Jaya Biosciences Secures U.S. Patent for Gene Therapy Approach to Alzheimer's Disease Treatment
核心洞察
Jaya Biosciences (搜索) received U.S. Patent No. 12,460,262 for methods of detecting, preventing, reversing, and treating neurological diseases linked to lysosomal enzyme gene mutations.
The patent covers a novel approach targeting heterozygous mutations in lysosomal enzyme genes (搜索) that increase susceptibility to adult-onset neurodegeneration (搜索), including Alzheimer's disease (搜索).
The company's lead therapy JB111, an AAV9-mediated PPT1 (搜索) gene therapy, demonstrated improved survival, cognition, and reduced amyloid pathology in preclinical models with a single intracerebroventricular injection.
Jaya Biosciences (搜索) announced it has received U.S. Patent No. 12,460,262 from the United States Patent and Trademark Office for "Methods of Detecting, Preventing, Reversing, and Treating Neurological Diseases." The patent covers proprietary methods for addressing neurological diseases linked to heterozygous mutations in lysosomal enzyme genes (搜索)—genetic variants shown to increase susceptibility to adult-onset neurodegeneration (搜索), including Alzheimer's disease (搜索).
This patent issuance marks a significant expansion of JayaBio's global intellectual property estate following prior patent issuances in Japan, China, and Singapore, strengthening protection for its pioneering approach to lysosomal enzyme gene-based interventions in Alzheimer's disease (搜索) and other neurodegenerative conditions.
Novel Genetic Target for Alzheimer's Disease
JayaBio's intellectual property portfolio is licensed from Washington University (搜索) in St. Louis, Missouri, where investigators discovered that mutations in lysosomal enzyme genes (搜索) represent key risk factors for adult-onset neurological disease, including Alzheimer's disease (搜索). 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 (搜索) in adults," said Prof. Mark Sands, Chair of JayaBio's Scientific Advisory Board and Professor at Washington University (搜索) 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."
Lead Therapy Shows Promise in Preclinical Studies
JayaBio's lead program, JB111, is an experimental CNS-directed, AAV-mediated gene therapy that targets upstream effector pathways in Alzheimer's disease (搜索) associated with PPT1 (搜索) haploinsufficiency. In preclinical models, a single intracerebroventricular (ICV) injection improved survival, cognition, and amyloid pathology, supporting further IND-enabling development.
JB111 is an experimental CNS-directed AAV9-mediated PPT1 (搜索) gene therapy currently being developed for Alzheimer's disease (搜索) associated with PPT1 haploinsufficiency (carrier status for mutations in palmitoyl protein thioesterase-1 (搜索) gene or PPT1 heterozygosity). In animal models of PPT1-associated Alzheimer's disease, a single intracerebroventricular injection of JB111 resulted in significantly increased life span, reduced amyloid pathology, and improved cognitive performance.
"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 (搜索).
Addressing Critical Unmet Medical Need
Alzheimer's disease (搜索) 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 (搜索) (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.
Strategic Market Position
"This granted U.S. patent represents an important milestone for JayaBio," said Pawel Krysiak, President and Chief Executive Officer of Jaya Biosciences (搜索). "It underscores our commitment to addressing the genetic and molecular underpinnings of Alzheimer's disease (搜索) 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."
Eric Grinstead, Chief Commercial Officer of Jaya Biosciences (搜索), added: "The U.S. market is critical for both the scientific validation and commercialization of innovative Alzheimer's disease (搜索) therapies. 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 (搜索)."
Jaya Biosciences (搜索) 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 (搜索). The company's platform is based on the discovery that carriers of loss-of-function mutations in lysosomal enzyme genes (搜索) have an increased risk of neurodegeneration (搜索) including early onset of symptoms.