- Prime Medicine is focused on advancing its **prime editing technology**, a versatile and safe gene editing approach that makes single-stranded DNA breaks, avoiding off-target editing and chromosomal rearrangements.  **[0001894562_2181298_1]** 
- The company has **reprioritized its pipeline** to liver programs, with **Wilson's disease** (PM577) and **alpha-1 antitrypsin deficiency** expected to enter the clinic in the first half and mid-**2026**, respectively, with Wilson's data anticipated in **2027**.  **[0001894562_2181298_1]** **[0001894562_2181298_5]** 
- Despite strong initial clinical data, Prime Medicine **discontinued its chronic granulomatous disease (CGD) program** due to its ultra-orphan nature and limited commercial opportunity.  **[0001894562_2181298_2]** 
- Prime Medicine reported a cash runway of approximately **$260 million** at the end of Q2 2025, which is projected to extend into **2027**.  **[0001894562_2181298_8]**

Prime Medicine Discusses Pipeline Reprioritization and Clinical Timelines

- Prime Medicine's CEO, Allan Reine, outlined the company's refined pipeline, focusing on **Wilson's disease**, **Alpha-1 antitrypsin deficiency (AATD)**, and **cystic fibrosis (CF)**, in addition to a collaboration with BMS for CAR-T therapies.  **[1894562_tx_379959_0]** 
- The company anticipates filing an **Investigational New Drug (IND) application for Wilson's disease in the first half of 2026** and for **AATD in mid-2026**, with clinical data for both programs expected in **2027**.  **[1894562_tx_379959_0]** **[1894562_tx_379959_4]** 
- Prime Medicine reported **positive clinical data** from two patients in its chronic granulomatous disease (CGD) program, showing **rapid engraftment** and **high levels of functional cure biomarkers**, but will discontinue the program due to the small patient population, while still engaging with the FDA.  **[1894562_tx_379959_1]** 
- The company's **cash runway extends into 2027** following a recent financing in August.  **[1894562_tx_379959_10]** 
- Prime Medicine estimates the global market opportunity for **Wilson's disease** to be **$20 billion to $40 billion**, and for **AATD** to be **$20 billion to $40 billion plus** in the US and Europe.  **[1894562_tx_379959_5]** **[1894562_tx_379959_3]**

Prime Medicine (PRME)