TFEB

Coave Therapeutics Receives Grant from the ALS Association to Advance its CTx-TFEB Program as a Potential Treatment for All Forms of ALS

Retrieved on: 
Thursday, February 29, 2024
ALS Association, Senior, ALS, Research, Alzheimer's disease, TFEB, Wang, Neuron, ALP, Hope, Trehalose, Neurodegenerative disease, Protein, Patient, Spinal cord, Gene, Disease, CSO, Diagnosis, Autophagy, AAV, Amyotrophy, Brain

The grant, which will support the development of Coave’s CTx-TFEB program through to preclinical proof-of-concept, has been made through the Association’s Lawrence and Isabel Barnett Drug Development Program .

Key Points: 
  • The grant, which will support the development of Coave’s CTx-TFEB program through to preclinical proof-of-concept, has been made through the Association’s Lawrence and Isabel Barnett Drug Development Program .
  • ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
  • Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe.
  • With this funding and invaluable support from the ALS Association, we are poised to move our CTx-TFEB program forward.

Coave Therapeutics to Collaborate with World-Renowned Institute of Neurodegenerative Diseases of Bordeaux to Develop Gene Therapy Programs Targeting Protein Degradation in Neurodegenerative Disorders

Retrieved on: 
Wednesday, September 14, 2022
Institute, Degenerative disease, Central nervous system, Neurodegeneration, University of Bordeaux 1, Autophagy, CNRS, AAV, MD, MSA, French National Centre for Scientific Research, Phenotype, Coronavirus Scientific Advisory Board (Turkey), Scientific method, IND, Parkinson's disease, Re Recher's Will Trusts, Partnership, National Centre for Disease Control, Therapy, INSERM, EB, CEO, TFEB, SAB, Multiple system atrophy, CNS, Eye, University, PD, IMN, Pharmaceutical industry, Vaccine, Medical device, Wood drying, Genetics, Idinvest Partners, Medicine

Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.

Key Points: 
  • Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.
  • We look forward to leveraging our collective strengths to best develop gene therapy programs for neurodegenerative diseases with the potential to improve patient outcomes."
  • "We are delighted to be collaborating with IMN to develop coAAVs carrying TFEB and explore these gene therapy constructs for the treatment of neurodegenerative diseases.
  • Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases.

Coave Therapeutics to Collaborate with World-Renowned Institute of Neurodegenerative Diseases of Bordeaux to Develop Gene Therapy Programs Targeting Protein Degradation in Neurodegenerative Disorders

Retrieved on: 
Wednesday, September 14, 2022
Institute, Degenerative disease, Central nervous system, Neurodegeneration, University of Bordeaux 1, Autophagy, CNRS, AAV, MD, MSA, French National Centre for Scientific Research, Phenotype, Coronavirus Scientific Advisory Board (Turkey), Scientific method, IND, Parkinson's disease, Re Recher's Will Trusts, Partnership, National Centre for Disease Control, Therapy, INSERM, EB, CEO, TFEB, SAB, Multiple system atrophy, CNS, Eye, University, PD, IMN, Pharmaceutical industry, Vaccine, Medical device, Wood drying, Genetics, Idinvest Partners, Medicine

Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.

Key Points: 
  • Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.
  • We look forward to leveraging our collective strengths to best develop gene therapy programs for neurodegenerative diseases with the potential to improve patient outcomes."
  • "We are delighted to be collaborating with IMN to develop coAAVs carrying TFEB and explore these gene therapy constructs for the treatment of neurodegenerative diseases.
  • Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases.

AI Therapeutics Announces Initiation of a Phase II Clinical Trial of AIT-101 for Treatment of ALS

Retrieved on: 
Thursday, January 13, 2022
Biomarker, Neurodegeneration, Coronavirus Scientific Advisory Board (Turkey), C9, Patient, ALS Functional Rating Scale - Revised, Clinical trial, Degenerative disease, Innovation, Population, Protein, Safety, Neuroscience, C9orf72, Disease, TFEB, GLOBE, Department, DNA, Deep learning, Intelligence, Evolutionism, Therapy, Pharmacokinetics, ALS, Butterfly network, Environment, Life expectancy, AI, Technology, Phase, National Medal, Vital capacity, Massachusetts General Hospital, PIKFYVE, Tesseract, Yale University, Amyotrophic lateral sclerosis, Pharmaceutical industry, Medical device, Genetics

GUILFORD, Conn., Jan. 13, 2022 (GLOBE NEWSWIRE) -- AI Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today the initiation of a Phase II study for a promising new approach to treat amyotrophic lateral sclerosis (ALS).

Key Points: 
  • GUILFORD, Conn., Jan. 13, 2022 (GLOBE NEWSWIRE) -- AI Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today the initiation of a Phase II study for a promising new approach to treat amyotrophic lateral sclerosis (ALS).
  • AI Therapeutics drug candidate AIT-101 employs a novel approach to clearing toxic protein aggregates that accumulate in the brains of ALS patients and are a hallmark of the disease.
  • The potential therapeutic value for PIKfyve kinase inhibition in ALS and other neurodegenerative diseases was initially predicted by both the AI Therapeutics Guardian Angel Platform and by our own observations.
  • The Phase II clinical trial will use LAM-002A, the current formulation of the active ingredient of AIT-101.

