Prime Medicine to Use Gene Editing for Various Conditions

Fresh from receiving FDA clearance for a clinical trial involving an ex vivo prime editing candidate aimed at treating a rare disease, Prime Medicine recently outlined its plans for next-generation gene editing therapies at the American Society of Gene & Cell Therapy's annual meeting in Baltimore. 

During a workshop focused on precision genome editing technologies, Prime Medicine showcased preclinical data, emphasizing the wide-ranging potential of its prime editing technology. This innovative approach utilizes CRISPR technology to correct defective genes without breaking both strands of DNA. Jonathan Levy, Prime Medicine’s principal scientist for platform delivery innovation, highlighted that prime editors could potentially rectify nearly all types of genetic mutations across various tissues, organs, and cell types, addressing thousands of potential medical conditions. "Off-target editing is very low," Levy stated during his presentation about the development and delivery of prime editors. "For our lead programs, we show no detectable off-targets."

The technology, which was first elaborated in a 2019 Nature paper, has since evolved to include dual-flap prime editors for larger genetic insertions and deletions. Additionally, the Prime Assisted Site Specific Integrase Gene Editing (PASSIGE) system integrates large genetic payloads by utilizing a recombinase target sequence. With these advanced platforms, Prime Medicine is developing a pipeline of investigational therapies targeting hematology, immunology, liver, lung, ocular, and neuromuscular diseases.

On April 29, the Cambridge, Massachusetts-based biotech firm disclosed that the FDA had cleared a study involving a drug candidate from its prime editing platform. This marks the first time the agency has approved the use of this genetic technology in humans. Prime Medicine aims to share initial data next year from a Phase I/II trial of PM359, an ex vivo candidate designed to correct a common mutation that causes chronic granulomatous disease, a rare inherited blood disorder.

To deliver its therapies, Prime Medicine is employing multiple delivery vehicles, including electroporation, lipid nanoparticles (LNPs), and adeno-associated viruses (AAVs). The company is particularly enthusiastic about developing novel LNPs, leveraging a proprietary library of over 800 lipids. "We’re very excited about the potential of novel LNPs for hepatic and non-hepatic delivery," Levy mentioned, aiming for a universal LNP that could be used across various medical conditions.

In January 2024, the company secured $15 million from the Cystic Fibrosis Foundation to advance the development of prime editors for treating cystic fibrosis (CF). Under this agreement, Prime Medicine plans to employ "hotspot editing" and the PASSIGE system to address nearly all CF patients with a single superexon insertion strategy. This approach has the potential to restore CF transmembrane conductance regulator (CFTR) expression in lung cells under natural conditions, regardless of the specific underlying CFTR mutation.

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