Today, on #RareDiseaseDay, we join the global effort to amplify the voices of a resilient?community of nearly 300 million people worldwide living with a rare disease. By listening, learning, and collaborating with communities and advocates, we help shape a future where those impacted by rare diseases are supported, empowered, and heard.
关于我们
Denali Therapeutics is dedicated to defeating neurodegenerative diseases by breaking through historical barriers in scientific research and clinical development in order to deliver safe and effective medicines to patients and families. Our scientific approach is based on three core principles: rigorous assessment of genetic targets, engineering brain delivery, and using biomarkers to guide development. Our team thrives in a work environment that is scientifically driven, impact-focused, supportive, and collaborative. Our ability to have a positive impact on people’s lives is directly related to the trust we have in each other and our ability to unify our diverse backgrounds and experience behind our purpose to defeat degeneration.
- 网站
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https://www.denalitherapeutics.com
Denali Therapeutics的外部链接
- 所属行业
- 生物技术研究
- 规模
- 201-500 人
- 总部
- South San Francisco，California
- 类型
- 上市公司
- 创立
- 2015
- 领域
- Biotechnology、Neurodegenerative Disease、Neurodegeneration和Pharmaceuticals
地点
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主要
获取路线
161 Oyster Point Blvd
US，California，South San Francisco，94080
Denali Therapeutics员工
动态
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We reported our fourth quarter and full year 2024 financial results and business highlights. Read our press release here: https://lnkd.in/g3i8HYyX
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Denali is heading to BioHive Live! We’re excited to join?BioHive Live 2025?on?Feb. 20?as part of?BioHive Week, bringing together Utah’s vibrant life sciences community. Don’t miss these insightful sessions from our leaders: Fireside Chat?| CEO & Co-founder?Ryan Watts?dives into?what truly sets apart the most promising healthcare solutions?with?David Schenkein, MD?(GV, formerly Google Ventures). Tackling Brain Diseases?| CMO?& Head of Development?Carole Ho?shares personal insights to how science, innovation, and patient focused clinical development can bring life-changing treatments to patients in need. ? Industry Leadership?| CPO & BioHive Board Vice-Chair?Cindy Dunkle?joins fellow BioHive leaders to discuss the impact of Utah’s life sciences ecosystem. With our?clinical?manufacturing facilities in Salt Lake City, Denali is proud to be part of this thriving biotech hub.?View our current job openings here:?https://lnkd.in/gzUqEQ2S See you at #BioHiveLive! #LifeSciences #Biotech #BioHiveWeek
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Denali is?pleased to share?that research fellow?Robert Thorne?will deliver the?keynote address?at this week's?Keystone Symposia?conference,?"Drug Delivery to the Brain: Emerging Modalities." In addition, our scientists Joy Zuchero, Kylie Chew, Michelle E. Pizzo, and Padma Akkapeddi, Ph.D., will present?the latest?research?advances?on our?differentiated?platform for delivering therapeutic enzymes, antibodies, and oligonucleotides beyond biological barriers and into the brain. Learn more about our?BBB-crossing?technology here:?https://lnkd.in/g_6kkJue. We invite scientists and industry professionals to join us in pioneering a new class of therapeutics for rare and common brain diseases. Explore our open positions:?https://lnkd.in/eyysVP6t. #KeystoneSymposia #BrainTherapeutics #DenaliScience #DrugDelivery #Neuroscience?
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This marks our seventh year in attendance at the #WORLDSymposium for lysosomal diseases. In this relatively short time, we have applied our TransportVehicle platform to invent and develop investigational therapeutics for individuals living with lysosomal diseases. We will build on the momentum from engaging with the research and advocacy community this week as we focus on an expected regulatory submission early this year for our Hunter syndrome program and continue to advance our programs for Sanfilippo syndrome (MPS IIIA), Pompe disease, and Gaucher disease.
