Prevail Therapeutics

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

Prevail Therapeutics was delighted to attend and sponsor the recent Gaucher Community Alliance 2024 Patient & Family Conference in Pittsburgh. These events are crucial to bring patients, families, and the Gaucher community together, share experiences, and gain valuable insights. Our team was thrilled to connect with the Gaucher community, fostering education and support. We are committed to advancing research and providing ongoing support for Gaucher disease, and this conference was an incredible way to strengthen those commitments. Thank you to everyone who attended and made this event a success. Together, we continue to build a strong, informed, and supportive Gaucher community. For more information about the Gaucher Community Alliance and their initiatives, visit their website: https://bit.ly/4chYbGI #GCA2024 #GaucherDisease #PatientSupport #CommunityAlliance #PatientConference #GaucherCommunity #RareDisease #PrevailTherapeutics

Join Prevail Therapeutics in spotlighting the important work of the American Society of Gene & Cell Therapy (ASGCT). The ASGCT is at the forefront of gene and cell therapy education, advocating for advancements that transform the practice of medicine and improve patient outcomes. As part of our commitment to advancing gene therapy, Prevail will be attending the ASGCT Policy Summit on September 23–24, 2024, in Washington, DC. This summit provides a crucial platform for collaboration, discussion and advancement of regulatory, legislative, and payment policies that impact the development of gene and cell therapies. To learn more about the ASGCT and their upcoming policy summit, visit their website: https://bit.ly/3B7XgvK #ASGCTadvocacy #GeneTherapy #CellTherapy #Neurodegeneration #RareDiseases #HealthcareInnovation #PrevailTherapeutics

Discover the power of teamwork! The Parkinson’s Foundation is hosting a Wellness Wednesday webinar, focusing on how healthcare professionals collaborate to enhance movement and function for those with Parkinson’s disease (PD). Learn about managing symptoms, using assistive devices, and coping with the physical impact of PD. This session will discuss how rehabilitation professionals, including physical therapists, occupational therapists, speech-language pathologists, and others, work together to maximize functional abilities and quality of life for individuals with PD. Speaker: Terry Ellis, Boston University Sargent College of Health & Rehabilitation Sciences Date: September 18, 2024  Time: 13:00–14:00 EST  Location: Virtual (Zoom) This event is free, but registration is required. Register here: https://bit.ly/3XwgBhw @ParkinsonDotOrg #ParkinsonsDisease #Together4PD #WellnessWednesday #KnowMorePD #PrevailTherapeutics #FreeEvent

Discover the International Gaucher Alliance's (IGA) new podcast series, focusing on living with Gaucher disease. In the inaugural episode, IGA CEO Tanya Collin-Histed introduces the series and invites listeners to suggest future topics. Episode 1 features host Simon Jones discussing gene therapy trials with two patients, sharing their personal experiences and insights. Aligned with the IGA's vision of ensuring all Gaucher patients receive necessary treatment and care, Prevail Therapeutics celebrates the IGA's 30 years of advocacy. We eagerly anticipate the impactful discussions this podcast series will bring to the community. Listen to Episode 1: "Gene Therapy Trials - The Patient's Perspective" on Spotify: https://spoti.fi/3yy5PPt #GaucherDisease #PatientSupport #GeneTherapy #GaucherCommunity #RareDisease #RareButNotAlone

We are delighted to be sponsoring the National Society of Genetic Counselors (NSGC) 43rd Annual Conference from September 17-21, 2024, in New Orleans, LA. This conference is a key gathering for the genetic counseling profession, showcasing new research and other advances, and providing invaluable opportunities for education and networking. The theme, 'Leading Beyond the Horizon', reflects the pivotal role genetic counselors play in shaping the future of genomics and innovation. Join us at #NSGC24 to gain insights into clinical and scientific best practices, explore emerging technologies, and engage in discussions about justice, equity, diversity, and inclusion within the field. We look forward to connecting with colleagues in-person or virtually, sharing knowledge, and exploring actionable strategies to support the growth of genetic counseling.   Register today and be part of this incredible experience. For more information and to register, visit: https://bit.ly/4dzNFf1 #NSGC24 #GeneticCounseling #PrevailTherapeutics #Genomics #ProfessionalDevelopment #Innovation #EquityInHealthcare