CAMBRIDGE -
CF is an inherited genetic disease, which can be caused by more than 1,700 different mutations in the gene that produces the CF transmembrane conductance regulator (CFTR) protein. These mutations, including nonsense and rare mutations, cause the CFTR protein to malfunction or not be made at all; this leads to a buildup of thick mucus, which in turn leads to persistent lung infections, destruction of the pancreas, and complications in other organs. CF affects close to 40,000 people in
Prime Medicine is uniquely positioned to develop potentially curative therapies for CF by using Prime Editing to correct CFTR mutations at the natural genetic locus. Funding from the
In parallel, using PASSIGE, Prime Medicine aims to address nearly all people with CF with a single superexon insertion strategy. This has the potential to restore CFTR expression in individuals' lung cells under native expression conditions, regardless of the underlying CFTR mutation.
'Leveraging our Prime Editing technology, we hope to create a one-time, non-viral therapy, which can precisely correct the underlying genetic mutation that causes CF and potentially offer the first cure for this progressive, devastating disease,' said
About Prime Medicine
Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is leveraging its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated, one-time, potentially curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types.
Prime Medicine is currently progressing a diversified portfolio of eighteen programs initially focused on genetic diseases with a fast, direct path to treating patients or with a high unmet need because they cannot be treated using other gene-editing approaches. Over time, the Company intends to maximize Prime Editing's therapeutic potential and advance potentially curative therapeutic options to patients for a broad spectrum of diseases.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements about Prime Medicine's beliefs and expectations regarding: the Company's position to develop potentially curative therapies for CF by using Prime Editing to correct CFTR mutations at the natural genetic locus; the use of up to
Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including: uncertainties related to the authorization, initiation, and conduct of preclinical and other development requirements for potential product candidates, including uncertainties related to regulatory approvals; risks related to the development and optimization of new technologies, the results of preclinical studies, or clinical studies not being predictive of future results in connection with future studies, including the work with CF; the scope of protection Prime Medicine is able to establish and maintain for intellectual property rights covering its Prime Editing technology; Prime Medicine's ability to identify and enter into future license agreements and collaborations and general economic, industry and market conditions. These and other risks and uncertainties are described in greater detail in the section entitled 'Risk Factors' in Prime Medicine's most recent Annual Report on Form 10-K, as well as any subsequent filings with the
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