JayaBio News
Jaya Biosciences Receives U.S. Patent for Novel Methods of Treating Neurological Diseases, Including Alzheimer’s Disease, Expanding its Global Intellectual Property Portfolio
U.S. patent grant broadens protection and strengthens Jaya Biosciences’ leadership in developing CNS- directed therapies for genetically defined neurodegenerative diseases
November 5, 2025
- U.S. patent grant broadens protection and strengthens Jaya Biosciences’ leadership in developing CNS- directed therapies for genetically defined neurodegenerative diseases -
South San Francisco, CA, November 5 2025 – 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, announced today that it has received from the United States Patent and Trademark Office (USPTO) U.S. Patent No. 12,460,262, entitled “METHODS OF DETECTING, PREVENTING, REVERSING, AND TREATING NEUROLOGICAL DISEASES.” This patent issuance marks a significant expansion of JayaBio’s global intellectual property estate following prior patent issuances in Japan, China, and Singapore, and strengthens protection for its pioneering approach to lysosomal enzyme gene–based interventions in Alzheimer’s disease and other neurodegenerative conditions. The patent covers proprietary methods for detecting, preventing, reversing, and treating neurological diseases linked to heterozygous mutations in lysosomal enzyme genes—genetic variants shown to increase susceptibility to adult-onset neurodegeneration, including Alzheimer’s disease.
“This granted U.S. patent represents an important milestone for JayaBio,” said Pawel Krysiak, President and Chief Executive Officer of Jaya Biosciences. “It underscores our commitment to addressing the genetic and molecular underpinnings of Alzheimer’s disease and further validates the novelty and therapeutic potential of our precision medicine gene therapy platform. The addition of U.S. patent protection reinforces the Company’s strategic position in one of the world’s most important biotechnology markets and enables us to pursue future partnerships and development opportunities.”
“The U.S. market is critical for both the scientific validation and commercialization of innovative Alzheimer’s disease therapies,” added Eric Grinstead, Chief Commercial Officer of Jaya Biosciences. “This patent further secures our foundational intellectual property and strengthens our position as a leader in the emerging field of gene therapy for lysosomal dysfunction–associated neurodegeneration.”
JayaBio’s intellectual property portfolio is licensed from Washington University in St. Louis, Missouri, where investigators discovered that mutations in lysosomal enzyme genes represent key risk factors for adult-onset neurological disease, including Alzheimer’s disease. Human genetic analyses showed that deleterious variants in several lysosomal enzyme genes are significantly enriched in the Alzheimer’s population compared to matched controls.
“These findings expand our understanding of the genetic factors associated with neurodegeneration in adults,” said Prof. Mark Sands, Chair of JayaBio’s Scientific Advisory Board and Professor at Washington University School of Medicine. “Carriers of lysosomal enzyme gene defects were historically considered unaffected throughout life, but our data demonstrate a strong association between carrier status and adult-onset neurological disease. This discovery represents a paradigm shift in how we view genetic risk and intervention strategies for Alzheimer’s and related disorders.”
A Growing Unmet Need
Alzheimer’s disease remains one of the most pressing medical and socioeconomic challenges in the United States. Nearly seven million Americans are currently living with Alzheimer’s, and another seven million are estimated to have mild cognitive impairment (MCI), which in many cases represents prodromal or early Alzheimer’s pathology. The combined public-health and economic burden underscores the urgent need for disease-modifying therapies that address upstream molecular mechanisms rather than symptomatic endpoints.
JayaBio’s lead program, JB111, is an experimental CNS-directed, AAV-mediated gene therapy that targets upstream effector pathways in Alzheimer’s disease associated with PPT1 haploinsufficiency. In preclinical models, a single intracerebroventricular (ICV) injection improved survival, cognition, and amyloid pathology, supporting further IND-enabling development. “Our preclinical data demonstrate that correcting lysosomal enzyme deficits in the CNS through precision gene therapy can potentially redefine Alzheimer’s treatment," said Dr. Neal Goodwin, Chief Scientific Officer of Jaya Biosciences.
