Traffic barrier

Brenig Therapeutics Presented New Data for the Best-in-Class LRRK2 Inhibitor Targeting Parkinson's Disease at the ACS Spring 2024 Conference

Retrieved on: 
Monday, March 18, 2024
IPD, Brain, Conference, Disease, PD, CSF, Lung, OrbiMed, Traffic barrier, Parkinson's disease, LRRK2, Therapy, Safety, ACS, Kidney, Pharmaceutical industry

DOVER, Del., March 18, 2024 /PRNewswire/ -- Brenig Therapeutics, a portfolio company of Torrey Pines Investments, OrbiMed Advisors, and BioGeneration Ventures, focusing on treatment of neurodegenerative diseases, presented new safety data on its LRRK2 inhibitor drug candidate at the ACS Spring 2024 Conference at the New Orleans Ernest N. Morial Convention Center, on March 17th, 2024.

Key Points: 
  • DOVER, Del., March 18, 2024 /PRNewswire/ -- Brenig Therapeutics, a portfolio company of Torrey Pines Investments, OrbiMed Advisors, and BioGeneration Ventures, focusing on treatment of neurodegenerative diseases, presented new safety data on its LRRK2 inhibitor drug candidate at the ACS Spring 2024 Conference at the New Orleans Ernest N. Morial Convention Center, on March 17th, 2024.
  • Brenig's innovative approach to the rationally designed LRRK2 inhibitor (BT-0267) marks a significant stride in the company's mission to combat neurodegenerative diseases.
  • LRRK2, a key protein implicated in Parkinson's disease (PD), has been a challenging therapeutic target.
  • Current data highlights the superior safety profile of LRRK2 Inhibitor BT-0267 with an exceptional CSF to plasma ratio, in vivo efficacy of BT-0267 in brain and no visible lung and kidney morphological changes compared to other LRRK2 inhibitor candidates.

Mustang Bio Announces Publication in Nature Medicine of Data from Phase 1 Trial Evaluating MB-101 IL13Rα2-targeted CAR T-Cells in High-Grade Glioma

Retrieved on: 
Thursday, March 7, 2024
T cell, Glioblastoma, Arms, ICT, ICV, Traffic barrier, Toxicity, Headache, Biopsy, DSM-IV codes, Cerebrospinal fluid, City, Cancer, Central nervous system, CXCL10, TME, Cerebral edema, Dual, Hope, GBM, Safety, CXCL9, Fatigue, Hypertension, Translation, CNS, Tumor microenvironment, Patient, Arm, Spinal cord, Survival, Brain, Nature Medicine, Infrared spectroscopy correlation table, Vaccine

WORCESTER, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced Phase 1 clinical data were published in Nature Medicine that demonstrated the promising safety and clinical activity of Mustang’s MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of patients with recurrent and refractory malignant glioma, including glioblastoma.

Key Points: 
  • MB-101 was developed by City of Hope, one of the largest cancer research and treatment organizations in the United States, and exclusively licensed to Mustang.
  • Central nervous system (CNS) increases in inflammatory cytokines, including IFNγ, CXCL9, and CXCL10, were associated with CAR T-cell administration and bioactivity.
  • Primary endpoints were safety and feasibility, with secondary endpoints measuring therapy-related cytokine dynamics, CAR T-cell persistence and clinical outcomes.
  • Dr. Brown has a financial interest in Mustang and has previously been a paid consultant for the company.

Alector Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, February 27, 2024
PD, GSK, Arnon Rosenthal, Investment, Interest, ARIA, AbbVie, Webcast, Plasma, PET, MRI, Central nervous system, Traffic barrier, GPNMB, Cerebrospinal fluid, Breakthrough therapy, ALEC, Frontotemporal dementia, FTD, DSM-IV codes, European Medicines Agency, Food and Drug Administration, Canadian International Council, Brain, SAN, Total, LTE, Amyloid-related imaging abnormalities, Alector, ALS, AAIC, PROGRESS, ABC, Manuscript, Conference, Safety, Biomarker, Patent, CSF, Mutation, IND, Microglia, European Patent Convention, Insurance, PIN, FDA, Food, Risk, TREM2, Dementia, Disease, Pharmaceutical industry

Alector and GSK are co-developing AL101 for the potential treatment of more prevalent neurodegenerative diseases, including AD and Parkinson’s disease.

