SOUTH SAN FRANCISCO, Calif., April 16, 2024 (Globe Newswire) — Jaya Biosciences, Inc. (“JayaBio” or the “Company”) is a privately held company developing gene therapies targeting the central nervous system. is an early stage life sciences company.Unmet needs in neurodegenerative diseases, preclinical data reported in 45 clinical trialsth Annual Meeting of the Society of Inherited Metabolic Disorders (SIMD), the leading research conference on inherited metabolic diseases, including lysosomal diseases.
Professor Mark Sands, Scientific Founder and Chair of the Scientific Advisory Board of Jaya Biosciences, said in a podium presentation that heterozygous loss-of-function mutations in lysosomal enzyme genes are abundant in Alzheimer's disease (AD) patients. We reported on a recently updated human genetic analysis that suggests that This new analysis, generated from a much larger whole-genome sequence database, confirmed previous human genetic findings from smaller whole-exome databases.Presented by Professor Sands in vivo The data validated some of the genes identified in human Alzheimer's disease cases and identified two additional lysosomal enzyme genes that affect amyloid beta (Aβ) processing in heterozygous animals. Finally, his CNS-directed AAV-mediated gene therapy with JB111 (the company's lead therapy) has been shown to improve biochemical, histological, and clinical signs of the disease in animal models. Ta. PPT1– Associated AD (5xFAD carries a heterozygous mutation) PPT1 gene).
“We are pleased that additional genetic analysis confirms our initial findings regarding lysosomal dysfunction in Alzheimer's disease,” said Pawel Krysiak, President and CEO of JayaBio. “The latest preclinical data we have presented continues to demonstrate high targeting potential. PPT1 This study of haploinsufficiency in Alzheimer's disease supports the need for a paradigm shift in Alzheimer's disease treatment development strategies. Our team is committed to further advancing JayaBio's platform targeting upstream effector lysosomal pathways to address the vast unmet needs in neurodegenerative diseases. ”
The following lectures were given at the 45th conference.th SIMD Annual General Meeting:
title: Haploinsufficiency of lysosomal enzymes and Alzheimer's disease
Date and time: Monday, April 15thth2024 at 5:00 PM ET.
session: science session 4
Presenter (face-to-face): Professor Mark Sands, Washington University School of Medicine, St. Louis, Missouri
Human genetic analysis suggests that Alzheimer's disease patients are enriched for heterozygous loss-of-function mutations in lysosomal enzyme genes.
Heterozygosity for five different lysosomal enzyme genes (PPT1, Nagle, GALC, Idua, GUSB) has a profound impact on amyloid precursor protein (APP) processing and promotes pro-amyloidogenic pathways.
PPT1 Haploinsufficiency significantly worsens Aβ plaque formation and significantly shortens lifespan in the 5xFAD mouse model of AD.
CNS-directed, AAV-mediated PPT1 Gene therapy significantly extends the lifespan of 5xFAD/PPT1+/- mice.
“The latest human genetic data confirm that patients with Alzheimer's disease are enriched for deleterious heterozygous mutations in a subset of lysosomal genes,” said Professor Mark Sands. “Coupled with the previously reported gene dosage effects of these mutations on Aβ processing, this highlights the utility of targeting haploinsufficiency of lysosomal genes in neurodegenerative diseases and provides insight into the status of lysosomal enzyme gene deficient carriers. ” Especially considering our main therapy, CNS-directed AAV-mediated therapy. PPT1 Gene therapy dramatically improved clinical symptoms in a mouse model of Alzheimer's disease. ”
“We continue to be encouraged by the positive preclinical results from our JB111 gene therapy study in the United States. PPT1added Dr. Neil Goodwin, co-founder and chief scientific officer of JayaBio.
For more information on 45,th Click here for information about the SIMD Annual General Meeting. SIMD2024 conference. The abstract will also be published on his JayaBio corporate website.
About the autophagy-lysosomal pathway in neurodegeneration
Lysosomes are intracellular organelles responsible for the normal degradation and turnover of cellular components and aggregation-prone proteins. Deficiencies in the autophagy-lysosomal pathway (ALP) lead to protein aggregation, generation of toxic protein species, and accumulation of dysfunctional organelles. These are hallmarks of neurodegenerative diseases, including Alzheimer's disease (AD) and Parkinson's disease (PD).1 Lysosomal degradation plays a critical role, as degradation of autophagosomal cargo cannot proceed unless it successfully fuses into an available functional lysosome.2 Lysosomal function declines with age and may contribute not only to the aging process itself but also to the development of age-related diseases such as neurodegenerative diseases and cancer.2,3 New evidence suggests that defects in certain lysosomal enzyme genes are associated with various neurodegenerative diseases, including Parkinson's diseaseFourfrontotemporal dementiaFive,Alzheimer's disease.6
1. Martini-Stoica H, et al. (2016) neuroscience trends 39(4):221-234
2. Levin B, Cromer G, (2019) cell 176(1-2):11-42
3. Hansen M, et al. Al, (2018) Nature Rev Mol Cell Biol19(9):579-593
4. Sidransky E et al. (2009) N English J Medicine 361(17):1651-61.
5. Baker M et al. (2006) Nature442(7105):916-9
6. Lopergolo D et al. (2023) J. Med GenetEpub ahead of print
About JB111
Jaya Biosciences' lead therapy, JB111, is an experimental CNS-directed AAV9-mediated therapy. PPT1 Gene therapy is currently being developed for neurodegeneration such as: PPT1 haploinsufficiency (carrier status of a loss-of-function mutation in the palmitoyl protein thioesterase-1 gene; or PPT1 heterozygosity). JB111 has shown very promising results in animal models. PPT1– Associated Alzheimer's disease (bearing 5XFAD mice) PPT1 heterozygous allele). Her single intracerebroventricular (ICV) injection of JB111 significantly extended lifespan and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held preclinical life sciences company developing central nervous system-directed gene therapies for genetically defined neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and frontotemporal dementia. . Jaya's platform is based on the breakthrough discovery that loss-of-function mutations in genes for lysosomal enzymes increase the risk of neurodegeneration, including early onset of symptoms. Jaya Biosciences' team has a proven track record in drug development and commercialization of lysosomal targeted and gene therapies, and is dedicated to addressing unmet needs in neurodegeneration and adding value to patients and healthcare communities worldwide. I am. Jaya's mission is to deliver on the promise of victory against neurodegeneration by identifying and targeting relevant pathways involved in the pathogenesis of these debilitating diseases. For more information, please visit www.jayabio.com.
Investor/Media Contact:
Jaya Bioscience Co., Ltd.
info@jayabio.com