About JB111
JB111, Jaya Biosciences’ lead therapy, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for Alzheimer’s disease associated with PPT1 haploinsufficiency (carrier status for mutations in palmitoyl protein thioesterase-1 gene or PPT1 heterozygosity). JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease. A single intracerebroventricular (ICV) injection of JB111 resulted in a significantly increased life span, reduced amyloid pathology, and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held preclinical-stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegenerative diseases, including Alzheimer’s, Parkinson’s, and frontotemporal dementia. Its platform is based on the groundbreaking discovery that carriers of loss- of-function mutations in lysosomal enzyme genes have an increased risk of neurodegeneration including early onset of symptoms. JayaBio’s team, proven in drug development and commercialization of lysosomal targets and gene therapies, is dedicated to addressing unmet needs in neurodegeneration and advancing solutions that transform patient lives worldwide. JayaBio’s mission is to fulfill a promise of victory over neurodegeneration by identifying and targeting the key pathways involved in the etiology of these debilitating diseases.
Investor/Media Contact:
Jaya Biosciences Inc.
Jaya Biosciences Receives Patent Notices of Allowance in Japan, China, and Singapore for Novel Methods of Treating Neurological Diseases, including Alzheimer’s
Patent allowances in Japan, China and Singapore support Jaya Biosciences’ commitment to addressing unmet needs in neurodegeneration in global markets
July 16, 2025
- Patent allowances in Japan, China and Singapore support Jaya Biosciences’ commitment to addressing unmet needs in neurodegeneration in global markets -
South San Francisco, CA, July 16, 2025 – 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 official patent notices of allowance in Singapore, Japan and China on July 6th, July 10th and July 12th, respectively, for the patent application entitled: METHODS OF DETECTING, PREVENTING, REVERSING, AND TREATING NEUROLOGICAL DISEASES. “We are very pleased to have our first patents allowed in China, Japan, and Singapore, which represent major markets and a large unmet need,” said Pawel Krysiak, JayaBio’s President & CEO. “This supports our strong commitment to addressing unmet needs in Alzheimer’s and other neurodegenerative diseases globally and paves the way for potential collaborations with strategic partners in Asian markets.”
JayaBio has licensed its technology from Washington University, St. Louis, MO. Investigators at Washington University School of Medicine have discovered that heterozygous loss-of-function (LoF) mutations in lysosomal enzyme genes represent risk factors for adult-onset neurological disease, including Alzheimer’s Disease (AD). Human genetic analyses show that deleterious coding variants in several lysosomal enzyme genes are significantly enriched in the AD population compared to matched controls. Preliminary analysis of pedigrees from families of known carriers for these mutations suggests that there is a several-fold increase in adult-onset neurologic disease compared to the general population. Animal studies support the human data and show that haploinsufficiency of lysosomal enzymes directly affects amyloid beta (Aβ) metabolism, greatly exacerbates plaque formation, and significantly reduces the life span of the 5xFAD mouse model of Alzheimer’s disease. A single dose of JB111, a CNS-directed, AAV-mediated gene therapy, ameliorates the pathological and clinical phenotype of the 5xFAD mouse harboring a heterozygous LoF mutation in the PPT1 gene. “Historically, carriers of lysosomal enzyme gene defects have been considered normal throughout life, but these data represent an extension of Mendel’s basic laws of autosomal recessive diseases and an important paradigm shift for carriers of lysosomal enzyme defects and adult-onset neurological disease, including Alzheimer’s,” said Prof. Mark Sands, JayaBio’s Science Advisory Board Chair.
Japan has one of the highest rates of Alzheimer’s disease worldwide, with 23% of women and 11% of men aged 60 or older, or over 6 million people, living with AD. China has more than 16 million people suffering from dementia, predominantly Alzheimer’s, which represents the world’s largest population and accounts for nearly 30% of the total global dementia cases. In Singapore, dementia affects about 1 in 11 adults over 60 years of age, and the number of people with dementia, of which Alzheimer’s disease constitutes the majority, is expected to more than double by 2030. “While the exact numbers are not yet known, we believe that JayaBio’s precision medicine approach targeting upstream effector pathways rather than downstream markers can address a significant portion of Alzheimer’s disease and related dementias in these markets as well as worldwide,” said Dr. Neal Goodwin, Chief Scientific Officer of JayaBio. “We are excited about guiding JB111 through IND-enabling toxicology and clinical development to provide a viable solution to Alzheimer’s patients with heterozygous mutations in lysosomal enzyme genes.”
