Myrtelle Canavan

Myrtelle to Present Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay-Sachs and Allied Diseases Annual Family Conference

Retrieved on: 
Wednesday, May 31, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Other Health, CD, Gross, Canavan disease, Inc., Drug development, Natural history, IV, Clinical trial, Mutation, Human, Brain, ASPA, AAV, Doctor of Philosophy, Language, NAA, System, MRI, MRS, Magnetic resonance imaging, Metabolism, MD, Patient, Preyasi Raave, White matter, CSF, FIH, Conference, Caregiver, Vaccine, Medical imaging, Myrtelle Canavan

Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.

Key Points: 
  • Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • “Connecting with patients and caregivers allows us to incorporate the patient voice into our drug development activities.”

Myrtelle to Host Symposium on Gene Therapies Targeting Oligodendrocytes and Implications in Brain Function and Disease States at the American Society of Gene and Cell Therapy 26th Annual Meeting in Los Angeles on Friday, May 19, 2023

Retrieved on: 
Wednesday, May 10, 2023
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, CD, Learning, Neuron, Canavan disease, Inc., University, Gene, Senior, Boonshoft School of Medicine, Clinical trial, Neuroscience, Brain, Cell, Multiple sclerosis, DSM-IV codes, Associate, Assistant, Cerebral cortex, Chromosome 11, Wright, Rita Raave, Leukodystrophy, Neurology, FIH, Patient, Society, Vaccine, Canavan, Myrtelle Canavan, Medicine

The symposium will highlight historical and emerging views of oligodendrocytes in normal brain function and a growing array of disease states, including leukodystrophies and disorders of myelin production such as multiple sclerosis.

Key Points: 
  • The symposium will highlight historical and emerging views of oligodendrocytes in normal brain function and a growing array of disease states, including leukodystrophies and disorders of myelin production such as multiple sclerosis.
  • Oligodendrocytes perform numerous key functions in the brain - including the production of myelin, the insulating material that enables proper neuronal function – and are now appreciated as having roles in a range of diseases.
  • Myrtelle is developing a unique toolkit, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells.
  • “Given the intractable nature of many neurological diseases, these cells represent important brain components worthy of interrogation in the quest for novel interventions, including gene therapies.

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Innovative Licensing and Access Pathway Designation from the UK Medicines and Healthcare Products Regulatory Agency

Retrieved on: 
Tuesday, April 25, 2023
Biotechnology, Genetics, Health, Neurology, CD, Neuron, Canavan disease, Inc., Senior, Classification, European Medicines Agency, N-Acetylaspartic acid, Mutation, Medication, Pfizer, Fast Track, Brain, ASPA, Cell, Medicines and Healthcare products Regulatory Agency, Doctor of Philosophy, Rita Raave, NAA, ATMP, US, Orphan drug, Genetic disorder, Metabolism, Orphan, Vivisection, United Kingdom, White matter, MAA, MHRA, Pharmaceutical industry, Aspartoacylase, Myrtelle Canavan

Myrtelle Inc. , (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovative Licensing and Access Pathway (ILAP) designation to the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease.

Key Points: 
  • Myrtelle Inc. , (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovative Licensing and Access Pathway (ILAP) designation to the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease.
  • ILAP provides expedited access to a range of UK development services and tools for life-threatening or seriously debilitating conditions for which there is a significant patient need.
  • These services include the potential for frequent MHRA interactions, accelerated Marketing Authorization Application (MAA) assessment, innovative and flexible licensing routes, engagement on market access activities, and a continuous benefit-risk assessment integrating real world evidence.
  • 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.

Myrtelle and rAAVen Therapeutics to Develop Novel Gene Therapy Vectors

Retrieved on: 
Wednesday, January 11, 2023
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Inc., Myrtelle Canavan, Therapy, AAV, CNS, Rita Raave, Partnership, Nervous system, Central nervous system, Neuroscience, Canavan disease, Neuron, Research, Disorder, Doctor of Philosophy, Secondary research, Neurodegeneration, Vaccine, Pharmacology

Myrtelle Inc. , (“Myrtelle”) a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and rAAVen Therapeutics , (“rAAVen”) an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, today announced a partnership to develop novel recombinant adeno-associated virus (“rAAV”) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected.

Key Points: 
  • Myrtelle Inc. , (“Myrtelle”) a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, and rAAVen Therapeutics , (“rAAVen”) an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, today announced a partnership to develop novel recombinant adeno-associated virus (“rAAV”) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected.
  • When myelin is damaged or does not develop normally, these impulses slow down and affect normal brain function.
  • Myrtelle will test the vectors in a range of myelin-based disorders and, if successful, pursue further development.
  • “We appreciate the opportunity to help Myrtelle achieve their goals for their novel gene therapy programs.

Myrtelle Announces Positive Interim Data in Phase 1/2 Clinical Trial of Its Proprietary Investigational Gene Therapy rAAV-Olig001-ASPA in Canavan Disease

Retrieved on: 
Monday, January 9, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, White matter, CSF, Clinical trial, MRI, Associate, Gross, N-Acetylaspartic acid, Food, Cerebrospinal fluid, Wright, European Medicines Agency, MD, Boonshoft School of Medicine, Inc., ASPA, CD, Metabolism, FIH, NAA, Patient, Myrtelle Canavan, Pediatrics, Classification, US Foods, Mutation, Canavan disease, Magnetic resonance imaging, Rita Raave, Orphan drug, Hospital, Fast Track, Neurodegeneration, Pharmaceutical industry, Medical imaging

Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.

