Traffic barrier

Cellectar Biosciences Announces Enrollment of the First Patient in Pediatric High-Grade Gliomas Phase 1b Clinical Study

Retrieved on: 
Tuesday, January 30, 2024
Traffic barrier, Survival, NCI, Fast Track, Patient, Safety, Central nervous system, Glomus tumor, MOA, CNS, Bone marrow, Hope, SBIR, Ependymoma, Cancer, Pharmaceutical industry

“Treatment of pediatric patients with relapsed and refractory high-grade glioma and ependymoma is extremely challenging.

Key Points: 
  • “Treatment of pediatric patients with relapsed and refractory high-grade glioma and ependymoma is extremely challenging.
  • There are limited treatment options and high unmet clinical need for patients suffering from this aggressive cancer,” said Dr. Sameer Farouk Sait, pediatric hematologist-oncologist and assistant attending at Memorial Sloan Kettering Cancer Center.
  • “We understand the profound impact pediatric high-grade gliomas have on the lives of young patients and their families,” commented James Caruso, president and CEO of Cellectar.
  • We are hopeful its novel MOA will provide a meaningful treatment option for these young patients.”

Vect-Horus Enters Exclusive License Agreement with Ionis Pharmaceuticals to Advance Systemic Delivery of RNA-Targeted Medicines for Neurological Diseases

Retrieved on: 
Tuesday, January 30, 2024
Mental Health, Research, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Oncology, Traffic barrier, Ionis Pharmaceuticals, RNA, Therapy, Patient, Central nervous system, DSM-IV codes, IONS, Ionis, Brain, Medication, Tissue, Vaccine

The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.

Key Points: 
  • The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.
  • Ionis has an option to add additional targets to the license.
  • Vect-Horus retains the rights to use its technology, for these specific targets, for all non-RNA-Targeted Medicines.
  • “This license is aligned with our strategy of using our technology to deliver drug payloads, such as RNA-targeted therapeutics, to the brain and other tissues.

EQS-News: Kizoo leads financing round in Reservoir Neuroscience to restore health to the aging brain

Retrieved on: 
Tuesday, January 30, 2024
Notifiable disease, Patient, Vascular disease, Research, Health, Partner, UC, Reservoir, University of California, Berkeley, Traffic barrier, Myenteric plexus, Doctor of Philosophy, Blood, Brain, Ageing, Neurodegenerative disease, Medical imaging

San Francisco, CA, U.S. / Berlin, Germany – January 18, 2024 – Reservoir Neuroscience announced today the completion of a $4M financing round to support the development of new therapies for age-related neurodegeneration.

Key Points: 
  • San Francisco, CA, U.S. / Berlin, Germany – January 18, 2024 – Reservoir Neuroscience announced today the completion of a $4M financing round to support the development of new therapies for age-related neurodegeneration.
  • Reservoir will use the funds to develop its novel class of drug compounds designed to restore health to the brain’s blood vessels to rejuvenate the aging brain.
  • The company aims to develop the first drug that specifically targets vascular disease as a new way to restore brain health during natural aging and in age-related diseases.
  • Reservoir's therapeutic strategy seeks to restore health to blood vessels to reverse degenerative damage that spreads from the blood vessels to the brain.

Inhibikase Therapeutics Announces Publication Highlighting Results from its Phase 1 Studies with Risvodetinib

Retrieved on: 
Monday, January 29, 2024
Ageing, Patient, Therapy, Cmax, CSF, Dark Ages, Parkinson's disease, PD, SAD, Journal, Safety, Central nervous system, Traffic barrier, DOI, Cerebrospinal fluid, Brain, Pharmacokinetics, Pharmaceutical industry

“Parkinson’s disease remains one of the most prevalent neurodegenerative diseases worldwide, affecting more than a million people in the U.S. alone.

Key Points: 
  • “Parkinson’s disease remains one of the most prevalent neurodegenerative diseases worldwide, affecting more than a million people in the U.S. alone.
  • “Our publication in the Journal of Parkinson’s Disease highlights our early clinical work with risvo, our lead selective inhibitor of c-Abl.
  • In Phase 1 studies, risvo was shown to have a favorable safety profile that was well tolerated by older or elderly healthy subjects and by patients with Parkinson’s disease.
  • Inhibikase continues to actively enroll patients in its Phase 2 201 Trial, evaluating risvo in untreated Parkinson’s disease.

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

Retrieved on: 
Thursday, January 25, 2024
Death, Pharmaceutical policy, Patient, MHRA, Therapy, Busulfan, Health care, Civil service commission, European Commission, Health, Mutation, Food, MLD, Central nervous system, Traffic barrier, ARSA, FDA, Priority review, Genome, HSC, Nerve, PDUFA, Orchard, Pharmaceutical industry

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • 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.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.

Cellectar Biosciences Reports Complete Central Nervous System (CNS) Clearance in Relapsed/Refractory Waldenstrom’s Macroglobulinemia Patient

Retrieved on: 
Monday, January 22, 2024
WM, Patient, Syndrome, BNS, Harvard Medical School, Dana–Farber Cancer Institute, Cancer, NCI, Mutation, Traffic barrier, Malignancy, MYD88, Cerebrospinal fluid, CNSL, CNS, Pharmaceutical industry

“Attainment of complete CNS clearance in a patient with Bing-Neel Syndrome after previous exposure to BTKi therapy is encouraging,” commented Jorge Castillo, M.D., associate professor at Harvard Medical School and director of Bing Center Clinic at Dana Farber Cancer Institute.