About Autophagy-Lysosomal Pathway in Neurodegeneration
Lysosomes are subcellular organelles responsible for the normal degradation and turnover of cellular components and aggregation-prone proteins. Deficits in the autophagy-lysosomal pathway (ALP) result in protein aggregation, the generation of toxic protein species, and accumulation of dysfunctional organelles, which are hallmarks of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease¹. Lysosomal degradation plays a critical role, as the degradation of autophagosomal cargo cannot proceed without successful fusion to an available and functional lysosome². Lysosomal function declines with age and likely contributes both to the aging process itself as well as the development of age-related diseases such as neurodegenerative diseases and cancer²,³. Emerging evidence suggest that defects in certain lysosomal enzyme genes are associated with various neurodegenerative diseases such as Parkinson’s Disease4, Frontotemporal Dementia⁵, and Alzheimer’s Disease⁶.
1. Martini-Stoica H, et al., (2016) Trends Neurosci 39(4):221-234
2. Levine B, Kroemer G, (2019) Cell 176(1-2):11-42
3. Hansen M, et. al., (2018) Nature Rev Mol Cell Biol, 19(9):579-593
4. Sidransky E, et al., (2009) N Engl J Med 361(17):1651-61.
5. Baker M, et al., (2006) Nature, 442(7105):916-9
6. Lopergolo D, et al., (2024) J Med Genet, 61:332-9
About JB111
JB111, Jaya Biosciences’ lead therapy, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for Alzheimer’s disease associated with PPT1 haploinsufficiency (carrier status for loss-of-function mutations in palmitoyl protein thioesterase-1 gene or PPT1 heterozygosity). JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease (5XFAD mouse harboring PPT1 heterozygous allele). A single intracerebroventricular (ICV) injection of JB111 resulted in a significantly increased life span, reduced plaque load, and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held pre-clinical stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegenerative diseases, including Alzheimer's, Parkinson’s, and frontotemporal dementia. JayaBio’s platform is based on the groundbreaking discovery that carriers of loss-of-function mutations in genes for lysosomal enzymes have an increased risk of neurodegeneration, including early onset of symptoms. JayaBio’s team, proven in drug development and commercialization of lysosomal targets and gene therapies, is dedicated to addressing unmet needs in neurodegeneration and adding value to patient and medical communities worldwide. JayaBio’s mission is to fulfill a promise of victory over neurodegeneration by identifying and targeting the relevant pathways involved in the etiology of these debilitating diseases.
Investor/Media Contact:
Jaya Biosciences Inc.
Jaya Biosciences Presents Updated Preclinical Data in Alzheimer’s Disease at the 45th SIMD Annual Meeting
Study results confirm potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s disease
April 16, 2024
- Study results confirm potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s disease -
South San Francisco, CA, April 16, 2024 – 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 (Aβ) 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 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 favors pro-amyloidogenic pathway.
PPT1 haploinsufficiency greatly exacerbates Aβ 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 Aβ 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
Lysosomes are subcellular organelles responsible for the normal degradation and turnover of cellular components and aggregation-prone proteins. Deficits in the autophagy-lysosomal pathway (ALP) result in protein aggregation, the generation of toxic protein species, and accumulation of dysfunctional organelles, which are hallmarks of neurodegenerative diseases, including Alzheimer's disease (AD) and Parkinson's disease (PD).1 Lysosomal degradation plays a critical role, as the degradation of autophagosomal cargo cannot proceed without successful fusion to an available and functional lysosome.2 Lysosomal function declines with age and likely contributes both to the aging process itself as well as the development of age-related diseases such as neurodegenerative diseases and cancer.2,3 Emerging evidence suggest that defects in certain lysosomal enzyme genes are associated with various neurodegenerative diseases such as Parkinson’s Disease4, Frontotemporal Dementia5, and Alzheimer’s Disease.6
