Intracerebroventricular injection

CANbridge and UMass Chan Medical School Spinal Muscular Atrophy Gene Therapy (CAN203) Improves Lifespan and Motor Function in Mice When Administered via Intracerebroventricular Injection

Retrieved on: 
Thursday, May 18, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, University, Genetics, Education, Tissue, SMN, ICV, SMA, Mouse, Central nervous system, University of Massachusetts, Gene, Spinal cord, Survival, SMN1, Toxicity, Research, Doctor of Philosophy, AAV, CNS, Brain cell, Patient, Dorsal root ganglion, Cell, DRG, Intracerebroventricular injection, American Society of Gene and Cell Therapy, IND, Society, Microbiology, Vaccine

The benchmark vector is identical in design to the only approved gene therapy for SMA.

Key Points: 
  • The benchmark vector is identical in design to the only approved gene therapy for SMA.
  • Furthermore, the second-generation gene therapy resulted in higher levels of SMN protein in the central nervous system (CNS), particularly within motor neurons, and lower expression in the peripheral tissues, compared to benchmark vector-treated mice.
  • Superior transgene expression was observed in disease-vulnerable cells, including ChAT-positive motor neurons in the spinal cord.
  • No evidence of DRG toxicity associated with superior SMN expression was observed at 90 days after ICV injection with the high dose of the second-generation gene therapy.

Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome

Retrieved on: 
Monday, January 23, 2023
Biotechnology, FDA, People with Disabilities, Health, General Health, Women, Research, Genetics, Consumer, Science, Clinical Trials, Toxicity, Organization, Caregiver, AAV, Pediatrics, University, IRSF, Mouse, Trial of the century, Hope, Assistant, MECP2, Safety, Intracerebroventricular injection, Disorder, Patient, Baylor University, Clinical trial, Food, Neurology, Rett syndrome, DSM-IV codes, Male, Female, Gene expression, Survival, RSRT, Construct, Disease, Inc., Baylor College of Medicine, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ICV, IND, Pharmaceutical industry, Vaccine, Rett

NGN-401 is the first investigational adeno-associated virus (AAV) gene therapy candidate to be administered to pediatric patients using Neurogene’s proprietary Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Key Points: 
  • NGN-401 is the first investigational adeno-associated virus (AAV) gene therapy candidate to be administered to pediatric patients using Neurogene’s proprietary Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.
  • Embedding EXACT technology into NGN-401 is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that safely regulates MECP2 transgene expression without causing toxic effects associated with overexpression.
  • Importantly, expression data for NGN-401 demonstrated well-controlled MeCP2 protein levels in key brain regions affected by Rett syndrome, while conventional gene therapy, without EXACT regulation, generated more variable and undesirable higher MeCP2 levels.
  • IND clearance enables Neurogene to initiate a Phase 1/2 trial to assess the safety, tolerability and efficacy of NGN-401 in female pediatric patients with Rett syndrome.

Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs & Allied Diseases Association Conference

Retrieved on: 
Friday, July 8, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Cell, FIH, Inc., Mutation, Science, Intracerebroventricular injection, Boonshoft School of Medicine, Patient, ICV, Magnetic resonance imaging, Brain, Trial of the century, Walking, Metabolism, Disease, Intellectual property, White matter, Irritability, Forward-looking statement, Spasticity, Magnetic resonance, ASPA, Gross, Canavan disease, Kaay Raav Tumhi, MPH, Pfizer, N-Acetylaspartic acid, Clinical trial, Wright, OH, Doctor of Philosophy, Seizure, Pediatrics, CD, Time, Associate, Safety, MRI, VG, Program, NAA, Neurodegeneration, Conference, Pharmaceutical industry, Medical imaging, Canavan, Myrtelle Canavan, Aspartoacylase, MD

In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA).

Key Points: 
  • In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA).
  • These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.
  • Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases.
  • The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases.

Considerations for Neuroscience Trials with Direct Administration to the CNS, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Tuesday, June 14, 2022
Neuroscience, Clinical trial, Food, Traffic barrier, Doctor of Philosophy, Central nervous system, Method, Medical director, Intracerebroventricular injection, MD, Industry, History, CNS, United States Army Medical Department Center and School, Medical device, Medicine, Medpace

TORONTO, June 14, 2022 /PRNewswire-PRWeb/ -- In this free webinar, learn about the history of direct central nervous system (CNS) administration leading to modern day applications. Attendees will learn about the unique challenges from the investigative site perspective with strategies to mitigate them. The featured speakers will also share key operational and regulatory considerations for neuroscience trials with direct CNS administration.

Key Points: 
  • The featured speakers will also share key operational and regulatory considerations for neuroscience trials with direct CNS administration.
  • The featured speakers will also share key operational and regulatory considerations for neuroscience trials with direct CNS administration.
  • The speakers will also share key operational and regulatory considerations for neuroscience trials with direct CNS administration.
  • For more information, or to register for this event, visit Considerations for Neuroscience Trials with Direct Administration to the CNS.

Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease

Retrieved on: 
Tuesday, February 15, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Chromosome 11, Patient, ASPA, Biomarker, NAA, Neurodegeneration, Brain, VG, White matter, CD, Clinical trial, N-Acetylaspartic acid, Neurosurgery, Safety, Data monitoring committee, FIH, Aspartoacylase, Myrtelle Canavan, Pfizer, Boonshoft School of Medicine, Inc., Cell, Rita Raave, MD, ICV, Mutation, Wright, Metabolism, Intracerebroventricular injection, OH, Assistant, Canavan disease, Pharmaceutical industry, Medicine, Neuroscience

In Canavan disease (CD), the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) responsible for coding the enzyme Aspartoacylase (ASPA).

Key Points: 
  • In Canavan disease (CD), the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) responsible for coding the enzyme Aspartoacylase (ASPA).
  • Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.
  • We are encouraged by the initial findings in the 3 patients treated thus far in this landmark gene therapy trial in Canavan disease.
  • The ongoing clinical trial is expected to continue to generate the critical data needed for further development of this novel gene therapy in the patients with Canavan disease, said Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle.