Denali Therapeutics

What an incredible day coming together with ALS Network to rally support and raise awareness for those living with ALS! A huge thank you to Cytokinetics and all our colleagues who joined us for the third consecutive year at the South San Francisco SPLASH to Cure ALS on August 14th. Coming together, taking the plunge, and standing with the local ALS community was truly inspiring. This month marks the 10th anniversary of the #ALSIceBucketChallenge, making our efforts this year even more impactful—together, we’re making a difference!

We are grateful to Jennifer Thompson and two remarkable young adults for joining us in an important discussion on the impact of ALS on families and the resilience of youth caregivers. Thank you for sharing your experiences with us and for everything Hope Loves Company, Inc. does to support families and children affected by ALS.  #ALS #Caregivers #CommunitySupport

We are pleased to share that Scientific Translational Medicine has published our research in an article titled, "Targeting the transferrin receptor to transport antisense oligonucleotides (ASOs) across the mammalian blood-brain barrier". This is the first publication of data showing that ASOs can be delivered uniformly throughout the primate CNS and to muscle via a less invasive route of intravenous administration enabled by Denali's Transport Vehicle (TV) platform. Read the article here: https://lnkd.in/gdW9fjMJ

We reported our second quarter financial results and business highlights. Read our press release here: https://lnkd.in/gsaNH-DP

We were thrilled to join the The ALS Association's first-ever NEXUS Summit this week! It was a powerful few days of connecting, listening, and learning from hundreds of dedicated participants who attended to contribute to the ongoing fight against ALS. We were also delighted to celebrate with the community, commemorating the 10th anniversary of the Ice Bucket Challenge, a global initiative that has significantly raised awareness and funds for ALS. #ALSNEXUS #IceBucketChallenge #CommunityImpact #FightALS

We are pleased to share the Reagan-Udall Foundation for the FDA's summary of the public workshop they hosted titled, "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways." This impactful meeting, held on February 21, 2024, convened experts in MPS to discuss the scientific evidence supporting CSF heparan sulfate levels as a biomarker to support accelerated approval of new treatments for neuronopathic MPS. Thank you to the Reagan-Udall Foundation for hosting this workshop and capturing the outcomes of this meeting. Read the full summary: https://bit.ly/4fjfMB9

We are pleased to share the recent article titled, "Community Consensus for Heparan Sulfate as a Biomarker to Support Accelerated Approval in Neuronopathic Mucopolysaccharidoses" in Molecular Genetics and Metabolism. This article highlights the 2020 FDA guidance supporting accelerated approval based on substrate reduction for diseases like neuronopathic MPS, characterized by heparan sulfate (HS) in the brain. The article reflects the incredibly collaborative efforts across academia, advocacy, and industry, which culminated in the Reagan-Udall Foundation for the FDA-hosted workshop in February 2024. Appreciation to the authors and countless others for their unwavering commitment to advocating for science-driven approaches to accelerate the approval path for new treatment options for individuals and families living with MPS diseases. Read the full article here: https://lnkd.in/dNA49ThV Congratulations to the authors of the article: Joseph Muenzer, UNC at Chapel Hill; Carole Ho, Denali; Heather Lau MD MS, Ultragenyx; Mark Dant, The Ryan Foundation; Maria Fuller, University of Adelaide; Nidal Boulos, PhD, CCRP, Regenxbio; Patti Dickson, Washington University School of Medicine; Matthew Ellinwood, National MPS Society; simon jones, Manchester University NHS Foundation Trust; Eric Zanelli, Allievex Corp.; Cara O'Neill, Cure Sanfilippo Foundation. #MPS #defeatdegeneration

On June 2nd, Denali team members joined the San Francisco Giants and Major League Baseball (MLB) to celebrate the fourth annual Lou Gehrig Day, honoring the legendary player and raising awareness for Amyotrophic Lateral Sclerosis (ALS).  It's inspiring to see the MLB community unite to honor Lou Gehrig and support those affected by ALS. The camaraderie among fans, players, and organizations demonstrates the power of collective efforts in raising awareness and advancing ALS research.  #LouGehrigDay #EndALS #DenaliTherapeutics #SanFranciscoGiants #MLB

We are pleased to share the FDA has selected DNL126 (ETV:SGSH), an investigational treatment for MPS IIIA (Sanfilippo syndrome Type A), for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. The FDA's CDER and CBER divisions jointly launched START in September 2023 to further accelerate development of rare disease therapeutics. Participating in START is a significant opportunity to collaborate further with the FDA to solve challenges unique to rare disease drug development and determine the most efficient development path for DNL126 in MPS IIIA, a devastating and progressive disease for which there are no approved treatments. Read our press release here: https://lnkd.in/gFyRdfsN

Our team was deeply inspired by "Love & Life: No Ordinary Campaign", a documentary highlighting the transformative power of love, hope, and activism within the ALS community. It was an extraordinary experience to watch this film alongside many members of the community during ALS Awareness month. Thank you Sandra Abrevaya, Brian Wallach, and Synapticure Inc. for inviting us to join this screening. The documentary will be available on Prime Video May 28th. #ALSawareness #ForLoveAndLife #NoOrdinaryCampaign