1. Martini-Stoica H, et al., (2016) Trends Neurosci 39(4):221-234
2. Levine B, Kroemer G, (2019) Cell 176(1-2):11-42
3. Hansen M, et. al., (2018) Nature Rev Mol Cell Biol, 19(9):579-593
4. Sidransky E, et al., (2009) N Engl J Med 361(17):1651-61.
5. Baker M, et al., (2006) Nature, 442(7105):916-9
6. Lopergolo D, et al., (2023) J Med Genet, Epub ahead of print
About JB111
JB111, Jaya Biosciences’ lead therapy, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for neurodegeneration associated with PPT1 haploinsufficiency (carrier status for loss-of-function mutations in palmitoyl protein thioesterase-1 gene or PPT1 heterozygosity). JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease (5XFAD mouse harboring PPT1 heterozygous allele). Single intracerebroventricular (ICV) injection of JB111 resulted in a significantly increased life span and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held pre-clinical stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegenerative diseases, including Alzheimer's, Parkinson’s, and frontotemporal dementia. Jaya’s platform is based on the groundbreaking discovery that carriers of loss-of-function mutations in genes for lysosomal enzymes have an increased risk of neurodegeneration, including early onset of symptoms. Jaya Biosciences’ team, proven in drug development and commercialization of lysosomal targets and gene therapies, is dedicated to addressing unmet needs in neurodegeneration and adding value to patient and medical communities worldwide. Jaya’s mission is to fulfill a promise of victory over neurodegeneration by identifying and targeting the relevant pathways involved in the etiology of these debilitating diseases. For more information, please visit www.jayabio.com
Investor/Media Contact:
Jaya Biosciences Inc.
Jaya Biosciences Presents New Paradigm for the Treatment of Alzheimer’s Disease at the 23rd Annual Needham Virtual Healthcare Conference
Data presented highlight the potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s
April 9, 2024
- Data presented highlight the potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s -
South San Francisco, CA, April 9, 2024 – Jaya Biosciences, Inc. (“JayaBio” or “the Company”), a privately held early-stage life-sciences company developing gene therapies to address unmet needs in genetically defined neurodegeneration, presented promising non-clinical data for a novel treatment approach in Alzheimer’s disease at the 23rd Annual Needham Virtual Healthcare Conference. JayaBio’s President and CEO, Pawel Krysiak, reviewed the rationale for targeting the autophagy-lysosomal pathway and discussed the Company’s lead program, JB111, including its strong animal proof-of-concept data and near-term development milestones.
“We are grateful for the opportunity to share a new paradigm for the treatment of genetically defined neurodegeneration with the investor community at the 23rd Annual Needham Virtual Healthcare Conference,” said Pawel Krysiak, President and CEO of JayaBio. “The preclinical update we presented demonstrates a tremendous promise of targeting PPT1 haploinsufficiency in Alzheimer’s disease, and with the right funding in place, we could transition this program into the clinic within 3 years. Given the size of the unmet need and the strong potential of our platform, we hope to attract experienced investors to support JayaBio’s progress and help us develop life-changing therapies for genetically defined neurodegeneration, including Alzheimer’s disease.”
The following talk was presented at the 23rd Annual Needham Virtual Healthcare Conference:
Title: Jaya Biosciences: New Paradigm for Genetically-Defined Neurodegenerative Diseases
Date and Time: Mon, Apr 8 at 11:45-12:25 PM
Session: Track 3
Presenter (virtual): Pawel Krysiak, President & CEO, Jaya Biosciences Inc.
Needham Moderator: James Diamond
About The 23rd Annual Needham Virtual Healthcare Conference
This fully virtual conference features fireside chats and presentations from leading public and private companies in the Biopharma, Medtech, Diagnostics and Digital Health sectors as well as thematic panel discussions.
