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Jaya Biosciences Presents Updated Preclinical Data in Alzheimer's Disease at the 45th SIMD Annual MeetingSOUTH SAN FRANCISCO, Calif., April 16, 2024 (GLOBE NEWSWIRE) -- Jaya Biosciences, Inc. (“JayaBio” or “the Company”), a privately held early-stage life-sciences company developing CNS-directed gene therapies for unmet needs in the neurodegenerative diseases, reported preclinical data at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), a leading research conference on inherited metabolic diseases, including lysosomal diseases. During a podium presentation, Jaya Biosciences’ scientific founder and science advisory board chair, Professor Mark Sands, reported on recently updated human genetic analyses suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s disease (AD) patients. This new analysis generated from a much larger whole genome sequence database confirmed their previous human genetic findings from a smaller whole exome database. Professor Sands presented in vivo data that validated several of the genes identified in the human AD cases and identified two additional lysosomal enzyme genes that affect amyloid-beta (Aß) processing in heterozygous animals. Finally, CNS-directed, AAV-mediated gene therapy with JB111 (the Company’s lead therapy) was shown to ameliorate the biochemical, histological, and clinical signs of disease in an animal model of PPT1-associated AD (5xFAD harboring a heterozygous mutation in the PPT1 gene). “We are pleased to see that the additional genetic analyses corroborated our initial findings of lysosomal dysfunction in Alzheimer’s disease,” said Pawel Krysiak, President and CEO of JayaBio. “The updated preclinical data we presented continue to show the high potential of targeting PPT1 haploinsufficiency in Alzheimer’s and support the necessity for a paradigm shift in the strategy for AD therapy development. Our team is committed to further advancing JayaBio’s platform targeting the upstream effector lysosomal pathway in an effort to address an enormous unmet need in neurodegenerative diseases.” The following talk was presented at the 45th SIMD Annual Meeting: Title: Haploinsufficiency of lysosomal enzymes and Alzheimer’s disease Date and Time: Mon, April 15th, 2024 at 5:00 PM ET Session: Science Session 4 Presenter (In-person): Prof. Mark Sands, Washington University School of Medicine, St. Louis, MO 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 favrs pro-amyloidogenic pathway. PPT1 haploinsufficiency greatly exacerbates Aß plaque formation and significantly reduces the life span of the 5xFAD mouse model of AD. CNS-directed, AAV-mediated PPT1 gene therapy significantly increases the life span of 5xFAD/PPT1+/- mice. “The updated human genetic data confirmed that heterozygous deleterious mutations in a subset of lysosomal genes are enriched in patients with Alzheimer’s disease,” said Prof. Mark Sands. “Together with previously reported gene dosage effects of these mutations on Aß processing, it underscores the utility of targeting lysosomal genes haploinsuficiencies in neurodegenerative diseases and adds to the evolving understanding regarding the status of lysosomal enzyme gene defects carriers. Especially considering that our lead therapy, the CNS-directed AAV-mediated PPT1 gene therapy, dramatically improved the clinical signs in a mouse model of AD.” “We continue to be encouraged by the positive preclinical results from our JB111 gene therapy study in PPT1-associated Alzheimer’s model and look forward to translating our strong animal proof of concept to IND-enabling toxicology and clinical studies,” added Dr. Neal Goodwin, Co-founder and Chief Scientific Officer of JayaBio. For more information about the 45th SIMD Annual Meeting, please go to SIMD2024 Meeting. 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 JB111 About Jaya Biosciences, Inc. Investor/Media Contact: Jaya Biosciences Inc. |