SGSH

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

Retrieved on: 
Wednesday, February 7, 2024
Traffic barrier, Sanfilippo, Genomics, Hearing, Growth, BBB, AFL, Metabolism, Feinberg School of Medicine, Spleen, Dermatan sulfate, Patient, Cerebrospinal fluid, University of North Carolina, FDA, Cognition, COMPASS, Mouse, CSF, Physician, Hunter syndrome, MPS II, Brain, Behavior, MPS, Mucopolysaccharidosis, Hunting, University, SGSH, ETV, Liver, Alfa, Glycogen storage disease, Pharmaceutical industry

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

Denali Therapeutics Announces Presentations on Its Investigational Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapies at the Upcoming 2024 WORLDSymposium™

Retrieved on: 
Thursday, February 1, 2024
Traffic barrier, SAN, BBB, Biomarker, Poster, Degeneracy, CNS, MPS II, Therapy, MPS, Interim analysis, SGSH, ETV, Glycogen storage disease

SOUTH SAN FRANCISCO, Calif., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.
  • Presentations on DNL310 will include new two-year data on clinical outcomes and previously presented data on biomarkers from the ongoing Phase 1/2 study in MPS II.
  • In addition, an oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model.
  • The presentation schedule at WORLDSymposium™ is as follows:
    Title: DNL310 Phase 1/2 Case Study Demonstrates Properties of Raw, Standard and Growth Scale Scores for Adaptive Behavior Scales (Poster #80)
    Title: DNL310 Normalizes Primary Storage Substrates, Corrects Biomarkers of Lysosomal Dysfunction and Reduces Biomarkers of Neuronal Injury (Neurofilament Light Chain) in MPS II: 2-Year Interim Analysis of a Phase 1/2 Study (Poster #34)
    Title: ETV:SGSH, a Brain-Penetrant Enzyme Transport Vehicle for SGSH, Improves Lysosomal and Microglial Morphology, Degeneration and Cognitive Behavior in MPS IIIA Mice
    Title: 3rd Annual Robert J. Gorlin Symposium – Beyond the Blood Brain barrier: Strategies for Treating the CNS

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

Retrieved on: 
Monday, January 8, 2024
CSF, Lysosomal storage disease, Cerebrospinal fluid, Risk, Sanfilippo syndrome, Amyloid, BBB, UC, Sanfilippo, Manuscript, ETV, ALS, OTV, SNCA, IDS, Asos, Conference, Cognition, RIPK1, Fluid mechanics, Neuroinflammation, Premedication, Glycogen storage disease, Amyloid beta, SGSH, Therapy, COMPASS, Traffic barrier, Hunting, N-sulfoglucosamine sulfohydrolase, Hunter syndrome, ISR, NewCo, Amyloid-related imaging abnormalities, Inflammation, CNS, Neurodegenerative disease, Takeda, Biomarker, Alpha-synuclein, K2, Biology, DSM-IV codes, LUMA, Brain, MPS, Investigational New Drug, PTV, ARIA, Nerve, AFL, Pathology, MPS II, Interim, BioRxiv, Plaque, MAPT, LRRK2, Elevator mechanic

The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, November 7, 2023
Neurofilament light polypeptide, MPS II, CLE, Biomarker, Therapy, MS, BioRxiv, ETV, Ulcerative colitis, BBB, ALS, PTV, ATV, ROFN, Definitive, Traffic barrier, Hunter syndrome, Phase 10, Denali, MPS, Antibody, Biology, Safety, Patient, AFL, Plaque, Fluid mechanics, ASO, ARIA, RIPK1, Asos, Research, Cerebrospinal fluid, ISR, SGSH, DSM-IV codes, Nerve, Brain, Society, Total, OTV, N-sulfoglucosamine sulfohydrolase, Hunting, LUMA, Glycogen storage disease, Manuscript, UC, CSF, Amyloid-related imaging abnormalities, Takeda, COMPASS, Pathology, CNS, Sanfilippo, Neuroinflammation, Collaboration, Multiple sclerosis, Sanfilippo syndrome, IDS, Cognition, Mutation, SAN, Pharmaceutical industry, Medical imaging, Fine chemical, Cryptocurrency, Biogen, LRRK2, Metabolism, Sanofi

SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.

Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT

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Friday, May 19, 2023
Kaufman, UNIPD, Prefrontal cortex, Cerebrospinal fluid, Plasma, Motion, Frasier, Protein, Thalamus, Acquisition, Patient, Atidarsagene autotemcel, Randomized controlled trial, The New England Journal of Medicine, Cell, Disease, IDUA, UOM, Enzyme replacement therapy, Phenotype, DSM-IV codes, Tissue, University, Hip, Gene, Orchard, GRN, Dementia, San Raffaele Hospital, Growth, HSC, Cognition, Natural history, Kyphosis, Biomarker, MPS, Șaeș, Frontotemporal dementia, Webcast, Mortality, Therapy, Central nervous system, Doctor of Philosophy, Speech, HSCT, CNS, EDT, Health, MRI, SGSH, Dysplasia, Society, FTD, Finnish heritage disease, Hippocampus, Medical imaging, Pharmaceutical industry, Vaccine, Medical device, Nursing, Dentistry

BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.

