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Codexis Announces Several Key Presentations from its Gene Therapy Programs at 19th Annual WORLDSymposium™

Retrieved on: 
Wednesday, February 22, 2023
Tissue, Lysosome, GM1, Skin, Glycogen storage disease, MBBS, Patient, Half-life, Pain, Disease, Heart, Kaay Raav Tumhi, Partnership, ERT, Fabry disease, Abstract, CNS, Enzyme, Kidney, AAV, Serum, Fatigue, Transgene, Peripheral nervous system, Takeda Pharmaceutical Company, Protein engineering, OnDemand, DSM-IV codes, Poster, Enzyme replacement therapy, Pharmaceutical industry, Vaccine, Takeda, Fabry, Codexis

REDWOOD CITY, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company, today highlights several key presentations from its gene therapy programs from 19 th Annual WORLDSymposium™ , taking place from February 22-26, 2023, in Orlando, Florida.

Key Points: 
  • REDWOOD CITY, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company, today highlights several key presentations from its gene therapy programs from 19 th Annual WORLDSymposium™ , taking place from February 22-26, 2023, in Orlando, Florida.
  • Takeda is combining these improved transgenes with its gene therapy capabilities to develop novel product candidates for the treatment of rare genetic disorders.
  • Takeda’s poster presentation, titled, “Preventing Fabry disease progression in a symptomatic mouse model with a recombinant adeno-associated virus (rAAV) based gene therapy,” highlights its rAAV-based gene therapy candidate for the potential treatment of Fabry disease.
  • Codexis is also presenting two abstracts highlighting its gene therapy program in GM1 Gangliosidosis (GM1) at WORLDSymposium™.

4D Molecular Therapeutics Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials & Development Plans for Fabry Disease Cardiomyopathy at WORLDSymposium™

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Wednesday, February 22, 2023
Death, CPET, Safety, Quality of life, KCCQ, Heart, Real-time, ERT, Rituximab, FDA, Patient, Toxicity, GLS, Inflammation, Chelsea Handler, AAV, Biopsy, Dorsal root ganglion, RNA, Pharmaceutical industry, Medical device, Nursing

All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints.

Key Points: 
  • All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints.
  • Treatment was generally well tolerated, with transient acute aHUS being the only significant adverse event.
  • Data will also be presented at the WORLDSymposium™ 2023 in Orlando, Florida on February 25th, 2023.
  • We are developing 4D-310 for the treatment of Fabry disease cardiomyopathy, which is the primary cause of death and not addressed by current therapies.

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

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Wednesday, February 22, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Pfizer, Grade 2, Therapy, Central nervous system, Sickle cell disease, STAAR, IND, BLA, ERT, FDA, Patient, Fabry disease, Haemophilia, ASH, CNS, Plasma, Fetal hemoglobin, CMC, Acceleration, AAV, Kite, Society, Biogen, Novartis, Enzyme replacement therapy, Pharmaceutical industry, Vaccine, Video game, Sangamo Therapeutics, Fabry, Chemistry, Sanofi

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2022 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2022 financial results.
  • All five patients who began the dose escalation phase on enzyme replacement therapy (ERT) had been successfully withdrawn from ERT and remain off ERT today.
  • Revenues for the fourth quarter ended December 31, 2022 were $27.2 million, compared to $28.0 million for the same period in 2021.
  • These increases were partially offset by decreases of $13.9 million in revenue related to our collaboration agreement with Biogen.

Sangamo Therapeutics Announces Evidence of Clinical Benefit in Phase 1/2 STAAR Study in Fabry Disease

Retrieved on: 
Wednesday, February 22, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Female, Therapy, Central nervous system, Annual report, Southwest General Health Center, Week, Sangamo Therapeutics, Form, Heart, STAAR, Cell, ERT, Marie Cassidy, Patient, Fabry disease, Trial of the century, Plasma, Kidney, Pathology, PTC, Urine, Good Samaritan Hospital Medical Center (West Islip, New York), Data, Pharmaceutical industry, MD, Eastern Time Zone, Fabry

These data, which present new biomarker data and results from the first kidney biopsies in this study, indicate evidence of clinical benefit for isaralgagene civaparvovec in Fabry disease.

