Prevail Therapeutics

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

The GARDIAN Registry has released data on the first 26 patients: 96% with Gaucher disease Type 3 (GD3) and 4% with Type 2 (GD2). GARDIAN (Gaucher Registry for Development, Innovation, and Analysis of Neuronopathic Disease) is a global initiative by the International Gaucher Alliance (IGA). It aims to improve understanding of GD2 and GD3 by collecting personal experiences from patients and families. At Prevail Therapeutics, we celebrate the remarkable work of the IGA as we recognize the critical need to include patient and caregiver voices throughout the drug development process. By collaborating with the IGA to help support GARDIAN, we hope to develop a more authentic understanding of the patient experience that will improve current drug development efforts. Discover more about the results here: https://bit.ly/4eipC5w #GARDIANRegistry #GaucherDisease #InternationalGaucherAlliance #PatientDrivenResearch #InnovationInHealthcare

Prevail Therapeutics is proud to sponsor the Inaugural End the Legacy Genetic ALS & FTD Community Summit, which runs from today until Sunday June 9th at Northwestern University in Chicago, Illinois. Hosted locally by the Les Turner ALS Foundation, this summit offers attendees training from top research clinicians through the Northeast ALS Consortium (NEALS) Clinical Research Learning Institute curriculum. This training empowers individuals living with ALS and their loved ones with essential knowledge about clinical research. The event will also feature three panels focused on developing skills crucial for a robust genetic ALS & FTD patient advocacy movement. Prevail Therapeutics is committed to advancing treatments and enhancing outcomes for the ALS and FTD community. We look forward to engaging with patients, patient advocates, ALS experts, and everyone impacted by genetic ALS and FTD at the summit! We hope to see you there! Learn more and register here: https://bit.ly/3VxfeQb #GeneticALS #FTD #ALSAwareness #FTDAwareness #PatientAdvocacy #AFTD

Prevail Therapeutics is thrilled to announce the publication of interim data from our PROCLAIM Phase 1/2 clinical trial in Nature Medicine. The PROCLAIM trial is investigating a potential disease-modifying, single-dose gene therapy designed to elevate progranulin levels for individuals living with #FrontotemporalDementia with GRN mutations (FTD-GRN). At Prevail, we are dedicated to increasing our understanding of FTD-GRN and exploring the possibilities of how gene therapy could address the underlying genetic causes. We are committed to advancing our research to improve the outcomes for individuals affected by FTD. Read the full article in Nature Medicine here: https://lnkd.in/eVrV7bfs To learn more about PROCLAIM, visit our website: https://bit.ly/3QVktqt #FrontotemporalDementia #GeneTherapy #FTD #ClinicalTrials #NeurodegenerativeDiseases #BiomedicalResearch

Prevail Therapeutics is thrilled to announce the publication of interim data from our PROCLAIM Phase 1/2 clinical trial in Nature Medicine. The PROCLAIM trial is investigating a potential disease-modifying, single-dose gene therapy designed to elevate progranulin levels for individuals living with #FrontotemporalDementia with GRN mutations (FTD-GRN). At Prevail, we are dedicated to increasing our understanding of FTD-GRN and exploring the possibilities of how gene therapy could address the underlying genetic causes. We are committed to advancing our research to improve the outcomes for individuals affected by FTD. Read the full article in Nature Medicine here: https://lnkd.in/eVrV7bfs To learn more about PROCLAIM, visit our website: https://bit.ly/3QVktqt #FrontotemporalDementia #GeneTherapy #FTD #ClinicalTrials #NeurodegenerativeDiseases #BiomedicalResearch

Prevail Therapeutics is excited to be participating in the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Baltimore. Our team of presenters eagerly anticipates the chance to showcase posters covering a variety of significant topics in gene and cell therapy research. Discover more about our contributions by exploring poster numbers 457, 472, 575, 1012, 1013, 1034, and 1110 in the abstract booklet available here: https://bit.ly/3y3tpTP   Join us as we discuss the latest advancements in this field at #ASGT2024. #GeneTherapy #CellTherapy