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Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
Gastroenteritis, Genomics, Metabolism, Neutropenia, Brain, Nerve, Mutation, RPD, Disease, Incidence, Partnership, Central nervous system, Genetics, GSK, Stomatitis, Patient, Research, Fats, PRV, Traffic barrier, Child, Viral, Gene therapy, SAN, Genome, Urinary tract infection, Rash, Hepatomegaly, Priority review, Stem cell, Infection, Therapy, MLD, Kyowa Kirin, Febrile neutropenia, Fever, PSLI, Prognosis, Health, Family, FDA, San Raffaele Hospital, ARSA, Orchard, Food, Pharmaceutical industry

“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Clene Announces Peer-Reviewed Publication Characterizing the Protein Corona of the Investigational Neurodegenerative Disease Drug, CNM-Au8®

Retrieved on: 
Friday, March 15, 2024
Pharmacology, Toxicity, Protein corona, Brain, Plasma, Longevity, Multiple sclerosis, BBB, ALS, CNM, University college, Medicine, Gastrointestinal tract, Research, Gold, Blood, Traffic barrier, Radiology, Protein, Clinical trial, CSO, Neurodegenerative disease, Apolipoprotein, Research fellow, Copper

Simultaneously, the depletion of opsonin-based proteins in the corona extends their circulation time in the blood, as observed clinically.

Key Points: 
  • Simultaneously, the depletion of opsonin-based proteins in the corona extends their circulation time in the blood, as observed clinically.
  • “When we invented the electro-crystal-chemical method to grow clean-surfaced, highly faceted gold nanocrystals, we certainly had catalytic optimization in mind.
  • However, we also understood that the protein corona can control the location of our nanocrystals in vivo.
  • A Phase 3 registrational clinical trial of CNM-Au8 for the treatment of the progressive neurodegenerative disease, amyotrophic lateral sclerosis, is presently planned to launch in 2024.

Voyager Therapeutics Announces Appointment of Toby Ferguson as Chief Medical Officer

Retrieved on: 
Wednesday, March 13, 2024
AAV, ALS, Assistant, Central nervous system, University, Florida College, SOD1, CNS, Running, Traffic barrier, IND, Biogen, DSM-IV codes, Therapy, CMO, Medical director, University of Florida, Pharmaceutical industry

LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.

Key Points: 
  • LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.
  • During his tenure, he built and developed teams focused on neuromuscular and movement disorders, overseeing strategy for these areas across Biogen R&D.
  • Prior to joining Biogen, Ferguson was Assistant Professor of Neurology, Shriners Pediatric Research Center and Temple University School of Medicine.
  • Voyager represents for me an extraordinary opportunity to fulfill this mission.”

Novocure Announces 20 Presentations On Tumor Treating Fields, Highlighting Preclinical Effects in Pancreatic Cancer and Immune Effects, at American Association for Cancer Research (AACR) Annual Meeting 2024

Retrieved on: 
Friday, April 5, 2024
Technology, Research, Nanotechnology, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Oncology, Clinical trial, PDT, Macrophage, Downregulation and upregulation, Ovarian cancer, BRCA, ECM, Pericyte, Apoptosis, Nanoparticle, Cervical cancer, GBM, Patient, Neoplasm, Doxorubicin, Traffic barrier, Cancer, Triple-negative breast cancer, Proteomics, AACR, Glioblastoma, Pancreatic cancer, Phenotype, PARP, Cisplatin, Immunogenic cell death, Large intestine, TNBC, Animal, PDGFR, Pharmaceutical industry

Novocure (NASDAQ: NVCR) today announced 20 presentations on Tumor Treating Fields (TTFields) therapy will be delivered at the American Association for Cancer Research (AACR) Annual Meeting 2024, to be held April 5 to 10 in San Diego.

Key Points: 
  • Novocure (NASDAQ: NVCR) today announced 20 presentations on Tumor Treating Fields (TTFields) therapy will be delivered at the American Association for Cancer Research (AACR) Annual Meeting 2024, to be held April 5 to 10 in San Diego.
  • Presentations from Novocure-sponsored and partner programs include:
    Tumor Treating Fields (TTFields) targeted self assembling nanoparticles for pancreatic cancer treatment: In vitro and in vivo assessment.
  • Tumor Treating Fields (TTFields) induce an anti-tumor immune response in a pancreatic cancer mouse model.
  • Tumor Treating Fields (TTFields) show efficacy in Triple-Negative breast cancer (TNBC) cells alone and in combination with PARP inhibitor Talazoparib.

New Findings From Sunbird Bio Reveal Significant Potential of Specific Tau Proteins to Serve as Blood-Based Biomarker for Alzheimer’s Disease

Retrieved on: 
Monday, March 25, 2024
Oncology, Health, Neurology, General Health, Research, Science, Biotechnology, Diagnosis, EV, Brain, Plasma, Conference, Dementia, EVS, CSF, Tau, Alzheimer's disease, Sunbird, AUC, Patient, PET, Biomarker, Traffic barrier, APEX, Cerebrospinal fluid, Protein, Neuron, Trial of the century, Aggregation, DSM-IV codes, Blood, Medical imaging

“These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.