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Today, we?are pleased to?share?the primary analysis and results from long-term follow-up?of our?Phase 1/2?study for?Hunter syndrome?(MPS II). This program is on track for an early 2025 regulatory submission?for accelerated approval in the U.S.?We are committed to bringing a new treatment option to individuals and families affected by this disease, with the aim of making a meaningful and lasting difference in their lives. Please see our news release here:??https://lnkd.in/gXSeCc4T #WORLDSympoisum #HunterSyndrome
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Thank you?Kristin McKay for bringing the patient community perspective to this?important?conversation today at #WORLDSymposium2025.
Honored to Speak at the 2025 WORLDSymposium! I’m thrilled to share that I will be speaking at the Satellite Symposium presented by Denali Therapeutics at the 2025 WORLDSymposium! The session, Voices in Unison: Insights into the Unmet Needs in MPS II from Patient Community and Physician Perspectives, will bring together critical voices to discuss the ongoing challenges in Hunter syndrome (MPS II). I am deeply honored to join a panel alongside Dr. Joseph Muenzer and Dr. Barbara Burton, two esteemed experts in the field. I will be presenting the patient community perspective, shedding light on the real-world impact of MPS II and the urgent need for continued innovation, advocacy, and support. Hunter syndrome families inspire my work every day, and I’m grateful for the opportunity to ensure their voices are heard in this important discussion. Thank you to Denali Therapeutics for creating a platform where patient experiences and clinical expertise can come together to drive progress. If you’re attending WORLDSymposium, I hope to see you there! Let’s keep pushing forward for better treatments, better care, and a better future for those living with MPS II. #WORLDSymposium2025 #MPSII #HunterSyndrome #RareDisease #PatientAdvocacy #ProjectAlive #UnmetNeeds
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We’re looking forward to the?#WORLDSymposia??[https://worldsymposia.org/] 2025, the annual research conference dedicated to lysosomal diseases, taking place Feb. 3-7 in San Diego. Learn more here:?https://lnkd.in/gTqum-_p ? Updates from Denali?include?an?oral?presentation?on our Phase 1/2 Hunter syndrome (MPS II) clinical?study?(Thurs, Feb 6, Clinical Applications Session) and two poster?presentations highlighting the unmet needs for the treatment and care of somatic manifestations in people with MPS II (Poster 44) and establishing age-based reference intervals (Poster 132). We are also collaborating with?Barbara Burton, Joseph Muenzer, Kristin McKay?on a symposium discussion?focused on the unmet needs in MPS II from patient community and physician perspectives (Wed, Feb 5, at 6:45 a.m. PT). We look forward to a week of engaging with all those?seeking to better understand and to improve?care and support?for?individuals and families living with?Hunter Syndrome and other lysosomal?storage?diseases.
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Exciting year ahead at?Denali! Today, we shared our 2025 priorities, including: - Submitting our first BLA and preparing for the commercial launch of tividenofusp alfa for MPS II (Hunter syndrome). - Enabling?an accelerated approval path for DNL126 for MPS IIIA (Sanfilippo syndrome). - Expanding our portfolio of TransportVehicle? (TV)?enabled programs for brain delivery of enzyme, oligonucleotide, and antibody therapeutics. As leaders in the blood-brain barrier (BBB) field, we've pioneered a new class of barrier-crossing therapeutics. In 2025, we are driven to realize the potential of the TV platform and deliver on its potential to transform treatment for people living with rare and common brain-impacting diseases. Read more about our 2025 outlook here: https://lnkd.in/gGycy-p4
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U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II) ? We are?thrilled to share?that the U.S. FDA has granted Breakthrough Therapy Designation to our therapeutic candidate, tividenofusp alfa (DNL310), for the treatment of Hunter syndrome (MPS II). Read more here: https://lnkd.in/emTs7S6y ? The designation?reflects?the urgent need for therapies?that treat?both brain and body, addressing the full spectrum of Hunter syndrome, a progressive and devastating disease. We look forward to submitting a Biologics License Application (BLA) in early 2025 for review under an accelerated approval pathway. ? We recognize the collective efforts that have supported?this?progress, and we look forward to continued collaboration with the FDA to bring an effective therapy to the Hunter syndrome community as soon as possible.?? ? #HunterSyndrome #RareDisease
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