Casma Therapeutics Announces Publication in Science Advances Highlighting Novel Mechanism Related to TFEB Activation and Lysosomal Biology

Retrieved on: 
Monday, October 4, 2021
Biotechnology, Therapy, Molecular biology, Arrest, Glycogen storage disease, FLCN, Organelle, TFEB, Inflammation, EB, Science Advances, Patient, Casma, Autophagy, MTOR, Lysosome, Disease, Research, Drug discovery, Lists of diseases, Neurodegeneration, Enzyme, GLOBE, Pharmaceutical industry

TFEB is the master regulator of lysosomal function and is required for maintaining cellular and whole-body health.

Key Points: 
  • TFEB is the master regulator of lysosomal function and is required for maintaining cellular and whole-body health.
  • TFEB regulates the levels of these enzymes, many of which are mutated in lysosomal storage disorders and neurodegeneration.
  • TheCasma publication describes a novel mechanism to regulate TFEB activation without impacting the ability of mTOR to phosphorylate other targets involved in general cellular activity, said Andrea Ballabio, M.D.
  • The Science Advances paper concluded that the GABARAP-FLCN/FNIP-TFEB axis serves as a molecular sensor that regulates lysosomal homeostasis within the autophagy-lysosomal network.

Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 to Treat Krabbe Disease

Retrieved on: 
Thursday, September 2, 2021
Glycogen storage disease, Drug development, Phenotype, Therapy, Lists of diseases, Genetic disorder, Life, Gene, Trial of the century, Krabbe disease, Metabolic disorder, GLOBE, GALC, EB, Food, Disease, Myelin, Death, KD, Lipid storage disorder, LLM, Inflammation, Degenerative disease, Board, Enzyme, CEO, Psychosine, Apoptosis, TFEB, FDA, Neurodegeneration, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

PARAMUS, N.J., Sept. 02, 2021 (GLOBE NEWSWIRE) -- PolaryxTherapeutics, Inc. ("Polaryx"), abiotechcompany developing small molecule therapeutics forlysosomalstorage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.

Key Points: 
  • PARAMUS, N.J., Sept. 02, 2021 (GLOBE NEWSWIRE) -- PolaryxTherapeutics, Inc. ("Polaryx"), abiotechcompany developing small molecule therapeutics forlysosomalstorage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.
  • Krabbe disease is a rare, genetic disorder caused by the deficiency of lysosomal enzyme, galactocerebrosidase (GALC).
  • Under the U.S. Orphan Drug Act, the FDA's Office of Orphan Products Development provides sponsors with special status and incentives to facilitate the drug development for rare disease affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial cost, exemptions from certain FDA application fees, and assistance in clinical trial design.
  • "Granting by the FDA of Orphan Drug Designation for PLX-200 in Krabbe disease supports the use of PLX-200 to treat key lysosomal storage disorders with unmet medical needs.

Polaryx Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses

Retrieved on: 
Monday, August 30, 2021
Hearing, Ganglioside, Niemann–Pick disease, Glycogen storage disease, Tay–Sachs disease, RXR, Drug development, Therapy, Lists of diseases, GM2, Gene, Lysosome, Trial of the century, Sandhoff disease, HEXB, Seizure, EB, Food, Disease, Neuron, LLM, Inflammation, Lysosomal storage disease, Board, Neurodegeneration, HEXA, Enzyme, Patient, CEO, Hexosaminidase, Apoptosis, TFEB, PPAR, FDA, Gangliosidosis, GM2 gangliosidoses, Nervous system, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, Vaccine

PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.

Key Points: 
  • PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
  • "We are very pleased to be granted Orphan Drug Designation for PLX-200 from the FDA for the treatment of GM2 gangliosidoses.
  • Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients.
  • Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options available.

Seelos Therapeutics Announces Issuance of a Patent for Trehalose (SLS-005) in Australia

Retrieved on: 
Friday, January 29, 2021
Branches of biology, Biology, TFEB, Autophagy, Trehalose, Lysosome, MTOR, Protein aggregation

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells.

Key Points: 
  • Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells.
  • In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material.
  • Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression.
  • Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

Seelos Therapeutics Announces Issuance of a Patent for Trehalose (SLS-005) in Israel

Retrieved on: 
Monday, December 21, 2020
Branches of biology, Biology, TFEB, Autophagy, Trehalose, Lysosome, MTOR

Seelos recently received orphan drug designation for SLS-005 in ALS and announced their inclusion in the HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.

Key Points: 
  • Seelos recently received orphan drug designation for SLS-005 in ALS and announced their inclusion in the HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.
  • Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells.
  • Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression.
  • Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

Seelos Therapeutics Receives Notice of Allowance for an Additional US Patent for Trehalose (SLS-005)

Retrieved on: 
Thursday, September 10, 2020
Branches of biology, Neurological disorders, Organ systems, Rare diseases, Autosomal dominant disorders, Cognitive disorders, TFEB, Trinucleotide repeat disorders, Autophagy, Trehalose, Dentatorubral–pallidoluysian atrophy, SBMA

The allowed claims cover a method of using trehalose (SLS-005) to treat several neurodegenerative conditions including spinocerebellar ataxia (SCA), spinal and bulbar muscular atrophy (SBMA), dentatomral-pailidoluyssan atrophy (DRPLA), Pick's disease, corticobasaldegeneration (CBD), progressive supranuclear palsy (PSP), and frontotemporal dementia.

Key Points: 
  • The allowed claims cover a method of using trehalose (SLS-005) to treat several neurodegenerative conditions including spinocerebellar ataxia (SCA), spinal and bulbar muscular atrophy (SBMA), dentatomral-pailidoluyssan atrophy (DRPLA), Pick's disease, corticobasaldegeneration (CBD), progressive supranuclear palsy (PSP), and frontotemporal dementia.
  • Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells.
  • Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression.
  • Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.