Glycosaminoglycan

Denali Therapeutics Announces Presentations on DNL310 (ETV:IDS) Development Program in MPS II (Hunter Syndrome) at the Upcoming WORLDSymposium™

Retrieved on: 
Tuesday, February 1, 2022
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DNL310 is an investigational brain-penetrant enzyme replacement therapy intended to treat both central nervous system (CNS) and peripheral manifestations of MPS II (Hunter syndrome).

Key Points: 
  • DNL310 is an investigational brain-penetrant enzyme replacement therapy intended to treat both central nervous system (CNS) and peripheral manifestations of MPS II (Hunter syndrome).
  • A poster presentation will provide details on a planned, potentially registrational Phase 2/3 clinical trial of DNL310 in MPS II.
  • Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disease caused by mutations in the gene that encodes for the enzyme iduronate-2-sulfatase (IDS).
  • Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases.

Immusoft Receives $4M in Funding from the California Institute for Regenerative Medicine (CIRM)

Retrieved on: 
Monday, November 29, 2021
Biotechnology, Health, Stem Cells, Pharmaceutical, Clinical Trials, Patient, Tissue, IDS, Heart, MPS II, Cell, Iduronate-2-sulfatase, Technology, Enzyme replacement therapy, Industry, Regenerative medicine, California Institute for Regenerative Medicine, Glycosaminoglycan, Human, ERT, Program, MPS, Hurler syndrome, Research, Lists of diseases, Microdeletion syndrome, CA, ISP, CIRM, Immune system, Disease, Degenerative disease, Pharmaceutical industry, Vaccine, Renewable energy, Immusoft, CIRM, IMMUSOFT, CIRM

Currently available treatments for MPS II do not sufficiently provide therapeutic agents at stable levels, resulting in considerable residual burden of disease.

Key Points: 
  • Currently available treatments for MPS II do not sufficiently provide therapeutic agents at stable levels, resulting in considerable residual burden of disease.
  • Immusofts therapeutic is specially tailored to combine a cell therapy with a gene-encoded medicine.
  • In the case of ISP-002, the patients own B cells are programmed to express the therapeutic enzyme iduronate sulfatase (IDS).
  • Our goal is to always move the most promising research forward as fast as we can, said Dr. Maria Millan, President and Chief Executive Officer, California Institute of Regenerative Medicine (CIRM).

Orchard Therapeutics Announces New England Journal of Medicine Publication of Interim Proof-of-concept Study Results of OTL-203 for Hurler Syndrome

Retrieved on: 
Thursday, November 18, 2021
Quotient, Private Securities Litigation Reform Act, Patient, VCN, Review, Safety, Securities Act of 1933, NEJM, Time, Enzyme replacement therapy, The New England Journal of Medicine, Forward-looking statement, Genome, Hearing, Gene, Glycosaminoglycan, HSC, Cerebrospinal fluid, Motion, Șaeș, Hurler syndrome, SEC, Central nervous system, Hurler, IDUA, CD34, Manuscript, COVID-19, Lists of diseases, Goal, Health, Immunoglobulin G, GSK, U.S. Securities and Exchange Commission, MPS, Frasier, Twitter, Research, San Raffaele Hospital, Risk, GLOBE, Social media, File, Antibody, Therapy, LinkedIn, GAG, Disease, Medical device, Pharmaceutical industry, Birth control, Vaccine

BOSTON and LONDON, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) evaluating the safety and efficacy of OTL-203 for the treatment of the Hurler subtype of Mucopolysaccharidosis type I (MPS-IH). OTL-203 is an investigational autologous hematopoietic stem cell (HSC) gene therapy comprising an individual’s own CD34+ HSCs transduced ex vivo with a lentiviral vector encoding the alpha-L-iduronidase (IDUA) gene.

Key Points: 
  • Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme.
  • Current treatment options are associated with significant limitations and do not adequately address some of the more severe manifestations of disease.
  • In this ongoing non-randomized, single center proof-of-concept study, eight patients diagnosed with MPS-IH were treated with OTL-203 between July 2018 and December 2019.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.