Key Points: 
  • “These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.
  • “This confirmed correlation between EV-bound tau in the blood and tau tangles in the brain combined with the early validation of our new tau assays is incredibly exciting.
  • The Sunbird Bio study being shared at Tau2024 evaluated the potential of EV-bound tau proteins in the blood as a method of directly detecting the tau proteins that aggregate in the brain.
  • They then used Sunbird Bio’s new proprietary tau assays to distinguish between EV-bound and unbound soluble forms of tau in plasma.

Intranasal Delivery of shRNA Edits to Silence 5-HT2A Receptor Shows Promise in Enhancing Memory and Alleviating Anxiety

Retrieved on: 
Thursday, March 21, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Mental Health, Genetics, Clinical Trials, Depression, Brain, Dementia, Rat, BBB, Memory, Central nervous system, Public, RNA, Mouse, Traffic barrier, Anxiety, Translational Psychiatry, MCI, Neurological disorder, DSM-IV codes, HTR2A, Intrinsic termination, Therapy, Optimism, Cas9, Vaccine

In a groundbreaking study, they successfully used a non-invasive intranasal delivery platform to administer short-hairpin RNA (shRNA) that targets the 5-HT2A receptor resulting in long-lasting improvements in memory and reduced anxiety in animal models.

Key Points: 
  • In a groundbreaking study, they successfully used a non-invasive intranasal delivery platform to administer short-hairpin RNA (shRNA) that targets the 5-HT2A receptor resulting in long-lasting improvements in memory and reduced anxiety in animal models.
  • Neurological disorders such as Mild Cognitive Impairment (MCI), depression, and anxiety disorders pose significant challenges to public health worldwide.
  • In this study, the researchers designed shRNA sequences to specifically target and silence the human HTR2A gene, which is associated with anxiety and memory.
  • The ability to bypass the blood-brain barrier using intranasal delivery opens up new avenues for precision-based therapeutics in neurological disorders."

Early Efficacy Results Show Primary Lung Tumor Stabilization and Complete Disappearance of Brain Metastases in Second Patient in Kiromic BioPharma’s Deltacel-01 Clinical Trial

Retrieved on: 
Tuesday, March 19, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Other Health, Principal, Safety, Lung cancer, Patient, BHCC, OTCQB, MRI, Traffic barrier, NSCLC, GDT, Medical imaging

Kiromic BioPharma, Inc. (OTCQB: KRBP) (“Kiromic” or the “Company”) reports early efficacy results showing primary lung tumor stabilization and the complete disappearance of brain metastases in the second patient in the Company’s Deltacel-01 Phase 1 clinical trial.

Key Points: 
  • Kiromic BioPharma, Inc. (OTCQB: KRBP) (“Kiromic” or the “Company”) reports early efficacy results showing primary lung tumor stabilization and the complete disappearance of brain metastases in the second patient in the Company’s Deltacel-01 Phase 1 clinical trial.
  • Deltacel-01 is evaluating Deltacel™ (KB-GDT-01), Kiromic’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC).
  • Preliminary results obtained six weeks from the beginning of treatment show Deltacel’s efficacy in controlling tumor growth, as well as favorable safety and tolerability.
  • Kiromic expects to announce initial safety, tolerability, and preliminary efficacy results from the third patient enrolled in Deltacel-01 by the end of March.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

Retrieved on: 
Wednesday, March 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.

73% CNS ORR! FDA Granted ODD to Utidelone Injectable (UTD1) from Biostar Pharma for the Treatment of Breast Cancer Brain Metastasis

Retrieved on: 
Friday, March 29, 2024
ODD, Lung cancer, HER2, Epothilone, Survival, Prognosis, Brain, BBB, Central nervous system, Synthetic biology, Patient, SAN, CNS, Etoposide, Triple-negative breast cancer, Glioma, Traffic barrier, CBR, Bevacizumab, Breast cancer, PFS, FDA, TNBC

As of now, there is only one drug approved for the treatment of BCBM globally, suggesting huge unmet medical needs [1].

Key Points: 
  • As of now, there is only one drug approved for the treatment of BCBM globally, suggesting huge unmet medical needs [1].
  • Utidelone has the ability to cross BBB due to its unique physicochemical characteristic and insusceptibility to P-glycoprotein-mediated efflux.
  • The FDA granted ODD to utidelone injectable for the treatment of BCBM based on these promising data.
  • Considering utidelone's excellent BBB-crossing capability and its therapeutic potential for brain tumors, Biostar Pharma also plans to advance the clinical studies of utidelone injectable for the treatments of other brain tumors such as lung cancer brain metastasis and glioma this year.