Latest news with #CysticFibrosisFoundation
Business Wire
16-07-2025
- Health
- Business Wire
Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to Develop Gene Editing Therapy
BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of a gene editing therapy for people with cystic fibrosis (CF). Prime Medicine uses a gene editing technology called prime editing — a technology that enables a wide range of modifications to the DNA with a high degree of precision. The company — founded by Drs. David Liu and Andrew Anzalone, who pioneered the development of this unique editing technology — is investigating whether prime editing could treat several diseases, including CF. The CF Foundation's initial investment in Prime Medicine supported work to demonstrate the versatility of prime editing to correct multiple CF-causing mutations in the lab. This additional investment by the CF Foundation will focus on the development of a prime editing therapy targeting the nonsense mutation G542X — one of the most prevalent CF-causing nonsense mutations and one for which there are no available therapies. 'We believe gene editing offers the best hope for a cure for cystic fibrosis because it could permanently correct the mutations that cause this disease,' said Michael P. Boyle, MD, president and chief executive officer of the Cystic Fibrosis Foundation. 'Our investment will help support gene editing research for CF through early development stages, including efforts to overcome the challenges of delivery to the lungs.' As part of its ongoing efforts to develop a prime editing therapy, Prime Medicine is investigating the best way to deliver its gene editing treatment to the lungs of people with CF, a major challenge because of the thick, sticky mucus that clogs the airways and the body's natural immune response to block foreign entities from entering cells. If successful, the gene editing cargo will enter the lung cells that normally make CFTR protein and correct the CFTR mutation in the cell's DNA, resulting in a potentially permanent fix to the CFTR gene. With continued advancement in prime editing and delivery to the lungs, this technology may eventually be used to correct many types of CF mutations. Prime Medicine is advancing multiple approaches in CF, including 'hotspot,' which uses prime editing to make smaller corrections to specific CFTR mutations and PASSIGE, which uses prime editing to make large gene insertions. These combined strategies eliminate the need to develop new prime editing therapies for each individual mutation. Previously, the Foundation committed up to $15 million to Prime Medicine to pursue development of their gene editing technology for CF. About the Cystic Fibrosis Foundation The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit
Yahoo
16-07-2025
- Business
- Yahoo
Prime Medicine Announces Additional Funding of Up to $24 Million from the Cystic Fibrosis Foundation to Advance Prime Editing Treatments for Cystic Fibrosis
-- Expanded funding builds on the initial agreement to accelerate development of Prime Editors for Cystic Fibrosis (CF) -- -- Prime Medicine to receive up to $24 million in additional funding from the Cystic Fibrosis Foundation -- -- Multiple hotspot Prime Editors may benefit more than 93% of people with CF -- CAMBRIDGE, Mass., July 16, 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 announced that the Cystic Fibrosis Foundation (CF Foundation) has agreed to provide the Company with up to $24 million in additional funding to accelerate the development of Prime Editors designed to permanently correct cystic fibrosis-related lung disease. The CF Foundation's additional investment builds on initial funding received under Prime Medicines' January 2024 agreement, and reflects its interest in Prime Editing as a potentially curative approach for CF. Prime Editing can correct a wide range of genetic mutations, and Prime Medicine intends to leverage the technology's versatility and modularity to address multiple disease-causing mutations, potentially treating the vast majority of people with CF. Prime Medicine will initially focus on a program targeting G542X, one of the most prevalent CF-causing nonsense mutations and one for which there are no available therapies. In addition, the Company will continue to advance hotspot and PASSIGE-based approaches for other mutations with funding received from the CF Foundation under its initial commitment in 2024. 'We are honored to receive continued support from the CF Foundation, whose longstanding commitment to driving innovation has reshaped the treatment landscape for people living with CF,' said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. 'This additional funding reflects our shared belief in the transformative potential of Prime Editing therapy for people with this devastating genetic disease, especially for those where the current standard of care is either ineffective or poorly tolerated. This funding also exemplifies our strategic approach to business development, and our commitment within Prime Medicine to leveraging external resources to accelerate innovation and ensure the broadest application of our technology.' The CF Foundation will provide Prime Medicine with up to $24 million in two tranches, subject to certain closing conditions and scientific milestones; the first tranche includes a $6 million equity investment in Prime Medicine. About Cystic Fibrosis Cystic fibrosis (CF) is a serious, inherited genetic disease caused by mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. These mutations result in reduced or absent function of the CFTR protein, which is critical for maintaining the balance of salt and fluids across epithelial surfaces in organs such as the lungs, pancreas, and gastrointestinal tract. When CFTR is not functioning properly, thick, sticky mucus accumulates, leading to chronic lung infections, progressive respiratory decline, and impaired digestion and nutrient absorption. CF affects approximately 100,000 people globally, including over 40,000 in the United States. While disease-modifying therapies have improved outcomes for many individuals, they are not curative and are ineffective for some people with certain mutations. There remains a significant unmet need for a one-time, potentially curative treatment that can address the underlying genetic cause of CF across diverse genotypes. 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 deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time 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. Taken together, Prime Editing's versatile gene editing capabilities could unlock opportunities across thousands of potential indications. Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: liver, lung, and immunology and oncology. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing's broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit © 2025 Prime Medicine, Inc. All rights reserved. PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are trademarks of Prime Medicine, Inc. All other trademarks referred to herein are the property of their respective owners. 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 agreement with the CF Foundation and the intended and potential benefits thereof, including the receipt of payments based on scientific milestones; the potential for Prime Editing to correct the causative mutations of diseases, including CF; the potential for Prime Editing to treat the vast majority of people with CF; the continued advancement of hotspot and PASSIGE-based approaches for correcting mutations other than G542X; the breadth of Prime Editing technology and the implementation of its strategic plans for its business, programs, and technology; and the potential of Prime Editing as a transformative gene editing technology and its ability to unlock opportunities across thousands of potential indications 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, without limitation, risks associated with: uncertainties related to Prime Medicine's product candidates entering clinical trials; the authorization, initiation, and conduct of preclinical and IND-enabling studies and other development requirements for potential product candidates, including uncertainties related to opening INDs and obtaining 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; 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; Prime Medicine's expectations regarding the anticipated timeline of its cash runway and future financial performance; 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 Securities and Exchange Commission. In addition, any forward-looking statements represent Prime Medicine's views only as of today and should not be relied upon as representing its views as of any subsequent date. Prime Medicine explicitly disclaims any obligation to update any forward-looking statements subject to any obligations under applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Investor and Media Contacts Gregory DearbornPrime Medicine857-209-0696gdearborn@ Hannah DeresiewiczPrecision in to access your portfolio
Yahoo
09-06-2025
- Entertainment
- Yahoo
Foodies unite, raise money for cystic fibrosis at Chef's Challenge
Local chefs will cook sweet and savory bites, vying for big honors all while raising money for a great cause at the Cystic Fibrosis Foundation's Chef's Challenge.
