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Prime Medicine (PRME) investor relations material

Prime Medicine Morgan Stanley 23rd Annual Global Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Morgan Stanley 23rd Annual Global Healthcare Conference summary8 Sep, 2025

Strategic priorities and capital allocation

  • Focused on programs with high probability of early de-risking, unmet medical need, and strong commercial potential, especially two liver programs: Wilson’s disease and Alpha-1 antitrypsin deficiency.

  • Cystic fibrosis program is advancing with funding from the Cystic Fibrosis Foundation; delivery remains a challenge but progress is ongoing.

  • Ex vivo CAR-T cell therapy collaboration with Bristol Myers Squibb is progressing, with potential for future proprietary and partnered programs in neurological and other diseases.

  • Long-term vision includes leveraging Prime Editing’s unique capabilities for neurological and other high-value indications.

  • Capital allocation is focused on core programs, with future expansion possible as opportunities arise.

Differentiation and technology platform

  • Prime Editing offers safer, more precise gene editing compared to CRISPR-Cas9, avoiding double-stranded breaks and off-target effects.

  • The technology enables writing multiple base pairs, expanding the range of treatable diseases beyond what base editing or CRISPR can achieve.

  • Regulatory outlook is favorable due to the platform’s ability to restore wild-type protein without bystander edits, potentially easing approval pathways.

Clinical and preclinical program updates

  • First clinical program in chronic granulomatous disease showed high editing efficiency and strong biomarker response, but limited commercial opportunity led to no further investment.

  • Plans to discuss potential regulatory paths with the FDA for this program within the next six months.

  • High editing efficiency demonstrated in multiple tissue types; delivery is the main focus for in vivo applications, with lipid nanoparticles used for liver targeting.

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Frequently asked questions

Prime Medicine, Inc. is a biotechnology company specializing in next-generation gene editing technologies aimed at developing curative therapies for a broad range of genetic diseases. The company utilizes its proprietary Prime Editing platform, a highly precise and versatile method of correcting disease-causing mutations in DNA, targeting nearly 90% of known genetic mutations. Prime Medicine's therapeutic focus includes areas like hematology, immunology, liver, ocular, and neuromuscular diseases. The company is headquartered in Cambridge, Massachussetts, and its shares are listed on the NASDAQ.

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