Homology Medicines Initiates Clinical Trial for HMI-203, a One-Time Investigational Gene Therapy Candidate for Adults with MPS II (Hunter Syndrome)

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Monday, October 18, 2021
Plasma, Phenylketonuria, Private Securities Litigation Reform Act, Nasdaq, IND, Genetics, Antibody, Enzyme replacement therapy, Neurological disorder, Chapel Hill, Paroxysmal nocturnal hemoglobinuria, Serum, PKU, ASHG, Tissue, Glycosaminoglycan, Glycogen storage disease, SEC, Therapy, MPS II, JumpStart, FDA, Disease, ERT, IDS, Clinical trial, Patient, Male, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Failure, GLOBE, Food, PNH, Gene editing, Company, University, Microdeletion syndrome, Investigational New Drug, MPS, Safety, University of North Carolina School of Law, Hunter syndrome, Homology, Cerebrospinal fluid, GAG, COVID-19, Pharmaceutical industry, Vaccine

BEDFORD, Mass., Oct. 18, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it has initiated the Phase 1 trial for HMI-203, a one-time, in vivo gene therapy candidate for the treatment of adults with mucopolysaccharidosis type II (MPS II), or Hunter syndrome. The juMPStart trial is an open-label, dose-escalation study evaluating the safety and efficacy of a single intravenous (I.V.) administration of HMI-203. Hunter syndrome is a lysosomal storage disorder caused by mutations in the iduronate 2-sulfatase (IDS) gene leading to absent or deficient I2S enzymatic activity, which causes toxic lysosomal accumulation of glycosaminoglycans (GAGs). There are no treatments currently available that address both the peripheral organ and cognitive manifestations of the disease, and there remains a high unmet medical need for patients despite the availability of enzyme replacement therapy (ERT).

Key Points: 
  • The juMPStart trial is an open-label, dose-escalation study evaluating the safety and efficacy of a single intravenous (I.V.)
  • With todays milestone, we have also accomplished our goal of having three clinical programs by the end of 2021, with our gene therapy and gene editing clinical trials for PKU and the initiation of the juMPStart trial for Hunter syndrome.
  • We are executing on this and our other programs, and we look forward to our continued collaboration with the MPS II community.
  • In addition to safety endpoints, the trial plans to measure plasma I2S activity, urinary GAG levels and other peripheral disease manifestations.

Sigilon Therapeutics Announces Acceptance of Clinical Trial Application in the UK for SIG-005 for the Treatment of Mucopolysaccharidosis Type I

Retrieved on: 
Thursday, September 9, 2021

Our goal with SIG-005 is to develop a non-viral, durable, redosable, and controllable approach that can deliver sustained levels of IDUA to patients with MPS-1.

Key Points: 
  • Our goal with SIG-005 is to develop a non-viral, durable, redosable, and controllable approach that can deliver sustained levels of IDUA to patients with MPS-1.
  • With this approval, we remain on track to initiate our Phase 1/2 study in the UK in the second half of this year.
  • The Company has also filed a CTA in Brazil and plans to submit an Investigational New Drug Application with the U.S. Food and Drug Administration.
  • Sigilon Therapeutics seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform.

Halozyme Provides Summary Results Of Data For PEGPH20 Combination Treatments Presented At ESMO 2018 Congress

Retrieved on: 
Monday, October 22, 2018
Medicine, Cancer, Oncology, Health, Cancer organizations, Hyaluronidase, Antineoplastic drugs, Hyaluronic acid, PEGylation, Chemotherapy, Glycosaminoglycan, Halozyme, Inc.

The presentations were made at the annualEuropean Society for Medical Oncology (ESMO) 2018Congress, taking place October 19-23 in Munich.

Key Points: 
  • The presentations were made at the annualEuropean Society for Medical Oncology (ESMO) 2018Congress, taking place October 19-23 in Munich.
  • PEGPH20 is Halozyme's proprietary PEGylated recombinant human hyaluronidase enzyme that degrades hyaluronan (HA), a glycosaminoglycan or naturally occurring sugar in the body.
  • "These data reinforce the potential for PEGPH20 in combination with chemotherapy in these well-defined patient populations.
  • "PEGPH20 may provide a unique targeted approach for patients with late-stage cancers where new treatment options are needed."