New Paradigm for Genetically Defined Neurodegenerative Diseases

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About Jaya Biosciences
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. Our science challenges 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. Jaya’s mission is to fulfill a promise of victory over neurodegenerative diseases by identifying and effectively targeting the relevant pathways involved in the etiology of these debilitating diseases.

We are a group of experienced biotech executives, scientists, and clinicians unified by a common goal of addressing unmet needs in neurodegeneration and adding value to patient and medical communities worldwide. Collectively we have over 100 years of drug development and commercialization experience, including lysosomal targets, recombinant proteins and gene therapies, and we are backed by an expert science advisory board. Our team operates on the basis of integrity, innovation and excellence in action with a patient goal in mind. We value internal and external collaboration, knowing that teamwork results in better solutions.

Jaya Biosciences is a platform company with multiple shots on goal. To date, we validated five different lysosomal enzyme genes (PPT1, NAGLU, GALC, IDUA, GUSB) that significantly affect amyloid precursor protein (APP) processing and favor pro-amyloidogenic pathway. JB111, our 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.

Impaired autophagy-lysosomal pathway (ALP) is a key component in the etiology of genetically defined CNS dysfunction. 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 the accumulation of dysfunctional organelles, which are hallmarks of neurodegenerative diseases, including Alzheimers disease (AD) and Parkinsons 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 suggests that defects in certain lysosomal enzyme genes are associated with various neurodegenerative diseases such as Parkinson’s Disease (4), Frontotemporal Dementia (5), 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

Let's work together to end neurodegeneration and related suffering!

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