Aspartoacylase

Myrtelle Announces Presentation of Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the American Society of Gene and Cell Therapy 26th Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023
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Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.

Key Points: 
  • Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.
  • Improvements in multiple anatomic and biomarker measurements by magnetic resonance imaging (MRI) and spectroscopy (MRS) have also been observed.
  • 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.

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’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.

BridgeBio Pharma Presents Updated Positive Data from its BBP-812 Canavan Disease Gene Therapy Program at the 51st Annual Meeting of the Child Neurology Society

Retrieved on: 
Thursday, October 13, 2022
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PALO ALTO, Calif., Oct. 13, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today presented promising pharmacodynamic, tolerability and preliminary functional efficacy data from the first three participants dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease. An update on the CANinform natural history study, which continues to enroll new participants globally, was also presented. Canavan disease is an ultra-rare and fatal disease with no approved therapies.   New data from the third CANaspire participant are consistent with the first two treated children:

Key Points: 
  • BBP-812 is an investigational AAV9 gene therapy for Canavan disease.
  • Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source.
  • Preclinical proof-of-concept results have shown the approach restores survival and normal motor function in Canavan disease models.
  • Affecting approximately 1,000 children in the United States and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy.

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
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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’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
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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.

Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease

Retrieved on: 
Tuesday, March 15, 2022
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Myrtelle Inc. (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for the treatment of patients with Canavan Disease (CD).

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 U.S. Food and Drug Administration (FDA) has granted Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations for its lead clinical-stage gene therapy, rAAV-Olig001-ASPA, for the treatment of patients with Canavan Disease (CD).
  • The oligodendrocyte-targeted gene therapy is intended to restore ASPA function, enable metabolism of the abundant brain chemical N-Acetylaspartate (NAA), and support myelination.
  • 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.
  • Forward-looking statements are based upon current estimates and assumptions and include statements regarding the company having the ability to benefit from Fast Track, Rare Pediatric Disease (RPD), and Orphan Drug designations.

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.

Myrtelle Announces Successful Completion of Initial Stage of Phase 1/2 Clinical Trial of Proprietary Gene Therapy for Canavan Disease and Expands Treatment to Younger Patients

Retrieved on: 
Tuesday, January 11, 2022
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We are pleased with the initial clinical data obtained in the First-in-Human trial of our novel, targeted gene therapy for Canavan disease, said Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle.

Key Points: 
  • We are pleased with the initial clinical data obtained in the First-in-Human trial of our novel, targeted gene therapy for Canavan disease, said Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle.
  • Encouraged by our initial findings and the favorable decision by the Data Monitoring Committee, we are excited to embark on the next stages of clinical development and the treatment of younger patients.
  • Myrtelles trial utilizes a novel proprietary recombinant adeno-associated virus (rAAV) vector that for the first time directly targets oligodendrocytes in the brain.
  • The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurological diseases.