Key Points: 
  • These data, which present new biomarker data and results from the first kidney biopsies in this study, indicate evidence of clinical benefit for isaralgagene civaparvovec in Fabry disease.
  • “Fabry is a debilitating disease with life-long impact,” said Dr. Robert Hopkin, MD, Cincinnati Children’s Hospital Medical Center, and investigator of the Phase 1/2 study.
  • Dosing of the remaining patients in the expansion phase of the Phase 1/2 STAAR study is ongoing, with a total of 20 sites active and recruiting.
  • Dosing of the first patient in the Phase 3 trial could begin as early as the first part of 2024.

Additional Positive Interim Data from Phase I/II/III CAMPSIITE™ Trial of REGENXBIO's RGX-121 for the Treatment of MPS II (Hunter Syndrome) Presented at 19th Annual WORLDSymposiumTM

Retrieved on: 
Wednesday, February 22, 2023
Safety, Pediatrics, Central nervous system, Infant, Perelman School of Medicine at the University of Pennsylvania, Syndrome, University, Week, Vineland Adaptive Behavior Scale, Glycosaminoglycan, University of Pennsylvania, Presentation Public School, Bayley Scales of Infant Development, Biologics license application, GAG, MPS II, Hunter syndrome, Cohort, Acquisition, CSF, Biomarker, Socialization, ERT, Fluid mechanics, Patient, CNS, Immunosuppression, SD, Plasma, II, HS, MPS, Urine, Șaeș, Phase, Communication, Pharmaceutical industry, Vaccine, Medical imaging

"GAGs measured in CSF, specifically heparan sulfate, reflect disease manifestations in the CNS and are a direct cause of disease pathophysiology.

Key Points: 
  • "GAGs measured in CSF, specifically heparan sulfate, reflect disease manifestations in the CNS and are a direct cause of disease pathophysiology.
  • "The data presented today are encouraging and continue to show the potential of a one- time gene therapy to provide meaningful, durable clinical benefits to the MPS II community.
  • Data presented were from the Phase I/II portion of the CAMPSIITE trial in which the primary endpoint is to evaluate the safety of RGX-121.
  • Dr. Ficicioglu will share this data in a platform presentation at the WORLDSymposium Sunday, February 26, 2023, at 9 a.m.

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

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Friday, January 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.

ILUS Summarizes Its Progress and Projections Following Its 2nd Annual Shareholder Meeting   

Retrieved on: 
Monday, February 6, 2023
Annual report, Form 10-K, Acquisition, America's News Headquarters, OTC, Mergers and acquisitions, National Interest Party (Nigeria), ERT, Security (finance), Management

The company held its 2nd Annual Shareholder Meeting in Miami on the 27th of January 2023, summarizing 2022 performance and outlining the roadmap for 2023.

Key Points: 
  • The company held its 2nd Annual Shareholder Meeting in Miami on the 27th of January 2023, summarizing 2022 performance and outlining the roadmap for 2023.
  • “Thank you to those Shareholders who attended our Annual Shareholder Meeting both in person and online, particularly those who travelled from far to join us in person.
  • A lot of information was covered on the day which is why we have provided this brief summary with some key highlights.
  • A full recording of the 2nd ILUS Annual Shareholder Meeting can be found on the ILUS International YouTube channel or at this link: https://youtu.be/ADoERd1JDns
    For further information on the companies, please see their communication channels:

First Patient Dosed in CANbridge Pharmaceuticals CAN103 Phase 2 Trial for the Treatment of Gaucher Disease in China

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Thursday, January 19, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, General Health, Health, Clinical Trials, Hepatosplenomegaly, Disease, WuXi Biologics, Fracture, Anemia, Degenerative disease, Peking Union Medical College Hospital, Bone pain, Enzyme replacement therapy, Death, Safety, Macrophage, Diagnosis, Thrombocytopenia, Treatment, Partnership, Patient, GD, Glycogen storage disease, ERT, III, Doctor of Philosophy, Hematology, Gaucher's disease, Pharmaceutical industry, Gaucher

Most patients with Gaucher disease in China do not have access to approved treatments due to cost barriers.

