Denali Therapeutics

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

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 

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.

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

Thank you Kristin McKay for bringing the patient community perspective to this important conversation today at #WORLDSymposium2025.

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.

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

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

Update from Partner-Sponsored HEALEY ALS Platform Trial with DNL343   We are grateful to the participants, families, and investigators involved in the Phase 2/3 HEALEY ALS Platform Trial, including a regimen evaluating DNL343. While the study endpoints were not met, we look forward to additional analyses to fully understand the impact of DNL343 in ALS. Overall, DNL343 was found to be safe and well tolerated. Further analyses are anticipated later in 2025, including neurofilament light (NfL) and other fluid biomarkers, data from pre-specified subgroups, as well as extended findings from the active treatment extension period. We appreciate the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) in collaboration with the Northeast ALS Consortium (NEALS), the organizations that conducted and supported this study, and their efforts to advance therapies for ALS. https://lnkd.in/gvBq6Dgs

We had the privilege of joining the National MPS Society 38th Annual Family & Scientific Conference in Orlando, Florida! This special event brings families, clinicians, and researchers together each year to share the latest research advancements and foster meaningful connections. This year was extra special as we celebrated the MPS Society’s 50th anniversary—a significant milestone for the MPS community. We’re inspired by the incredible progress achieved over the years and are excited for the milestones still to come. A big congratulations and thank you to the National MPS Society—the future is looking bright!