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Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
-- Prime Editing has potential to be best-in-class approach for AATD -- -- Previously und...
About this update from Prime Drink Group Corp
[{"type":"text","content":"Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)\n\n\n\n\n -- Prime Editing has potential to be best-in-class approach for AATD --\n \n\n\n\n -- Previously undisclosed program now emerging from within liver platform; leverages proprietary, universal liver LNP --\n \n\n\n\n -- High levels of editing at the target site in preclinical studies with full restoration of circulating wild-type AAT protein (M-AAT) to normal human range --\n \n\n\n\n -- Expects to file IND and/or CTA in mid-2026 --\n \n\n\n CAMBRIDGE, Mass., March 18, 2025 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today unveiled a preclinical program for the treatment of alpha-1 antitrypsin deficiency (AATD), the next program within its liver franchise. Prime Medicine expects to file an investigational new drug (IND) and/or clinical trial application (CTA) in mid-2026.\n \n\n “We are excited to announce our AATD program, the second high-value program to emerge from our liver franchise, and the second liver program that we expect to advance into the clinic next year,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. “This program exemplifies our strategy of using our proprietary, modular liver LNP to accelerate the development of new Prime Editors, as well as our ability to leverage learnings, regulatory frameworks and manufacturing synergies to efficiently advance our efforts. We look forward to progressing our AATD and Wilson’s Disease programs toward clinical data in 2027, where we hope to demonstrate Prime Editing’s best-in-class potential across two of the largest genetic liver diseases.”\n \n\n Dr. Gottesdiener continued, “We are also pleased to share the first\n \n in vivo\n \n preclinical data for our AATD program. These data reinforce the potential of Prime Editing to restore the disease-causing mutation back to wild-type and address the underlying pathology of both lung and liver manifestations of AATD, without the risk of bystander edits or detectable off-target edits.”\n \n\n\n Prime Medicine’s Approach to AATD\n \n\n\n Prime Medicine’s program leverages the Company’s universal l...