Key Points: 
  • Alector and GSK are co-developing AL101 for the potential treatment of more prevalent neurodegenerative diseases, including AD and Parkinson’s disease.
  • In September 2023, Alector completed enrollment of 381 participants in the randomized, double-blind, placebo-controlled, dose-ranging, INVOKE-2 Phase 2 clinical trial.
  • In July 2023, Alector presented an update on INVOKE-2 at the Alzheimer’s Association International Conference (AAIC).
  • Alector’s management team will host a conference call discussing Alector’s results for the fourth quarter and full year 2023 and provide a business update.

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

Retrieved on: 
Tuesday, February 27, 2024
MPS II, Hearing, ARIA, Antibody, LRRK2, PTV, IDS, Transferrin receptor, MAPT, Traffic barrier, ALS, Hunting, Dermatan sulfate, Therapy, BBB, SAN, Total, Amyloid-related imaging abnormalities, Alpha-synuclein, COMPASS, ETV, Hunter syndrome, Neurodegenerative disease, Conference, MPS, Safety, Biomarker, OTV, LUMA, AFL, SNCA, Amyloid beta, CSF, PIPE, Glycogen storage disease, Research, ATV, FDA, Food, Risk, Sanofi, UC, CD98, Patient, Cognition, Oligonucleotide, Science, Alfa, Pharmaceutical industry

Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.

Key Points: 
  • Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.
  • Sanofi intends to present the detailed efficacy and safety results of the ALS Phase 2 HIMALAYA study at a future scientific forum.
  • There was no collaboration revenue for the quarter ended December 31, 2023, compared to $10.3 million for the quarter ended December 31, 2022.
  • Further, for the quarter ended December 31, 2023, there was also a decrease in other unallocated research and development expenses as a result of reduced facility costs.

Denali Therapeutics Announces $500 million Private Placement Equity Financing

Retrieved on: 
Tuesday, February 27, 2024
BBB, SAN, Traffic barrier, Prospectus, Investment, Sale, Exercise, Therapy, Acceleration, PIPE, Glycogen storage disease, Research, Security (finance)

Denali anticipates the gross proceeds from the PIPE to be approximately $500 million.

Key Points: 
  • Denali anticipates the gross proceeds from the PIPE to be approximately $500 million.
  • The financing is expected to close on February 29, 2024, subject to customary closing conditions.
  • The financing was led by a U.S.-based healthcare-focused investor, with participation from global asset managers based in Boston and on the West Coast.
  • This cash estimate is a preliminary estimate and based on information currently available to management, and these estimates could change.

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

Retrieved on: 
Monday, February 26, 2024
Traffic barrier, ALS, SOD1, Neurocrine Biosciences, Therapy, Neurocrine, FXN, GBA1, AAV, Research, FA, Vaccine

The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.

Key Points: 
  • The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform.
  • Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024.
  • Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.
  • We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA.

Ashvattha Therapeutics Announces First Patient Enrolled in Expanded Phase 1/2 Study of Imaging Agent 18F-OP-801 in Additional Neurological Indications

Retrieved on: 
Thursday, February 22, 2024
Therapy, Traffic barrier, PD, ALS, Inflammation, HD, Neuroinflammation, Multiple sclerosis, Safety, Biomarker, Nanomedicine, Pharmacokinetics, AMD, Patient, Clinical trial, Disease, Microglia, DME, PET, Medical imaging

The study will evaluate the safety, pharmacokinetics and biodistribution of an intravenously administered dose of 18F-OP-801 in patients with amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), multiple sclerosis (MS) and Parkinson’s disease (PD).

Key Points: 
  • The study will evaluate the safety, pharmacokinetics and biodistribution of an intravenously administered dose of 18F-OP-801 in patients with amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), multiple sclerosis (MS) and Parkinson’s disease (PD).
  • “This study builds on preclinical data which demonstrated 18F-OP-801's ability to cross the blood-brain barrier and be taken up by activated microglia in regions of neuroinflammation in multiple disease models.
  • Additional objectives include determining the correlation between PET imaging signal, biomarkers of disease activity and clinical disease assessments.
  • The study will also assess test/retest repeatability of 18F-OP-801 uptake in a subset of subjects.