About JB111
JB111, Jaya Biosciences’ lead therapy, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for neurodegeneration associated with PPT1 haploinsufficiency (carrier status for loss-of-function mutations in palmitoyl protein thioesterase-1 gene or PPT1 heterozygosity). JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease (5XFAD mouse harboring PPT1 heterozygous allele). Single intracerebroventricular (ICV) injection of JB111 resulted in a significantly increased life span and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held pre-clinical stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegenerative diseases, including Alzheimer's, Parkinson’s, and frontotemporal dementia. Jaya’s platform is based on the groundbreaking discovery that carriers of loss-of-function mutations in genes for lysosomal enzymes have an increased risk of neurodegeneration, including early onset of symptoms. Jaya Biosciences’ team, proven in drug development and commercialization of lysosomal targets and gene therapies, is dedicated to addressing unmet needs in neurodegeneration and adding value to patient and medical communities worldwide. Jaya’s mission is to fulfill a promise of victory over neurodegeneration by identifying and targeting the relevant pathways involved in the etiology of these debilitating diseases. For more information, please visit www.jayabio.com
Investor/Media Contact:
Jaya Biosciences Inc.
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
February 12, 2024
- Study results highlight potential utility of targeting lysosomal genes to treat neurodegenerative diseases, including Alzheimer’s -
South San Francisco, CA, February 12, 2024 – 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 (Aβ) plaque formation, increase insoluble Aβ40 and Aβ42, and significantly decrease the life span of the 5xFAD mouse model of AD.
CNS-directed, AAV-mediated 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 Ab 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
Lysosomes are subcellular organelles responsible for the normal degradation and turnover of cellular components and aggregation-prone proteins. Deficits in the autophagy-lysosomal pathway (ALP) result in protein aggregation, the generation of toxic protein species, and accumulation of dysfunctional organelles, which are hallmarks of neurodegenerative diseases, including Alzheimer's disease (AD) and Parkinson's disease (PD).1 Lysosomal degradation plays a critical role, as the degradation of autophagosomal cargo cannot proceed without successful fusion to an available and functional lysosome.2 Lysosomal function declines with age and likely contributes both to the aging process itself as well as the development of age-related diseases such as neurodegenerative diseases and cancer.2,3 Emerging evidence suggest that defects in certain lysosomal enzyme genes are associated with various neurodegenerative diseases such as Parkinson’s Disease4, Frontotemporal Dementia5, and Alzheimer’s Disease.6
1. Martini-Stoica H, et al., (2016) Trends Neurosci 39(4):221-234
2. Levine B, Kroemer G, (2019) Cell 176(1-2):11-42
3. Hansen M, et. al., (2018) Nature Rev Mol Cell Biol, 19(9):579-593
4. Sidransky E, et al., (2009) N Engl J Med 361(17):1651-61.
5. Baker M, et al., (2006) Nature, 442(7105):916-9
6. Lopergolo D, et al., (2023) J Med Genet, Epub ahead of print
About JB111
JB111, Jaya Biosciences’ lead therapy, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for neurodegeneration associated with PPT1 haploinsufficiency (carrier status for loss-of-function mutations in palmitoyl protein thioesterase-1 gene or PPT1 heterozygosity). JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease (5XFAD mouse harboring PPT1 heterozygous allele). Single intracerebroventricular (ICV) injection of JB111 resulted in a significantly increased life span and improved cognitive performance.
About Jaya Biosciences, Inc.
Jaya Biosciences is a privately held pre-clinical stage life sciences company developing CNS-directed gene therapies for genetically defined neurodegenerative diseases, including Alzheimer's, Parkinson’s, and frontotemporal dementia. Jaya’s platform is based on the groundbreaking discovery that carriers of loss-of-function mutations in genes for lysosomal enzymes have an increased risk of neurodegeneration, including early onset of symptoms. Jaya Biosciences’ team, proven in drug development and commercialization of lysosomal targets and gene therapies, is dedicated to addressing unmet needs in neurodegeneration and adding value to patient and medical communities worldwide. Jaya’s mission is to fulfill a promise of victory over neurodegeneration by identifying and targeting the relevant pathways involved in the etiology of these debilitating diseases. For more information, please visit www.jayabio.com
Investor/Media Contact:
Jaya Biosciences Inc.