Key Points: 
  • The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
  • At baseline, most patients presented with severe joint range of motion impairment, severe acetabular (hip) dysplasia and varying degrees of dorso-lumbar kyphosis.
  • Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
  • Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.

Denali Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Business Highlights

Retrieved on: 
Monday, February 27, 2023
Hearing, Asos, IND, Lupus, Biomarker, PTV, Biology, Therapy, Neuroinflammation, ETV, Multiple sclerosis, Hunter syndrome, ALS, Safety, MS, TREM2, Takeda, Standard of care, DSM-IV codes, Patient, ATV, SGSH, IDS, CLE, Hospital, ISR, Parkinson's disease, COMPASS, RIPK1, LRRK2, Type 3 submergence transport vehicle, MPS II, CSF, Brain, Massachusetts General Hospital, Bank, BBB, CNS, MGH, Glycogen storage disease, MPS, The Annual, International Symposium on Endovascular Therapy, Traffic barrier, Sanfilippo syndrome, Bank of America, Sanfilippo, N-sulfoglucosamine sulfohydrolase, LUMA, ASO, SAN, Conference, Hunting, OTV, Pharmaceutical industry, Medical imaging, Medical device, Vaccine, Fine chemical, Cryptocurrency, Biogen, Sanofi

In February 2023, at the WORLDSymposiumTM, Denali reported additional interim data from the open-label, single-arm Phase 1/2 study of DNL310.

Key Points: 
  • In February 2023, at the WORLDSymposiumTM, Denali reported additional interim data from the open-label, single-arm Phase 1/2 study of DNL310.
  • The Phase 2/3 COMPASS study continues to enroll up to 54 participants with MPS II with and without neuronopathic disease.
  • In October 2022, Denali raised net proceeds of approximately $296.2 million through a public offering of its common stock.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were $1.34 billion as of December 31, 2022.

Denali Therapeutics Announces New Interim Data from DNL310 Phase 1/2 Study for MPS II and DNL126 Preclinical Data for MPS IIIA at WORLDSymposium™

Retrieved on: 
Wednesday, February 22, 2023
Cerebrospinal fluid, Genetics, Hearing, COMPASS, Central nervous system, Vineland Adaptive Behavior Scale, Infant, University, September 2022 United Kingdom mini-budget, PDF, Chapel Hill, Sanfilippo, MPS II, Dermatan sulfate, University of North Carolina, IND, CSF, N-sulfoglucosamine sulfohydrolase, Biomarker, Interim, GM3, Standard of care, SGSH, Glycogen storage disease, Denali, Cognition, Behavior, MPS, ABR, GM2, Event, ETV, Antibody, Urine, Enzyme replacement therapy, Hunting, Traffic barrier, Sanfilippo syndrome, Hunter syndrome, Ganglioside, Medical device, Pharmaceutical industry

DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim Phase 1/2 data are being presented at the 19th Annual WORLDSymposium™ in Orlando, Florida, February 22-26, 2023.
  • “As the study progresses and enrollment continues for the global Phase 2/3 COMPASS study, I am excited to learn more about the potential of DNL310 to offer meaningful benefit for the entire MPS II patient population."
  • The safety profile of DNL310 remains consistent with standard of care, now with data up to two years of treatment with DNL310.

Denali Therapeutics Announces Presentations on Enzyme Transport Vehicle (ETV) Development Programs for MPS Diseases at the Upcoming WORLDSymposium™

Retrieved on: 
Wednesday, February 15, 2023
Biomarker, Brain, Hunting, Traffic barrier, PDF, Safety, Sanfilippo, Inflammation, MPS, Poster, Therapy, COMPASS, Investigational New Drug, SGSH, Hunter syndrome, Denali, MPS II, Glycogen storage disease, SAN, ETV, IND, Pharmaceutical industry

DNL310 is an investigational, ETV-enabled, brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II (Hunter syndrome).

Key Points: 
  • DNL310 is an investigational, ETV-enabled, brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II (Hunter syndrome).
  • Denali is evaluating DNL310 in the global Phase 2/3 COMPASS study, which is enrolling participants with MPS II in North America, South America, and Europe, and in an open-label, single-arm Phase 1/2 study.
  • In addition, another oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model.
  • PDFs of the oral presentations will be posted on Denali’s corporate website under the Investor Events section on the corresponding date and time of the presentation.