NAGLU | Jotup

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

Retrieved on:

Monday, February 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.

M6P Therapeutics Presents Promising Preclinical Data in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ 2022

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Friday, February 11, 2022

Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Other Health, Patient, Learning, NAGLU, Drug development, CEO, Tissue, 2021 Essex County Council election, Genetic distance, Glycosaminoglycan, A-DNA, Phosphorylation, Enzyme, LSD, Hope, Enzyme replacement therapy, Washington University in St. Louis, Technology, Gaucher's disease, Cell, Division, Glycogen storage disease, Fabry disease, Liver, Q&A, Glycogen, Therapy, Research and development, Spleen, Central nervous system, Mouse, Research, Publication, Glycogen storage disease type II, Metabolic disorder, Lysosome, Muscle, GAA, Pharmaceutical industry, Fine chemical, Medical device, Vaccine, Copper, LSD (disambiguation), Gaucher, Sanfilippo, Genetics, the Annual WORLDSymposium™, M6P Therapeutics, THE ANNUAL WORLDSYMPOSIUM™, M6P THERAPEUTICS

M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical data at the 18th Annual WORLDSymposium, a research conference dedicated to lysosomal diseases.

Key Points:

M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical data at the 18th Annual WORLDSymposium, a research conference dedicated to lysosomal diseases.
In four poster presentations, including two Contemporary Forum presentations, M6P Therapeutics researchers reported preclinical efficacy results for LSDs, including Sanfilippo B syndrome, Gaucher disease, and Pompe disease.
M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted enzyme replacement and gene therapies for lysosomal storage disorders (LSDs).
M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

M6P Therapeutics Presented Data on M041, A Recombinant Enzyme Therapy, for the Treatment of Sanfilippo B Syndrome at MPS 2021

Retrieved on:

Wednesday, July 28, 2021

These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.

Key Points:

These data illustrate M041s potential as a recombinant enzyme therapy utilizing a recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) with improved mannose 6-phosphorylation.
There are currently no approved therapies for Sanfilippo B syndrome, also known as MPS IIIB.
Sanfilippo B syndrome is characterized by a defect in the NAGLU gene providing instructions for producing the enzyme alpha-N-acetylglucosaminidase.
M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs).

Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH)

Retrieved on:

Tuesday, February 9, 2021

Syndromes, Health, Clinical medicine, Medicine, Sanfilippo syndrome, Mucopolysaccharidosis, Sanfilippo, NAGLU, Morquio syndrome

Stable motor function was seen in all patients compared to pre-treatment, with follow-up ranging from 9 months to 1.5 years.

Key Points:

Stable motor function was seen in all patients compared to pre-treatment, with follow-up ranging from 9 months to 1.5 years.
MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare and life-threatening neurometabolic disease with no approved treatments.
Enrollment is expected to complete (n=5) this year and the company intends to release additional interim results later in 2021.
Mucopolysaccharidosis typeIIIA(MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease.