Exhibit 99.2
Prime Medicine Announces Recent Progress and Highlights 2023 Strategic Priorities
— New preclinical data in Friedrich’s ataxia and cystic fibrosis provided further proof-of-concept for Prime Editing’s ability to achieve restoration of genetic function —
— New preliminary safety analyses demonstrated no detected off-target activity in Prime Edited Wilson’s disease cells —
— New data leveraging PASSIGE technology demonstrated ability to integrate kilobase-sized DNA in human T cells —
— Additional LNP and AAV delivery data demonstrated efficient in vivo delivery of Prime Editing to various target tissues in rodents —
— Multiple catalysts expected in 2023, including first development candidate nomination in 1Q and additional in vivo data in 2H —
— Strong corporate position, with robust intellectual property position and cash to fund operations into 2025 —
Cambridge, Mass., Jan. 9, 2023 – Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today provided an update on recent advancements of its Prime Editing technology and progress across its initial pipeline of eighteen programs, and outlined its strategic priorities and expected milestones for 2023.
“Since our inception, we have focused on building the necessary foundation to maximize Prime Editing’s therapeutic potential, identifying and progressing a diverse initial pipeline, introducing optimizations to extend the reach of our Prime Editing technology, and investing in the CMC and delivery capabilities that will ultimately be required to deliver our investigational therapies to patients,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. “Today, we are pleased to announce accomplishments across our portfolio and platform, including new preclinical proof-of-concept data in Friedrich’s ataxia and cystic fibrosis showing restoration of genetic function; new safety analyses detecting no off-target editing in Prime Edited Wilson’s disease cells; the first presentation of our ability to use PASSIGE technology to precisely insert a whole gene into human T cells; and further optimization of our lipid nanoparticle and adeno-associated virus delivery platforms. Together, these updates reinforce our belief in Prime Editing as an extremely powerful, differentiated technology, with the potential to deliver one-time, curative genetic therapies to address a wide spectrum of diseases. As we enter 2023, we look forward to progressing our most advanced programs toward the clinic, while continuing to optimize our technology and build the internal organization, culture and expertise necessary to meet our ambitious goals.”
Recent Business Updates
Pipeline
Prime Medicine is advancing a strategic pipeline of eighteen programs. The company is initially focused on indications with the opportunity for the fastest, most direct path to the clinic and technical success in humans, as well as indications that cannot be treated using other gene editing approaches. In vivo studies are progressing across Prime Medicine’s portfolio and, in recent months, the company has established preclinical proof-of-concept and expanded safety data in a variety of target tissues and indications. Today, Prime Medicine is announcing new preclinical data for several programs:
| • | | Friedrich’s Ataxia (FRDA), a multisystem, autosomal recessive neurodegenerative disorder affecting the central and peripheral nervous systems, as well as the heart and other organs. FRDA is caused by GAA-repeat nucleotide sequence expansions in intron 1 of the FXN gene encoding the frataxin protein, which plays important roles in mitochondria. In preclinical studies, Prime Medicine is using its technology to precisely remove the GAA pathological repeats at the FXN gene, restoring Frataxin protein expression and sensory neuron function in patient dorsal root ganglia. |
| • | | Today, Prime Medicine announced new preclinical data demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results in correction of hypermethylation at the FXN gene, restoring genetic function back to wild-type levels. The company believes these data also support the evaluation of Prime Editing for the potential treatment of other repeat expansion diseases, many of which exhibit hypermethylation as a key feature of the underlying pathogenesis. |