscored the significance of Prime Medicine’s advancements in genetic editing, which could offer transformative treatment options for genetic disorders like AATD and CGD. These developments reflect Prime Medicine’s ongoing efforts to deliver innovative healthcare solutions for diseases with significant unmet medical needs.
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