Prevail Therapeutics

Prevail Therapeutics is pleased to announce a significant milestone in our Phase 1/2 PROPEL clinical trial for Parkinson’s disease with GBA1 mutations (PD-GBA): Cohort 1 is fully recruited, and we have now initiated enrollment of the 2nd cohort! This marks an exciting step in our journey to find effective treatments for PD-GBA. We extend our deepest gratitude to the dedicated clinical research teams, patient advocacy groups, and, most importantly, the patients and their families who entrust us with their care. As we continue on this journey, Prevail remains driven by our shared passion for improving health outcomes and enhancing quality of life for the rare diseases community. The PROPEL trial is a gene therapy study actively recruiting patients with PD-GBA, ages 35–80 years. To learn more about the PROPEL trial, please visit: https://bit.ly/45MwYud #ParkinsonsDisease #GeneTherapy #PDGBA #ClinicalTrials #ABCsOfPD #GoTeamFox #OutrunPD

At Prevail Therapeutics, we are honored to spotlight the incredible work of the Michael J. Fox Foundation (MJFF). Dedicated to finding a cure for Parkinson's disease, MJFF drives a dedicated research agenda focused on developing improved therapies for those living with Parkinson's today. MJFF exists to accelerate the next generation of Parkinson’s disease treatments by identifying and funding vital projects, solving field-wide challenges, and streamlining efforts of diverse teams. Their mission is to leverage core values of optimism, tenacity, collaboration, boldness, adaptability, and curiosity on behalf of the 6 million people living with Parkinson’s worldwide. Putting patients first, MJFF works tirelessly to speed breakthroughs that make a real difference in patients' lives. They serve as a critical catalyst in the patient and research communities, pushing the most promising treatments forward through innovative approaches. Join us in supporting MJFF's mission to transform the PD research field and bring hope to millions. To learn more about their impactful work, visit: https://bit.ly/3WrdtmE #ParkinsonsDisease #PDResearch #MJFF #FindACure #PatientFirst #InnovativeResearch

We are proud to spotlight the Bluefield Project to Cure FTD, a non-profit foundation committed to advancing the development of treatments for frontotemporal dementia (FTD). The Bluefield Project funds innovative research and fosters collaboration among discovery scientists and clinical neurologists to accelerate the translation of findings into potential therapies. Their contributions are significant: funding over 26 investigators whose work led to two clinical trials and filing of two patent applications. Their investigators have screened nearly 500,000 compounds, published more than 100 papers, and trained over 50 scientists in FTD research. Through partnerships with pharmaceutical and biotech companies, the Bluefield Project is driving forward the development of much-needed therapies for FTD. Learn more about the Bluefield Project and their groundbreaking work: https://bit.ly/4d9jPhk #BluefieldProj #FTD #FrontotemporalDementia #Neurodegeneration #Research #CureFTD #endFTD

Today is World Brain Day!   At Prevail Therapeutics, we are dedicated to raising awareness about the importance of brain health and supporting the fight against neurological disorders. This year, World Brain Day focuses on ‘Brain Health and Prevention’, and is a great opportunity to unite healthcare professionals, researchers, and policymakers in a global movement to promote brain health and preventive care.   Neurological conditions are now the leading cause of ill health and disability worldwide. At Prevail, we are committed to advancing research and providing hope for patients and their families.   Our ongoing PROCLAIM and PROPEL trials are at the forefront of this mission: - PROCLAIM Trial: A Phase 1/2 clinical trial investigating a potentially disease-modifying, single-dose gene therapy for people living with frontotemporal dementia with GRN mutations (FTD-GRN). - PROPEL Trial: A Phase 1/2 clinical trial investigating a potentially disease-modifying, single-dose gene therapy for people living with Parkinson’s disease with GBA1 mutations (PD-GBA).   Join us in recognizing World Brain Day and learn how you can support brain health: https://bit.ly/3Wwoa8H   Visit our website to learn more about our PROCLAIM and PROPEL trials and how we are working to make a difference: https://bit.ly/3Yesrz1   #WorldBrainDay #WBD2024 #BrainHealthandPrevention #BrainHealth #NeurologicalDisorders #FTD #ParkinsonsDisease #PROCLAIM #PROPEL #ClinicalTrials #BrainAwareness

Prevail Therapeutics is committed to developing innovative treatments that offer hope to individuals living with #GaucherDisease (GD). The PROCEED trial is a gene therapy study actively recruiting patients with Type 1 Gaucher Disease (GD1), ages 18-65. To learn more, contact Prevail Therapeutics by email at Prevail.Patients@lilly.com or Jim.Howley@lilly.com. More about the PROCEED trial and our research in GD can be found here: https://bit.ly/4d67sT4 #GaucherDisease #GeneTherapy #ClinicalTrials #RareDiseases #MedicalResearch #GaucherCommunityAlliance #GCA #IGA #InternationalGaucherAlliance