Yahoo
11-05-2025
- Health
- Yahoo
Cystic Fibrosis walk raises $33K in Morgantown
MORGANTOWN, (WBOY) — A little more than $33,000 was raised on Saturday morning in Morgantown to help fund cystic fibrosis research, which will help extend the lives of CF patients. 'Great Strides' walks around the country are organized each year by the Cystic Fibrosis Foundation (CFF), which has worked since 1955 to raise awareness for CF as well as raise money for the research needed to develop new treatments for the disease. David Dent, an assistant swim coach for West Virginia University, was recently diagnosed with the disease in April, but said he's already starting to feel an improvement from his symptoms since he began treatment a few weeks ago. 'I was diagnosed on April 11th of this year, and it's been a whirlwind to get to this point, actually,' Dent said. 'It's a lot of years of just being told that I just had a cold or my anxiety was driving my sickness and things like that.' The CFF estimates that more than 40,000 people in the United States are living with cystic fibrosis, a genetic disease that affects the mucous membranes in the body. This can have severe impacts on a patient's lungs, intestines, liver, reproductive organs and more. In 1999, the life expectancy of a CF patient was around 30 years, but continued research has raised the median life expectancy for recently diagnosed CF patients to 60 years or more. One thing that makes cystic fibrosis a challenging disease to treat is the number of genetic variations for the disease; there are more than 2,000 known mutations according to the Cystic Fibrosis Trust, meaning some treatment options will be effective for some mutations but not others. Every West Virginia monster festival in 2025 'Once I got diagnosed, I saw that there was a walk coming up pretty quickly right after my diagnosis, and decided that I wanted to help out,' Dent said. 'I do have the mutations that qualified me for some of the best treatment in the cystic fibrosis community, and so luckily I was able to jump in right away and start treating it, but there's a lot that still aren't able to.' However, those patients who are able to benefit from the latest developments in CF medicine are seeing large improvements in their overall health. Associate Director of the Mountain State Cystic Fibrosis Center Dr. Kathryn Moffett said the latest drugs have been a 'game changer' for many people with CF, and those treatments are a direct result of fundraisers like the one held in Morgantown on Saturday. 'It has totally changed this disease,' Moffett said. 'Patients are healthy, we don't have patients in the hospital anymore. They get chronic infections, but [they don't need] to come in anymore for a course of, say, two weeks of IV antibiotics. It's amazing.' As of Saturday evening, the fundraiser for the Great Strides walk is still ongoing. Anyone who wishes to donate can find the Morgantown walk's fundraiser page here. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
08-05-2025
- Health
- Yahoo
36th annual Great Strides walk in Bethesda raises awareness for cystic fibrosis
MONTGOMERY COUNTY, Md. (DC News Now) — Many will gather in Bethesda Wednesday evening to join the 5K walk to raise awareness on finding a cure for cystic fibrosis. On May 7, the Cystic Fibrosis Foundation will host its 36th annual Great Strides walk at the Elm Street Urban Park. Check-in for the walk will begin at 5:30 p.m., and the run will begin at 6:30 p.m. Organizers noted that Great Strides is the Cystic Fibrosis Foundation's largest annual fundraiser. Each year, thousands come together to support the mission to cure cystic fibrosis and provide people living with the disease an opportunity to have a long, fulfilling life. DC small business owners take advantage of resources at annual 'POWERUP' summit 'Together, we can raise awareness and fund crucial scientific research, making a difference in the lives of those battling this condition,' wrote the Cystic Fibrosis Foundation in a release. Cystic fibrosis is a genetic disease that causes breathing problems and digestive issues, while impacting a person's lungs and other vital organs. According to the U.S. Centers for Disease Control and Prevention (CDC), the disease affects about 35,000 people across the U.S. Organizers said the money raised during the walk will go toward the Cystic Fibrosis Foundation to complete research and care needed to help advance a cure for the disease. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed. For the latest news, weather, sports, and streaming video, head to DC News Now | DC, Virginia, Maryland News, Weather, Traffic, Sports Live.
