PPT1

Jaya Biosciences Presents Updated Preclinical Data in Alzheimer’s Disease at the 45th SIMD Annual Meeting

Retrieved on: 
Martedì, Aprile 16, 2024
GUSB, Society, PPT1, IDUA, Haploinsufficiency, Gene dosage, Central Waqf Council, Animal, GALC, Research, AAV, IND, CNS, Disease, Science, NAGLU, Patient, DSM-IV codes, SIMD, Toxicology, Annual general meeting, Plaque, SAN, Gene, APP, Antibiotics

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.

Key Points: 
  • 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.
  • “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.
  • For more information about the 45th SIMD Annual Meeting, please go to SIMD2024 Meeting .

Jaya Biosciences Presents New Paradigm for the Treatment of Alzheimer’s Disease at the 23ʳᵈ Annual Needham Virtual Healthcare Conference

Retrieved on: 
Martedì, Aprile 9, 2024
SAN, PPT1, Conference, Haploinsufficiency, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., April 09, 2024 (GLOBE NEWSWIRE) -- 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.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., April 09, 2024 (GLOBE NEWSWIRE) -- 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.
  • 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:

Jaya Biosciences Presents Promising Preclinical Data in Alzheimer’s Disease at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Lunedì, Febbraio 12, 2024
SAN, Toxicology, Research, APP, Plaque, PPT1, Life, Haploinsufficiency, Science Advisory Board, CNS, GUSB, Patient, GALC, Gene, IDUA, Q&A, NAGLU, IND, AAV

SOUTH SAN FRANCISCO, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- 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.

Key Points: 
  • “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.
  • Heterozygosity of five different lysosomal enzyme genes (PPT1, NAGLU, GALC, IDUA, GUSB) significantly affects amyloid precursor protein (APP) processing and favors pro-amyloidogenic pathway.
  • CNS-directed, AAV-mediated gene therapy significantly increases the life span and improves cognitive function of 5xFAD/PPT1+/- mice.

Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease

Retrieved on: 
Giovedì, Dicembre 16, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, FFPM, European Commission, Survival, Disease, Mouse, Forward-looking statement, U.S. Securities and Exchange Commission, Glycogen storage disease, FDA, CLN, CTA, IND, AAV, Observational study, Serum, Quality of life, Drug development, Behavior, Development, Nasdaq, Severe cognitive impairment, COVID-19, SEC, CEO, DEM, Seizure, Prevalence, Associate, Dementia, Medical genetics, Natural history, Royal commission, MRCPCH, Food, MRCP, FRCPC, Death, Central nervous system, Life, CNS, Private Securities Litigation Reform Act, University, Annual report, Phenotype, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, Department, CSF, Division, Gene, Pharmaceutical industry, Vaccine, Pediatrics, Research, PPT1, MBBS, CLN1, CLN1 Disease, Taysha Gene Therapies, CLN1 DISEASE, TAYSHA GENE THERAPIES

There is also an open investigational new drug application (IND) in the United States for TSHA-118 in CLN1 disease.

Key Points: 
  • There is also an open investigational new drug application (IND) in the United States for TSHA-118 in CLN1 disease.
  • Introduction of a functional CLN1 gene with TSHA-118 treatment offers a potentially effective therapeutic approach that addresses the root cause of the disease.
  • TSHA-118 is a self-complementary AAV9 gene replacement therapy designed to express a human codon-optimized CLN1 transgene for the treatment of CLN1 disease.
  • The global trial is a single arm, open-label Phase 1/2 trial evaluating TSHA-118 for the treatment of CLN1 disease utilizing commercial grade material.

Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202, a Clinical Stage, Novel, One-time Gene Therapy for CLN1 Disease

Retrieved on: 
Lunedì, Agosto 17, 2020
Branches of biology, Articles, Biology, Gene delivery, PPT1, Applied genetics, Genetic engineering, Emerging technologies, Medical genetics, Therapy, Gene therapy, Lysosomal storage disease

We are excited to continue working closely with Dr. Gray to rapidly advance this promising gene therapy into the clinic.

Key Points: 
  • We are excited to continue working closely with Dr. Gray to rapidly advance this promising gene therapy into the clinic.
  • ABO-202 is a one-time, self-complementary AAV (serotype 9) gene therapy designed to deliver a functional copy of the palmitoyl-protein thioesterase 1 (PPT1) gene.
  • ABO-202 is a novel, one-time gene therapy for CLN1 disease, a rapidly progressing rare lysosomal storage disease with no approved therapy.
  • Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease.