Jaya Biosciences Presents Promising Preclinical Data in Alzheimer's Disease at the 20th Annual WORLDSymposium™ 2024
- Study results highlight potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s -
SOUTH SAN FRANCISCO, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Jaya Biosciences, Inc. (“JayaBio” or “the Company”), a privately held early-stage life-sciences company developing CNS-directed gene therapies to address unmet needs in genetically defined neurodegeneration, reported non-clinical data at the 20th Annual WORLDSymposium™, a leading research conference on lysosomal diseases. During a late-breaking news platform presentation, Jaya Biosciences’ scientific founder, Prof. Mark Sands, reported preliminary human genetic analysis suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s patients, as well as preclinical efficacy results in the animal model of Alzheimer’s disease (AD) for JB111, the Company’s lead therapy.
“We are thrilled to showcase a new paradigm for the treatment of genetically defined neurodegeneration at the 20th WORLDSymposium™,” said Pawel Krysiak, President and CEO of JayaBio. “The preclinical data we presented demonstrate a tremendous promise of targeting PPT1 haploinsufficiency in Alzheimer’s disease. Development of an effective treatment for Alzheimer’s has been elusive for decades and nearly 7 million Americans are the testament to the size of the unmet medical need. This underscores the necessity for a shift in the strategy for Alzheimer’s therapy development. Our team has identified and validated a number of genes as potential targets for the upstream effector lysosomal pathway, and JayaBio will build its clinical programs upon this groundbreaking foundation of scientific data.”
The following talk was presented at the 20th WORLDSymposium™:
Title: Haploinsufficiency of lysosomal enzymes and Alzheimer’s disease
Date and Time: Friday, Feb. 9, 2024 at 9:00 AM PT (12:00 PM ET)
Session: Late-Breaking Science
Presenter (In-person): Prof. Mark Sands, Washington University School of Medicine, St. Louis, MO
Preliminary human genetic analysis suggests that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s patients.
Heterozygosity of five different lysosomal enzyme genes (PPT1, NAGLU, GALC, IDUA, GUSB) significantly affects amyloid precursor protein (APP) processing and favors pro-amyloidogenic pathway.
Heterozygosity of PPT1 and NAGLU exacerbate amyloid–beta (Aß) plaque formation, increase insoluble Aß40 and Aß42, and significantly decrease the life span of the 5xFAD mouse model of AD.
CNS-directed, AAVmediated gene therapy significantly increases the life span and improves cognitive function of 5xFAD/PPT1+/- mice.
“These data strongly suggest that heterozygous deleterious mutations in a subset of lysosomal genes are enriched in patients with Alzheimer’s disease and there is a nearly perfect gene dosage effect of these mutations on Aß processing in five different mouse models,” said Prof. Mark Sands, Jaya Biosciences’ Scientific Founder and Science Advisory Board Chair. “Furthermore, CNS-directed AAV-mediated gene therapy complemented the haploinsufficiency of our lead target gene, palmitoyl protein thioesterase-1 (PPT1), and dramatically improved the clinical signs in a mouse model of AD. Collectively, these findings challenge the old dogma that carriers of lysosomal enzyme gene defects are normal throughout life and provides a conceptual and practical framework to treat neurodegeneration associated with these heterozygous mutations.”
“We are very encouraged by the positive preclinical results from our JB111 gene therapy study in PPT1-associated Alzheimer’s model,” added Dr. Neal Goodwin, Co-founder and Chief Scientific Officer of JayaBio. “We believe that having demonstrated strong animal proof of concept for JB111 supports raising additional capital and proceeding forward to IND-enabling toxicology and clinical studies.”
For more information on how to access the live streamed events, presentations, and Q&A sessions, please go to WORLDSymposium™. The abstract will also be made available on JayaBio’s corporate website.
About Autophagy-Lysosomal Pathway in Neurodegeneration
1. Martini-Stoica H, et al., (2016) Trends Neurosci 39(4):221-234
About Jaya Biosciences, Inc.
Jaya Biosciences Inc.