Key Points: 
  • Most patients with Gaucher disease in China do not have access to approved treatments due to cost barriers.
  • “We are pleased to be moving into the Phase 2 part of our registrational Chinese trial in Gaucher disease,” said James Xue, Ph.D., CANbridge founder, chairman and CEO.
  • “Gaucher disease was the first rare disease to have an enzyme replacement therapy approved in China.
  • “CAN103 has the potential to provide a much-needed new treatment for Gaucher’s disease, where there is a high unmet medical need in China.”

Asia Pacific Glycomic Therapeutics Market Report 2023: Featuring Biomarin Pharmaceutical, Genzyme, Bayer, Glaxosmithkline & F. Hoffmann-La Roche - ResearchAndMarkets.com

Retrieved on: 
Monday, January 9, 2023
Biotechnology, Pharmaceutical, Health, Glycosaminoglycan, Cataract, Bayer, Erythropoietin, GlaxoSmithKline, COVID-19, Enzyme replacement therapy, Government, Heparin, Precision medicine, Neuraminidase, Genzyme, Glycomics, Interferon, Glycoprotein, Alzheimer's disease, Proteoglycan, Gaucher's disease, Therapy, Center for Urban and Regional Analysis, Tissue plasminogen activator, Anemia, Research, Cancer, Thrombosis, Glycosphingolipid, GPI, ERT, Pharmaceutical industry, Fine chemical, Sanofi, Selectin

The Asia Pacific glycomics therapeutics market is segmented into China, Japan, India, South Korea, Australia, and the Rest of Asia Pacific.

Key Points: 
  • The Asia Pacific glycomics therapeutics market is segmented into China, Japan, India, South Korea, Australia, and the Rest of Asia Pacific.
  • The Asia Pacific glycomic therapeutics market is segmented on the basis of class, structure, indications, mode of action, and country.
  • Based on country, the Asia Pacific glycomic therapeutics market is segmented into China, Japan, India, Australia, South Korea, and the Rest of Asia Pacific.
  • ; and F. Hoffmann-La Roche Ltd. are the leading companies operating in the glycomic therapeutics market in Asia Pacific.

Insights on the Virtual Clinical Trials Global Market to 2027 - Key Players Include Dassault Systemes, Medable, Science 37 and THREAD

Retrieved on: 
Thursday, December 22, 2022
EMA, Clinical trial, Patient, EPRO, Cros, Medicine, Center for Drug Evaluation and Research, Medidata Solutions, European Medicines Agency, Drug development, AI, Growth, Science, COVID-19, Risk, HSA, ERT, Guest appearance, Medicines and Healthcare products Regulatory Agency, FDA, Human, CDE, Heads of Medicines Agencies, Dassault Systèmes, Accenture, DCT, Infection, Research, Artificial intelligence, MHRA, Medical device, Pharmaceutical industry, Medical imaging, IQVIA

The virtual clinical trials market is one of the innovative approaches for traditional clinical trials, through remote monitoring and more patient-centric approaches.

Key Points: 
  • The virtual clinical trials market is one of the innovative approaches for traditional clinical trials, through remote monitoring and more patient-centric approaches.
  • The COVID-19 pandemic is the significant factor that shifted the traditional clinical trials approach to the virtual clinical trials market.
  • The market players and the companies pioneer in clinical trials activities understand that decentralization in clinical trials is the future of the clinical trials space.
  • THREAD offers a unique, innovative, and highly advanced decentralized clinical trial platform in the virtual clinical trials market.