CF Foundation provides up to $7.3 million to Ride Therapeutics to use AI to discover genetic therapy delivery vehicles. Ride Therapeutics plans to use its artificial intelligence-powered screening technology to construct and test millions of genetic therapy delivery vehicles and identify the ones that could transport a genetic therapy into the lung cells of people with cystic fibrosis. The Cystic Fibrosis Foundation will provide up to $7.3 million to Ride Therapeutics to identify potential delivery vehicles to carry genetic therapies into the lungs of people with CF. Genetic therapies could one day offer a transformative treatment for everyone with CF, regardless of their mutations, but delivering those treatments into the correct lung cells is a significant challenge. Delivery is difficult because the body's natural defenses are designed to block germs and other foreign invaders from entering lung cells. Ride Therapeutics plans to build a library of millions of tiny particles (nanoparticles) from a variety of materials designed to work safely in the body. Similar to a pill capsule that protects medicines, the nanoparticles are designed to shield and direct genetic materials to cells without damaging the material along the way. Ride plans to test each of the nanoparticles, and the data from these millions of tests will be used to help train an AI system to design the best nanoparticles capable of delivering genetic therapies into the lung cells of people with CF. "The Foundation is committed to supporting multiple approaches to deliver transformative genetic therapies to all people with CF," said Steven M. Rowe, MD, MSPH, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. "This investment is part of our overall strategy to identify the best delivery method to reach target lung cells." In addition to Ride's work with nanoparticles, researchers are investigating other ways to overcome the delivery challenge by exploring options such as harmless viruses and tiny fat particles (lipid nanoparticles). CF scientists are currently focusing on the challenge of delivering genetic therapies to the cells of the lung, because that is one of the organs most severely affected by the disease. Different delivery vehicles would be needed to deliver genetic therapies to other CF-affected organs, such as the intestine or pancreas. Genetic Therapies | Its Research Approach

ReCode Therapeutics, a Menlo Park, California-based clinical-stage genetic medicines company, received an undisclosed investment from the Cystic Fibrosis Foundation. The funding will support a research collaboration to develop a novel gene editing therapy designed to correct mutations in the CFTR gene, the underlying genetic cause of cystic fibrosis. Led by CEO Heather Clark, ReCode uses its proprietary Selective Organ Targeting (SORT) lipid nanoparticle platform to enable precise delivery of genetic medicines directly to organs, tissues, and cells implicated in disease. The technology aims to improve efficacy and potency of mRNA and gene correction therapeutics.

130+ advocates urge Congress to protect NIH funding during 18th annual Teen Advocacy Day. In more than 120 meetings, advocates called on lawmakers to increase funding for the NIH and protect the future of cystic fibrosis research. On June 25, more than 130 advocates from across the country, including more than two dozen living with cystic fibrosis, met with members of Congress and their staff during the Cystic Fibrosis Foundation's annual Teen Advocacy Day, urging them to support increased funding to the National Institutes of Health and protect funding for the vital research driving breakthroughs toward a cure for CF. The event, now in its 18th year, brings together teens, young adults, and families in the CF community to speak up for policies that impact their loved ones living with cystic fibrosis. "The remarkable progress we've seen in cystic fibrosis is driven by sustained investment in science, including a robust commitment to fund NIH and fund essential research," said Mary Dwight, senior vice president and chief policy and advocacy officer of the Cystic Fibrosis Foundation. "We're grateful a new generation of advocates is stepping forward to protect that innovation and continued progress in CF research. Today's advocates' voices are critical to ensuring we continue moving closer to a cure for every person with CF." Alongside advocates on Capitol Hill, more than 1,300 community members from all 50 states participated in the Foundation's Online Day of Action. Together, they sent more than 6,400 messages to lawmakers, amplifying the community's collective voice and reinforcing the urgent need to sustain research funding. Previous Teen Advocacy Day advocates Michael Woody, a young adult living with CF, and Bailee Goldman and Gracie Salema, siblings of people with CF, served as Teen Advocacy Day interns. During their tenure, the group lent their expertise to help craft the event's program and connect with virtual and in-person attendees. Interested in joining the effort to maintain critical CF research? Sign up for the Foundation's advocacy alerts to stay informed and take action. Its Advocacy Work Milestones III Raises More Than $250M to Advance a Cure and Innovative CF Care CF Foundation Commits Up to $13 Million in Additional Funding to Advance Potential Splicing Mutation Treatment FDA Approves Expansion of Two CFTR Modulator Therapies to Additional Rare CF Mutations You might also be interested in...