Prevail Therapeutics

Next week, our very own Jim Howley, Global Patient Advocacy and Engagement Lead at Prevail Therapeutics, along with the International Gaucher Alliance and some of the world’s most influential Gaucher Disease advocates will gather to celebrate how far we have come and to plan the next leg of the journey. The biennial International Gaucher Alliance Members’ Meeting will be held in Lisbon, Portugal, on November 9th-10th. It is an opportunity for member organizations to collaborate with the IGA team, help guide the alliance’s future work – and celebrate its 30th anniversary. The IGA was founded in 1994, to help people with GD who lacked access to treatments by working with pharmaceutical companies to develop charitable access programs. Today, it is an international umbrella group that represents the interests of patients with GD and groups from around the world. Visit the alliance’s website in the coming weeks to read the key insights from the Members Meeting. https://bit.ly/4hv9O0K #IGABiennial2024 #patientadvocacy #30thanniversary #GaucherDisease #GD

Prevail was proud to attend the FDA’s Rare Disease Innovation Hub public meeting on October 16th, 2024.   The FDA’s Rare Disease Innovation Hub is designed to engage with the rare disease community, including patient and caregiver groups, trade organizations, and scientific and academic institutions. Despite recent advances in rare disease treatments, significant work remains. The Hub's ultimate goal is to enhance patient outcomes by promoting cross-center collaboration to address common challenges and facilitate the development of rare disease products. Prevail remains committed to improving health outcomes and enhancing the quality of life for the rare disease community.  To learn more about this ground breaking initiative, please visit: https://bit.ly/48l5fC5     #RareDisease #Innovation #Healthcare #PatientAdvocacy #Research  

Prevail congratulates Aparito - a wholly owned subsidiary of Eli Lilly and Company, on celebrating its 10-year anniversary. This marks a decade of pioneering advancements in healthcare technology. Aparito digitizes clinical trials and accelerates drug development for patients with life-limiting diseases by supporting patient-centric clinical trials. Here’s to another decade of growth, innovation, and impactful change!  To learn more about Aparito, please visit: https://bit.ly/3AiCcT5 #Aparito #EliLilly #DigitalHealth #HealthTech #RareDisease #Innovation

We can’t wait to share the latest information on our investigational gene therapy for Gaucher Disease at ESCGT 2024 in Rome!   Sarah Neuhaus, a senior medical director at Prevail, will talk about the PROCEED trial, a Phase 1/2 dose-finding study that is evaluating our AAV9-based therapy for the peripheral manifestations of type 1 GD. Olga Uspenskaya, Prevail Therapeutics' Vice President of Clinical Development will also have a poster, looking at data from the PROCLAIM trial for frontotemporal dementia, on display during the meeting.   Follow the link for more information https://bit.ly/4eSPBQS   #ESCGT #ESCGT2024 #rarediseases #genetherapy #gaucherdisease #GD

Could you be a part of the next breakthrough? At Prevail Therapeutics, we are passionate about advancing the conversation on genetic testing for rare diseases. Genetic testing involves analyzing a person’s DNA to identify changes or mutations in their genes or chromosomes, which can help in understanding and managing various conditions. Genetic testing can identify many disorders, including frontotemporal dementia (FTD). FTD is caused by mutations in the GRN gene in about 5–10% of cases. We are excited to highlight the Prevail and Sano Genetics Program that you can participate in from the comfort of your home. This study aims to better understand the genetics of FTD, empowering researchers to potentially identify treatment options. Free at-home genetic testing and counselling before and after your test are provided. To learn more and enroll visit: https://bit.ly/4f3y9ca Together, we can make a difference in the lives of those affected by FTD. #GeneticTesting #NeurodegenerativeDiseases #FrontotemporalDementia #PrevailTherapeutics #Research #Breakthroughs #AtHomeTesting #GeneticCounseling

Today is International Gaucher Day! It is the perfect opportunity for all of us in the Gaucher Disease community to raise awareness of this rare condition. This International Gaucher Day we are also sending our warmest birthday wishes to the International Gaucher Alliance, which has now been amplifying the voices of people living with GD for 30 years. To mark the occasion, supporters from all over the world have filmed themselves talking about how working with the patient advocacy group has helped them to manage their health and support each other. Hear their stories and discover ways you can support International Gaucher Day here https://bit.ly/4eC6uPy #GaucherDisease #PatientAdvocacy #PatientAdvocacyGroup #rarediseases #IGD #InternationalGaucherDay #GD

