Traffic barrier

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.

Apertura Gene Therapy Unveils Novel Engineered AAV Capsids Targeting Human Transferrin Receptor 1 for Neurological Gene Therapy Delivery

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Thursday, January 4, 2024
Health, Neurology, Genetics, Research, Science, Pharmaceutical, Biotechnology, Liver, Traffic barrier, Broad Institute, Transferrin receptor 1, DSM-IV codes, BBB, Vector, MIT, Astrocyte, Brain, Transferrin, Endothelium, Gene expression, AAV, Engineering, CNS, Harvard University, Transport, Central nervous system, Iron, Patient, Psychiatry, Gene therapy, Neuron, Vaccine

Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).

Key Points: 
  • Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).
  • This transportation mechanism mediates crossing of the blood-brain barrier (BBB) to enable gene therapy delivery throughout the central nervous system (CNS).
  • “Our TfR1 capsids, with their well-characterized mechanism of action, offer the unique advantage of significantly reducing human translation risk – a major historical challenge of engineering novel AAV capsids.
  • Apertura is currently advancing two programs utilizing its TfR1 capsids for undisclosed neurologic conditions and is leveraging its platform technologies to engineer novel payloads to regulate genetic expression and develop additional AAV capsids targeting specific human receptors.

Breakthrough in Treating Alzheimer's Using Targeted Drug Delivery Reported in New England Journal of Medicine

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Wednesday, January 3, 2024
Health, Family, Neurology, Consumer, Pharmaceutical, University, Mental Health, Research, Managed Care, Education, General Health, Science, Seniors, Clinical Trials, Traffic barrier, RNI, FUS, Male, Female, BBB, Blood, Insightec, PET, Donanemab, Ageing, Brain, Clinical trial, Patient, Safety, Therapy, Medical imaging

Anti-amyloid-beta (Aβ) monoclonal antibody therapies, such as aduncaumab, lecanemab, and donanemab, can reduce amyloid-beta plaques and slow the progression of Alzheimer’s.

Key Points: 
  • Anti-amyloid-beta (Aβ) monoclonal antibody therapies, such as aduncaumab, lecanemab, and donanemab, can reduce amyloid-beta plaques and slow the progression of Alzheimer’s.
  • More than 98 percent of drugs do not readily cross the BBB, thus requiring systemic treatments with higher doses and more frequent therapies.
  • The FUS MRI-guided treatment helmet with more than 1,000 ultrasound transducers were directed to specific brain regions with high amyloid-beta plaques.
  • “We are hopeful that the work we are doing may lead to improvements in outcome for many other patients and their families coping with Alzheimer’s.”

Human medicines European public assessment report (EPAR): Brineura, cerliponase alfa, Date of authorisation: 30/05/2017, Revision: 7, Status: Authorised

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Tuesday, January 2, 2024
Patient, Brain damage, Medical Subject Headings, Marketing, Traffic barrier, European Commission, Agency, INN, Risk, Fever, Lower respiratory tract infection, Metabolism, Infection, Movement, Allergy, ATC, International, European, Spinal cord, Disease, Nose, Language, Brain, Pharyngitis, Skull, Hypersensitivity, European Medicines Agency, TPP1, Select, CLN2, Safety, Anatomy, Medicine, Vomiting, IIA, CHMP, Medical device

Human medicines European public assessment report (EPAR): Brineura, cerliponase alfa, Date of authorisation: 30/05/2017, Revision: 7, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Brineura, cerliponase alfa, Date of authorisation: 30/05/2017, Revision: 7, Status: Authorised

Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

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Tuesday, January 2, 2024
Spleen, Liver, Medical Subject Headings, Marketing, Lysosome, Traffic barrier, C-reactive protein, INN, Fever, Risk, Metabolism, Niemann–Pick disease, Allergy, Caregiver, ATC, Anaphylaxis, Itch, International, Enzyme replacement therapy, Headache, Disease, Nausea, ASMD, Tissue, Brain, Blood, Language, Heart, Inflammation, Hypersensitivity, Carbon monoxide, DSM-IV codes, European Medicines Agency, CNS, Myalgia, Select, Patient, Facial muscles, Safety, Fats, Anatomy, Vomiting, IIA, Rash, Medical device

Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Xenpozyme, olipudase alfa, Date of authorisation: 24/06/2022, Revision: 2, Status: Authorised

NKGen Biotech Announces Clearance of Clinical Trial Application by Health Canada for SNK01 NK Cell Therapy to Treat Alzheimer’s Disease

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Wednesday, December 27, 2023
Traffic barrier, Dementia, FDA, Disease, Clinical trial, Health Canada, Patient, Safety, Therapy, CTA, NK, Pharmaceutical industry

SANTA ANA, Calif., Dec. 27, 2023 (GLOBE NEWSWIRE) -- NKGen Biotech Inc. (Nasdaq: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic, and CAR-NK natural killer cell therapeutics, today announced that it has received a No Objection Letter (“NOL”) from Health Canada for its Clinical Trial Application (“CTA”) for a Phase 1/2a study to evaluate the safety, tolerability, and exploratory efficacy of SNK01 natural killer (“NK”) cell therapy for treatment of patients with moderate Alzheimer’s Disease (“AD”). SNK01 is an autologous, non-genetically modified NK cell product that has enhanced cytotoxicity and activating receptor expression.