Clinical trials are essential research studies that test new medical treatments in people. These trials can investigate a variety of treatments, including new drugs, surgical procedures, and medical devices. Clinical trials help researchers understand the safety and effectiveness of a new treatment and can explore new ways to prevent, detect, or treat diseases. Each clinical trial is carefully planned and reviewed before it begins and follows a detailed plan called a protocol, which outlines the study's objectives, methods, and organizational structure. Clinical trials are conducted in distinct phases, each designed to answer different research questions about the new treatment. These phases assess how the treatment works, its safety and effectiveness, and how it compares to existing therapies. People of all ages, including children, can participate in clinical trials, contributing to medical advancements. At Prevail Therapeutics, we are continually striving to improve patient outcomes and advance gene therapies for people living with neurodegenerative diseases and rare genetic disorders. We are committed to working with the communities affected by these conditions to advance research through our clinical trials. Join us in recognizing the role of clinical trials in healthcare innovation! To learn more about clinical trials please visit the American Society of Gene and Cell Therapy (ASGCT): https://bit.ly/4cY2Q1f #ClinicalTrials #MedicalResearch #HealthcareInnovation #PrevailTherapeutics #PatientCare

Prevail Therapeutics is pleased to share highlights from the recent educational webinar hosted by the International Gaucher Alliance (IGA) focused on Gaucher Disease Type 1 gene therapy trials. This informative session provided valuable updates on current GD1 clinical trials, including important data on safety and efficacy to help inform the community considering gene therapy. We were delighted to join Spur Therapeutics (formerly Freeline Therapeutics) in presenting at this event and grateful for the opportunity to engage with the community. For those who couldn't attend, you can watch the full recording here: http://tiny.cc/gtwebinar #GaucherDisease #GeneTherapy #ClinicalTrials #PatientEducation #RareDiseases #PrevailTherapeutics #FreelineTherapeutics #InternationalGaucherAlliance #GD1

At Prevail Therapeutics, we are dedicated to providing new options for individuals with neurodegenerative disorders and related rare diseases. We develop potentially disease-modifying gene therapies that target the root genetic causes of conditions, including Parkinson’s disease, frontotemporal dementia, and Gaucher disease. Our approach involves selecting patient populations with specific genetic mutations that we believe can be treated by introducing a healthy copy of a missing or deficient gene. Our gene therapies use adeno-associated virus (AAV) vectors, small non-replicating viruses that act as shuttles to deliver engineered DNA to human cells. Each of our gene therapy candidates is intended to be a one-time treatment to correct the key underlying genetic mutation that we believe drives disease progression. To learn more about gene therapy, visit: https://bit.ly/45Myble #GeneTherapy #NeurodegenerativeDiseases #ParkinsonsDisease #FrontotemporalDementia #GaucherDisease

This month we’re wearing purple! June is Alzheimer's and Brain Awareness Month, a time to join together to raise awareness and support for those affected by Alzheimer's and other dementias. Worldwide, more than 55 million people are living with these challenging conditions. At Prevail Therapeutics, we are committed to advancing research and pioneering novel gene therapies that target the root genetic causes of neurodegenerative disorders, including frontotemporal dementia. We stand with the Alzheimer’s Association in spreading the word by wearing purple and engaging in activities to train and protect your brain. Together, we can make a difference. You can find out more about the Alzheimer's Association here: https://bit.ly/3W4Mej3 #EndAlz #AlzheimersAndBrainAwarenessMonth #BrainHealth #Neurodegeneration #WearPurple

Prevail Therapeutics is a proud sponsor of the inaugural GBA1 Meeting hosted by The Neuro (Montreal Neurological Institute-Hospital) at McGill University from June 27-29 in Montreal! This three-day international conference will bring together leading health professionals and stakeholders from both academia and industry to discuss all aspects of GBA1 research, from basic science to clinical trials.   At Prevail Therapeutics, we are committed to advancing the understanding and treatment of GBA1-associated neurodegenerative diseases. We are pleased to announce that we have completed enrollment in Cohort 1 and initiated enrollment of the 2nd cohort of our ongoing PROPEL trial, a Phase 1/2 clinical study designed to evaluate a potentially disease-modifying, single-dose gene therapy for individuals with #ParkinsonsDisease carrying at least one GBA1 mutation (PD-GBA).   Join us in Montreal for insightful lectures, discussions, and workshops, including a talk from Prevail’s Sebastian Boland on the progress the scientific community has made in developing gene therapies for Parkinson’s disease. Let’s collaborate and network to drive innovation and progress in GBA1 research!   View the preliminary program and register to attend here: https://bit.ly/3RJ3S9u Learn more about our PROPEL trial and our work in PD-GBA by visiting our website: https://bit.ly/45MwYud More about The Neuro here: https://bit.ly/3znJ3dc More about McGill University here: https://bit.ly/4eLiYFo   #GBA1Meeting #GBA1Research #NeurodegenerativeDisease #ClinicalTrials #ParkinsonsDisease #GeneTherapy #PROPELTrial #MichaelJFoxFoundation #ParkinsonsFoundation