The International Congress of Parkinson and Movement Disorders (MDS) is committed to tackling unmet medical need – and, at Prevail Therapeutics, so are we. This year’s MDS theme is 'Movement Disorders in the Digital Age: Harnessing AI for Better Lives’. It will bring some of the world’s leading movement disorder clinicians, scientists, and healthcare professionals together to share groundbreaking research and multidisciplinary approaches to care and therapeutics. We are so excited to be part of it. PROPEL, our phase I/II clinical trial in Parkinson's Disease with GBA1 mutations (PD-GBA), has recently started enrolling for Cohort 2. Our candidate is a potentially disease-modifying, single-dose gene therapy. If you are attending MDS, please reach out. We look forward to sharing how we plan to drive innovation in gene therapy, and advance treatment options for people living with neurodegenerative and rare diseases. If you would like more information on the PROPEL trial, please visit our website https://bit.ly/3LB3Rkm #MDSCongress #MDSM #ParkinsonsDisease #GeneTherapy #NeurodegenerativeDisorders #PrevailTherapeutics #HealthcareInnovation

It’s time for the International Congress of Parkinson and Movement Disorders (MDS) – where Prevail Therapeutics will share data on our work to develop an RNA therapy with the potential to slow or even halt neurodegeneration and progression in Sporadic Parkinson’s disease (sPD). Our investigational, small interfering RNA (siRNA) therapy has recently entered a Phase I clinical trial. It has been designed to target α-synuclein mRNA and reduce α-synuclein protein aggregation. PROSPECT, a first-in-human single and multiple ascending dose trial, will evaluate its safety, tolerability, and PK/PD. With current PD treatments being symptomatic and providing diminishing benefit and increasing side effects over time, disease-modifying therapies are desperately needed. If you are attending MDS, you can visit us at our poster 762 on Sunday September 29 from 1-3pm and speak to a member of our clinical team. #MDSCongress #MDSM #ParkinsonsDisease #GeneTherapy #siRNA #NeurodegenerativeDisorders #PrevailTherapeutics #HealthcareInnovation

Prevail Therapeutics was proud to be part of 14th International Conference on Frontotemporal Dementias (ISFTD2024), held from September 19-22 in Amsterdam, the Netherlands. This vital gathering brought together leading neurologists, psychiatrists, geriatricians, neuropsychologists, and basic scientists in the dementia and FTD fields. Our Prevail team engaged with fellow professionals, shared insights, and discussed the latest advancements in understanding the molecular basis of FTD and developing therapeutic approaches. We were honored to present our latest research and innovations, further highlighting our commitment to the FTD community. Highlights from the conference included keynote speeches, panel discussions, poster presentations, and invaluable networking opportunities. The caregiver’s program provided crucial support and information for those caring for individuals with FTD. As sponsors, we are deeply committed to advancing research and therapeutic development for FTD. Thank you to everyone who made this event a success. To discover more about our work in FTD and our PROCLAIM trial, visit: https://bit.ly/3WOrBrL International Society for Frontotemporal Dementia (ISFTD) #ISFTD2024 #FTD #DementiaResearch #Neurology #PrevailTherapeutics #HealthcareInnovation #PatientSupport

World FTD Awareness Week is here! Between September 22-29, we are coming together with the Association for Frontotemporal Degeneration to raise awareness of FTD. FTD is the most common form of dementia for people under 60. FTD affects the frontal and temporal lobes of the brain, leading to changes in behavior, personality, language, and motor skills. Despite being a devastating diagnosis, FTD often goes undiagnosed or misdiagnosed. That’s why it is so important to shine a light on those affected and work together to drive research forward. Prevail Therapeutics places ground-breaking research and innovative gene therapies at the core of our mission, targeting the root genetic causes of FTD and other neurodegenerative disorders. 👉 Visit The Association for Frontotemporal Degeneration (AFTD) website for resources, research updates, and ways to get involved: https://bit.ly/47zHoyg You aren’t alone. We’re in this together. #endFTD #FTDawareness #TogetherforFTD