Key Points: 
  • “Attainment of complete CNS clearance in a patient with Bing-Neel Syndrome after previous exposure to BTKi therapy is encouraging,” commented Jorge Castillo, M.D., associate professor at Harvard Medical School and director of Bing Center Clinic at Dana Farber Cancer Institute.
  • Cerebrospinal fluid flow cytometry showed no evidence of clonal B-cells, and molecular analysis did not detect the MYD88 mutation.
  • This very encouraging patient response further supports iopofosine’s development in CNS-related malignancies, including CNS lymphoma and pediatric high-grade gliomas.”
    Cellectar previously announced a complete response in a patient with relapsed/refractory primary central nervous system lymphoma (CNSL) as part of its ongoing Phase 2a trial of iopofosine I 131.
  • The study objective is to identify the recommended iopofosine I 131 Phase 2 dose in pHGG patients.

Kizoo leads financing round in Reservoir Neuroscience to restore health to the aging brain

Retrieved on: 
Thursday, January 18, 2024
Notifiable disease, Patient, Vascular disease, Research, Health, Partner, Reservoir, SAN, UC, University of California, Berkeley, Traffic barrier, Myenteric plexus, Doctor of Philosophy, Blood, Brain, Ageing, Neurodegenerative disease, Medical imaging

Reservoir will use the funds to develop its novel class of drug compounds designed to restore health to the brain’s blood vessels to rejuvenate the aging brain.

Key Points: 
  • Reservoir will use the funds to develop its novel class of drug compounds designed to restore health to the brain’s blood vessels to rejuvenate the aging brain.
  • Reservoir’s unique approach to neurodegeneration was developed by co-founders Aaron Friedman and Vlad Senatorov, PhD neuroscientists from UC Berkeley who focus on understanding how aging blood vessels effect brain health.
  • The company aims to develop the first drug that specifically targets vascular disease as a new way to restore brain health during natural aging and in age-related diseases.
  • Reservoir's therapeutic strategy seeks to restore health to blood vessels to reverse degenerative damage that spreads from the blood vessels to the brain.

Lisata Therapeutics Announces First Patient Treated in the Phase 2a Trial of LSTA1 in Patients with Glioblastoma Multiforme

Retrieved on: 
Wednesday, January 17, 2024
Patient, Permeability, Therapy, Glioblastoma, SOC, Standard of care, Tartu University Clinic, GBM, Traffic barrier, Doctor of Philosophy, Temozolomide, Glioma

The trial is an investigator-initiated study by Lenne-Triin Kõrgvee, MD, PhD, principal investigator of the study.

Key Points: 
  • The trial is an investigator-initiated study by Lenne-Triin Kõrgvee, MD, PhD, principal investigator of the study.
  • The study is a Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating LSTA1 when added to standard of care (“SoC”), temozolomide, versus SoC and placebo in subjects with newly diagnosed GBM.
  • “We are very pleased to announce the first patient treated in this Phase 2a study evaluating LSTA1 in patients with newly diagnosed GBM, a very aggressive brain tumor that is often fatal.
  • “GBM has, historically, been very difficult to successfully treat and we believe LSTA1 may be an important tool in improving those outcomes for patients.”

JCR Pharmaceuticals to Present at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Thursday, January 25, 2024
Health, Other Science, General Health, Research, Science, Pharmaceutical, Biotechnology, Hurler, Patient, Enzyme replacement therapy, PST, Hunter syndrome, MPS II, JCR, Eto, BBB, MPS, Medicine, MHLW, Ikeda, ERT, Syndrome, Safety, Traffic barrier, Glycogen storage disease, Scheie syndrome, Ministry, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.
  • In addition to posters being available for each of the presentations, two of them will be shared as oral presentations given by a clinical investigator and a researcher from JCR Pharmaceuticals.
  • A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Ikeda, et al.)
  • WORLDSymposium™ attendees who would like to receive more information about JCR Pharmaceuticals can visit JCR’s on-site conference booth (#506) or visit its virtual booth on the WORLDSymposium™ conference website.

Myrobalan Therapeutics Completes Series A Financing of $24 Million to Develop Potentially First-in-Class CNS Therapeutics with Restorative Potential

Retrieved on: 
Wednesday, January 10, 2024
Health, Neurology, Research, Pharmaceutical, Science, Biotechnology, Constellation, Patient, Therapy, Biology, Remyelination, Drug design, ALS, Broad Institute, Multiple sclerosis, Partnership, Harvard Medical School, National academy, GPR17, Venture, Neuroinflammation, MIT, Sympathetic nervous system, Drug development, TYK2, Traffic barrier, Harvard University, Capital, Limited partnership, CNS, Pharmaceutical industry

Myrobalan has the potential to overcome such obstacles with the development of highly selective, brain-penetrant, orally available compounds against promising novel targets involved in demyelination and neuroinflammation.

Key Points: 
  • Myrobalan has the potential to overcome such obstacles with the development of highly selective, brain-penetrant, orally available compounds against promising novel targets involved in demyelination and neuroinflammation.
  • These currently untreatable disease mechanisms have been linked to multiple sclerosis (MS), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), and several rare neurological conditions.
  • “Our deep understanding of remyelination and neuroinflammation has broad application in high-unmet need CNS diseases,” said Dr. Jing Wang, CEO & Co-Founder, Myrobalan Therapeutics.
  • Myrobalan Therapeutics' unique strategy holds great promise in overcoming challenges associated with central nervous system diseases.