Voyager Therapeutics Reports Robust Preclinical Activity in Tau Silencing Gene Therapy Program for Alzheimer’s Disease and Advances Program into Late Research

Retrieved on: 
Tuesday, February 20, 2024
BBB, Traffic barrier, Messenger, Voyager, Conference, MicroRNA, RNA, Therapy, Pharmacokinetics, Patient, Cognition, VY, IND, AAV, DSM-IV codes, PET, Mouse, Brain, Vaccine

LEXINGTON, Mass., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced new data from its two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease. Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy program will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2024), taking place March 5-9, 2024, in Lisbon, Portugal.

Key Points: 
  • Tau Silencing Gene Therapy Program:
    Intravenous administration of BBB-penetrant AAV containing primary artificial microRNA targeting tau reduces tau broadly and robustly in hTau mouse brain.
  • Voyager’s tau silencing gene therapy program combines vectorized tau-targeted siRNA with a proprietary, blood-brain barrier (BBB)-penetrant capsid derived from the Company’s TRACER™ discovery platform.
  • Based on these data, Voyager has advanced this program into late research and expects to file an investigational new drug (IND) application in 2026.
  • Pharmacokinetics and tolerability of VY-TAU01, an anti-tau antibody for the treatment of Alzheimer’s disease, in P301S mouse and nonhuman primate.

Arvinas Announces First-in-Human Dosing of ARV-102, an Investigational PROTAC® Protein Degrader for Neurodegenerative Disease

Retrieved on: 
Tuesday, February 20, 2024
Proteolysis, Traffic barrier, Senior, DSM-IV codes, Palsy, LRRK2, ARVN, Safety, Event, Biomarker, Pharmacokinetics, Patient, CHDR, PROTAC, Pharmaceutical industry

NEW HAVEN, Conn., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, today announced that the first subject was dosed in its Phase 1 clinical trial of ARV-102, the Company’s first oral PROTAC® (PROteolysis-TArgeting Chimera) protein degrader in development to treat neurodegenerative diseases. In preclinical studies, ARV-102 has been shown to cross the blood-brain barrier and degrade leucine-rich repeat kinase 2 (LRRK2), which is a large multidomain scaffolding kinase. Increased activity and expressions of LRRK2 is genetically involved in the pathogenesis of neurological diseases including Parkinson’s disease and progressive supranuclear palsy.

Key Points: 
  • In preclinical studies, ARV-102 has been shown to cross the blood-brain barrier and degrade leucine-rich repeat kinase 2 (LRRK2), which is a large multidomain scaffolding kinase.
  • Increased activity and expressions of LRRK2 is genetically involved in the pathogenesis of neurological diseases including Parkinson’s disease and progressive supranuclear palsy.
  • In non-human primates, orally administered ARV-102 has been shown to reach deep-brain regions and degrade LRRK2 by nearly 90%.
  • The Phase 1 clinical trial of ARV-102 is enrolling healthy volunteers at the Centre for Human Drug Research (CHDR) in Leiden, the Netherlands.

City of Hope-developed Chimeric Antigen Receptor (CAR) T Cell Therapy Shows Clinical Activity in Patients With Aggressive Brain Tumors in a Phase 1 Trial

Retrieved on: 
Thursday, March 7, 2024
Oncology, Health, Surgery, Stem Cells, Neurology, Biotechnology, Glioblastoma, Bangladesh Technical Education Board, Traffic barrier, Neoplasm, Regenerative medicine, Neurosurgery, Valadez, Multimedia, CIRM, Cerebrospinal fluid, City, Food, Cancer, GBM, Radiation, National Cancer Institute, Heritage, Human, Stroke, Safety, Organization, Patient, Spinal cord, Translational Genomics Research Institute, Brain, Survival, DSM-IV codes, Research, Environment

One of the main challenges for treating brain cancer is that medications have difficulty crossing the blood-brain barrier.

Key Points: 
  • One of the main challenges for treating brain cancer is that medications have difficulty crossing the blood-brain barrier.
  • Twenty-nine of the 58 patients with recurrent high-grade glioma brain tumors, mostly glioblastoma, achieved stable disease after treatment with CAR T cells for at least two months.
  • There were two partial responses, one complete response and a second complete response after additional CAR T cell therapy cycles were delivered under compassionate use.
  • The study team also included researchers from Translational Genomics Research Institute in Phoenix, which is part of City of Hope.