Key Points: 
  • SNK01 is an autologous, non-genetically modified NK cell product that has enhanced cytotoxicity and activating receptor expression.
  • Phase 1 is an open label safety evaluation to determine the maximum tolerated dose and/or recommended Phase 2 dose of SNK01.
  • Phase 2 is a randomized, placebo controlled, multicenter trial evaluating the safety and efficacy of SNK01 in moderate AD patients.
  • We believe the CTA clearance by Health Canada further validates the potential of our neurodegenerative disease program to help address this growing problem,” said Paul Y.

U.S. Neurologists Eagerly Await Promising Oral Bruton’s Tyrosine Kinase Inhibitors in Multiple Sclerosis Treatment Pipeline, Though Recent Trial Readouts Might Hamper Initial Excitement

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Wednesday, December 13, 2023
AB, Traffic barrier, HCP, FDA, Multiple sclerosis, RealTime, Neurology, BTK, Merck Serono, Patient, Safety, Non-Euclidean geometry, RMS, Pharmaceutical industry

Exton, PA, Dec. 13, 2023 (GLOBE NEWSWIRE) -- Several clinical trials are underway for Bruton’s tyrosine kinase inhibitors (BTKi) targeting relapsing and progressive multiple sclerosis (RMS/PPMS).

Key Points: 
  • Exton, PA, Dec. 13, 2023 (GLOBE NEWSWIRE) -- Several clinical trials are underway for Bruton’s tyrosine kinase inhibitors (BTKi) targeting relapsing and progressive multiple sclerosis (RMS/PPMS).
  • As orally administered BTKis are already established in oncology, there was a notable level of awareness, familiarity, and anticipation surrounding these developmental assets amongst neurologists.
  • Through Spherix’s quarterly RealTime Dynamix™ market tracker, neurologists and MS specialists have expressed an increasing level of enthusiasm for these investigational therapies over time.
  • With the recent trial outcomes impacting two of the prominent BTKis in development, the optimistic prospects for this class now face uncertainty.

Skye Announces Clinical Development Plan in Obesity for Differentiated Peripheral CB1 Inhibitor, Nimacimab

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Monday, December 11, 2023
GIP, NAFLD, Traffic barrier, Cholesterol, Food, Șaeș, IND, Non-alcoholic fatty liver disease, Drug discovery, FDA, Kidney disease, Weight loss, Syringe, Trial of the century, Half-life, Obesity, Investigational New Drug, Chronic kidney disease, OTCQB, CNS, CB1, Biomarker, Prediabetes, Central nervous system, Patient, Safety, Albuminuria, Pharmaceutical industry

SAN DIEGO, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Skye Bioscience, Inc. (OTCQB: SKYE) ("Skye"), a pharmaceutical company developing drugs targeting the endocannabinoid system, announced today that it plans to develop nimacimab, the Company’s monoclonal antibody recently acquired from Bird Rock Bio, for weight loss and the treatment of obesity. The Company has filed an Investigational New Drug ("IND") application with the U.S. Food and Drug Administration ("FDA") for the initiation of a Phase 2 clinical study of nimacimab in patients with obesity and chronic kidney disease.

Key Points: 
  • Nimacimab is a negative-allosteric modulating antibody targeting the cannabinoid 1 receptor ("CB1"), which has been implicated as an important target in multiple cardiometabolic diseases including obesity and renal complications.
  • Obesity and kidney disease are highly correlated: 80% of patients who have kidney disease are also obese; 30% of obese patients have kidney disease.
  • Nimacimab effectively inhibits CB1 signaling and, based on preclinical and early clinical studies, is devoid of the CNS liabilities typically seen by small molecule drugs that target the CB1 receptor because it does not cross the blood-brain barrier.
  • Skye owns the worldwide rights to nimacimab, with patents issued in the U.S. and other territories including claims to cannabinoid 1 receptor antibodies with inverse agonist function.

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
Monday, December 11, 2023
Nerve, MLD, Traffic barrier, European Commission, Food, Agency, Priority review, Health care, Death, FDA, PDUFA, Civil service commission, HSC, MHRA, Therapy, Busulfan, ARSA, Mutation, Atidarsagene autotemcel, Genome, Central nervous system, Patient, Health, Pharmaceutical industry

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

YDS Pharmatech Launches Data Partner Program with N1 Life, Leveraging Proprietary Peptide Experimental Data to co-develop Generative AI-driven peptide design platform

Retrieved on: 
Tuesday, December 19, 2023
Traffic barrier, Partnership, Drug discovery, Doctor of Philosophy, AI, Biophysics, YDS, Skin

This groundbreaking collaboration is set to maximize the potential of AI for peptide design, utilizing N1 Life's proprietary experimental data on peptide tumor targeting, cell permeabilization, and barrier-penetration across cornea, skin and blood-brain barrier.

Key Points: 
  • This groundbreaking collaboration is set to maximize the potential of AI for peptide design, utilizing N1 Life's proprietary experimental data on peptide tumor targeting, cell permeabilization, and barrier-penetration across cornea, skin and blood-brain barrier.
  • "We are excited to welcome N1 Life into our Data Partner Program," said Dr. Xing Che, founder and CEO of YDS Pharmatech.
  • Moreover, the scarcity of negative data, which is seldom published but extremely valuable, is essential for training our AI models.
  • We are thrilled to leverage this model in conjunction with generative AI for novel peptide design.