IDUA

Cellectis Presents Novel TALEN® Editing Processes Enabling Highly Efficient Gene Correction and Gene Insertion in HSPCs

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星期一, 四月 22, 2024
Brain, Euronext Growth, Pericyte, IDUA, Traffic barrier, Integrin alpha M, HSPC, Mouse, CD4, Myelocyte, CD20, Cell, Intron, DNA, Therapy, Patient, Gene, Society, BBB, DSM-IV codes, Mucopolysaccharidosis, Vaccine

NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ:  CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, will present first data exploring novel TALEN® editing processes in hematopoietic stem and progenitor cells (HSPCs) at the American Society of Gene and Cell Therapy (ASGCT) being held on May 7-11, 2024.

Key Points: 
  • “These two posters showcase the potential and versatility of the TALEN® technology to promote efficient gene insertion in HSPCs.
  • Cellectis has developed a TALEN® mediated promoter-less intron editing technology that enables the expression of a therapeutic transgene exclusively by monocyte derived from edited HSPCs.
  • This novel editing approach is an important addition to the HSPC gene editing toolbox that might unlock new strategies for the treatment of metabolic and neurological diseases.
  • Cellectis has developed and optimized a novel gene editing process, leveraging the TALEN® technology and circular single strand DNA template delivery, enabling highly efficient gene insertion in HSPCs.

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

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星期二, 四月 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 Promising Preclinical Data in Alzheimer’s Disease at the 20th Annual WORLDSymposium™ 2024

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星期一, 二月 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.

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

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星期一, 二月 5, 2024
Partnership, Mortality, Hurler, Orchard, Growth, Congress, Cell, Division, Heart, ERT, Glycosaminoglycan, GAG, Health, Disease, Patient, Safety, Transplant, FDA, EMA, Șaeș, San Raffaele Hospital, ESGCT, HSCT, Food, POC, Therapy, MPS, Hurler syndrome, Kyowa Kirin, Acquisition, Enzyme replacement therapy, European Medicines Agency, Gene, University, Eye, IDUA, HSC, RPD, Pharmaceutical industry

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Immusoft To Present on ISP-001 Clinical Trial at WORLDSymposium™ 2024 Conference

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星期三, 一月 3, 2024
Research, Genetics, Clinical Trials, Stem Cells, Other Health, Biotechnology, Pharmaceutical, Health, Science, Other Science, Immunosuppression, Lysosomal storage disease, Conference, Risk, Infection, Iduronidase, Bone marrow, Hair loss, Infertility, Disease, Enzyme replacement therapy, Nausea, MPS I, Immune system, Clinical trial, MPS, University, ERT, Corporation, IDUA, ISP, Cell, Patient, Cell biology, Genetic engineering, Vaccine

Immusoft Corporation , a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024.

Key Points: 
  • Immusoft Corporation , a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024.
  • Immusoft is developing a new modality of gene encoded medicine that holds the promise of being durable and redosable.
  • The Company’s lead investigational drug candidate ISP-001, is a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease.
  • In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy).

Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH

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星期四, 十一月 30, 2023
Quality of life, Glycosaminoglycan, Food, ESGCT, Health, Antibody, FDA, Cell, Disease, Mortality, Tissue, Congress, Gene, HSC, POC, Therapy, RPD, Heart, Hurler syndrome, Eye, San Raffaele Hospital, Death, San Raffaele, ERT, European Medicines Agency, Cognitive development, IDUA, Growth, HSCT, Patient, Safety, Diagnosis, Hurler, Fast Track, Pharmaceutical industry

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

Key Points: 
  • Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs.
  • Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.
  • “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality.
  • Throughout the PoC study, treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.

Immusoft Administers the First Engineered B Cell in a Human Clinical Trial

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星期五, 十二月 15, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Disease, Regenerative medicine, Research, IDUA, MPS I, MPS, Therapy, Immunosuppression, Risk, Clinical trial, CIRM, Patient, ISP, Sugar, Division, Pediatric Blood & Cancer, Heart, DSM-IV codes, Infertility, Nausea, Infection, University, Cell, Hair loss, Pharmaceutical industry, Vaccine

Neither chemotherapy nor immunosuppression is required by Immusoft’s novel engineered B cell approach.

Key Points: 
  • Neither chemotherapy nor immunosuppression is required by Immusoft’s novel engineered B cell approach.
  • Immusoft has pioneered B cell engineering to create biofactories for durable in vivo therapeutic protein delivery, leading the field with over 60 issued and pending patents.
  • “Immusoft is advancing the world’s first engineered B cell therapy, which may have significant impact for patients with this rare disease and potentially many others.
  • We are excited by the potential of expanding the Immusoft engineered B cell platform to trials in additional therapeutic areas,” said Robert Sikorski, M.D., Ph.D., Chief Medical Officer, Immusoft.

Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023

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星期四, 十月 26, 2023
Glycosaminoglycan, Heart, Hurler syndrome, Hearing loss, Congress, Photophobia, POC, ERT, IDUA, Cell, Disease, MPS, Antibody, Gene, San Raffaele Hospital, HSC, The New England Journal of Medicine, ESGCT, Eye, Safety, European Society for Primary Immunodeficiencies, Therapy, Patient, Diagnosis, Death, Hurler, San Raffaele, European Society of Gene and Cell Therapy, HSCT, Pharmaceutical industry, Medical device, Medical imaging

The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.

Key Points: 
  • The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.
  • “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life.
  • Importantly, following treatment with OTL-203, no patients reported photophobia (light sensitivity), or any other ophthalmological symptoms typically associated with MPS-IH.
  • Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability.

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

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星期五, 五月 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.

Immusoft Awarded $8M in Funding from the California Institute for Regenerative Medicine (CIRM) for MPS I Clinical Program

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星期五, 一月 27, 2023
Biotechnology, Pharmaceutical, Health, Hematopoietic stem cell transplantation, Iduronidase, California Institute for Regenerative Medicine, Enzyme replacement therapy, Trial of the century, Regenerative medicine, HSCT, Traffic barrier, ERT, Tissue, Safety, CIRM, Patient, Takeda Pharmaceutical Company, Engineering, MPS, Food, Research, Disease, MPS II, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Quality of life, DSM-IV codes, IDUA, Pharmaceutical industry, Vaccine

This clinical study is both significant for Immusoft and historic in the field of cell and gene therapies, as ISP-001 will be the first engineered B cell therapy to enter into human clinical trials.

Key Points: 
  • This clinical study is both significant for Immusoft and historic in the field of cell and gene therapies, as ISP-001 will be the first engineered B cell therapy to enter into human clinical trials.
  • Immusoft has pioneered the engineering of B cells to create biofactories for in vivo therapeutic protein delivery, leading the field with over 60 issued and pending patents.
  • MPS I affects the body’s ability to produce an essential enzyme, IDUA, resulting in progressive damage to tissues and organs.
  • “We are delighted to partner with CIRM for a second program, this time for MPS I, and appreciate their recognition of the importance of this program.