Key Points: 
  • Early data from the study, being conducted at Dayton Children’s Hospital (Dayton, Ohio), at three months following gene therapy treatment have shown encouraging results to date.
  • The observed improvements following gene therapy are in contrast to the continuous clinical decline expected for untreated CD patients.
  • The first three patients treated with the gene therapy in cohort one are now at least 18 months post therapy.
  • “The early results to date in patients treated in Myrtelle’s Phase 1/2 clinical trial are encouraging.

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Orphan Drug Designation from the European Medicines Agency

Retrieved on: 
Wednesday, December 14, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Fast Track, CD, Re Recher's Will Trusts, OD, Neurodegeneration, Rita Raave, COMP, Myrtelle Canavan, Irritability, Spasticity, ATMP, Walking, Intellectual property, Doctor of Philosophy, Royal commission, Metabolism, EMA, Patient, Orphan drug, ASPA, N-Acetylaspartic acid, Inc., FDA, Canavan disease, European Commission, European Medicines Agency, Cell, Senior, Plasma protein binding, Safety, Brain, NAA, Mutation, Seizure, Pharmaceutical industry, Aspartoacylase, EU

Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug (OD) designation for rAAV-Olig001-ASPA, the companys lead gene therapy product candidate for the treatment of Canavan disease.

Key Points: 
  • Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug (OD) designation for rAAV-Olig001-ASPA, the companys lead gene therapy product candidate for the treatment of Canavan disease.
  • The OD application is examined by the EMAs Committee for Orphan Medicinal Products (COMP) who provides their review to the European Commission for designation approval.
  • In CD, the production of myelin is impaired due to a mutation in the Aspartoacylase gene (ASPA) that encodes the enzyme Aspartoacylase (ASPA).
  • In addition to EMAs orphan designation, rAAV-Olig001-ASPA also received EMAs Advanced Therapy Medicinal Product Classification (ATMP) and US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA.

Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

Retrieved on: 
Tuesday, October 4, 2022
Health, Genetics, Clinical Trials, Research, Science, Biotechnology, Classification, Time, Canavan disease, Association, Walking, ATMP, Clinical trial, Magnetic resonance, Spasticity, Gross, Inc., FDA, Brain, Patient, MRI, MPH, Pfizer, N-Acetylaspartic acid, Mutation, FIH, Irritability, CA, CD, Safety, Kaay Raav Tumhi, Regenerative medicine, Mesa, ASPA, Magnetic resonance imaging, Gene, European Medicines Agency, Fast Track, Forward-looking statement, NAA, Neurodegeneration, Metabolism, Seizure, EMA, Intellectual property, Company, Pharmaceutical industry, Medical imaging, MD, Aspartoacylase, Myrtelle Canavan

Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced the completion of dosing of 8 patients with the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD).

Key Points: 
  • Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced the completion of dosing of 8 patients with the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD).
  • The oligodendrocyte targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and myelination in patients with CD.
  • Additionally, observations of these patients at 6 months post-treatment using Magnetic Resonance Imaging (MRI) demonstrated increases in brain white matter and myelin content.
  • Observed improvements in these treated patients are in contrast to the continuous clinical deterioration expected with the natural progression of CD.

Myrtelle and Forge Biologics Announce Viral Vector and Plasmid DNA cGMP Manufacturing Partnership

Retrieved on: 
Monday, October 3, 2022
Health, Biometrics, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Ignition, CNS, Forge, GMP, Technology, AAV, CEO, Multimedia, Inc., CGMP, RHI, Pfizer, Ear, Partnership, Rita Raave, Program, Patient, Hearing, Neurodegeneration, Intellectual property, HEK 293, Pharmaceutical industry, Vaccine, TMPRSS3, Hearth, Myrtelle Canavan

View the full release here: https://www.businesswire.com/news/home/20221003005359/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20221003005359/en/
    Forge will provide research-grade and GMP-Pathway plasmid manufacturing services as well as cGMP adeno-associated viral (AAV) process development and scale-up manufacturing services for Myrtelles program, Myr-201.
  • The program will utilize Forges platform process including its proprietary HEK 293 suspension Ignition Cells and pEMBR adenovirus helper plasmid.
  • All development and cGMP manufacturing activities will occur at the Hearth, Forges 200,000 square foot gene therapy cGMP facility in Columbus, Ohio.
  • The Hearth is a custom-designed cGMP facility focused on AAV manufacturing and can host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European Medicines Agency

Retrieved on: 
Wednesday, September 7, 2022
Health, Genetics, Other Science, Clinical Trials, Research, Science, Biotechnology, European Medicines Agency, N-Acetylaspartic acid, EMA, Safety, Gene, Neuron, Walking, Scientific Advice Mechanism, Myrtelle Canavan, CD, Seizure, Brain, Doctor of Philosophy, NAA, Pfizer, Time, Genetic disorder, Senior, Forward-looking statement, Canavan disease, Cell, Spasticity, Fast Track, Classification, Inc., Re Recher's Will Trusts, Committee, FDA, Company, Neurodegeneration, Compliance, Irritability, Centralisation, ATMP, ASPA, White matter, Mutation, Intellectual property, CAT, Metabolism, Kaay Raav Tumhi, Patient, Pharmaceutical industry, EU, Aspartoacylase

Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP).

Key Points: 
  • Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP).
  • The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA).
  • "The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy.
  • More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing [email protected] .

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.