Rita Raave

Voyager Therapeutics Presents Robust, Multi-Species Results from Preclinical Studies of IV-Delivered, TRACER™-Generated Novel Capsids at the ASGCT 26th Annual Meeting

Retrieved on: 
Thursday, May 18, 2023
Infection, Neuron, DNA, GBA1, Human, Central nervous system, Capsid, Spinal cord, TRACER, Traffic barrier, Brain, Research, Gene expression, Tropism, RNA, Antibody, AAV, CNS, Astrocyte, ESGCT, BBB, Transgene, Rita Raave, DRG, Mouse, Liver, Sf9 (cells), Disability studies, Therapy, Glucocerebrosidase, Neurology, Oligonucleotide, Family, Society, Vaccine, Substantia nigra, Putamen

These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023, in Los Angeles.

Key Points: 
  • These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023, in Los Angeles.
  • “These data demonstrate that our TRACER capsid discovery platform is able to generate brain-penetrant capsids in a highly reproducible manner,” said Todd Carter, Chief Scientific Officer of Voyager Therapeutics.
  • In these studies, VCAP-102 demonstrated de-targeting of the dorsal root ganglia (DRG) and liver.
  • Researchers identified a new capsid called VCAP-103, which demonstrated enhanced brain and spinal cord transduction in marmosets and African green monkeys.

NewBiologix Launches with $50M to Advance Novel Technology Platform for the Manufacturing of Gene and Cell Therapies

Retrieved on: 
Thursday, May 11, 2023
Research, Engineering, Genetics, Health Technology, Manufacturing, Stem Cells, Biotechnology, Health, Science, Immortalised cell line, NGS, CHO, Coronavirus Scientific Advisory Board, ATMP, COGS, Multimedia, Doctor of Philosophy, FRS, Rita Raave, Cell, B cell, CHF, Genome, Vaccine, MD

NewBiologix SA emerged from stealth today as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies.

Key Points: 
  • NewBiologix SA emerged from stealth today as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies.
  • The Company’s DNA-based platform is being developed to address key manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to produce advanced therapy medicinal products (ATMPs).
  • NewBiologix’s $50 million (CHF 45 million) Series A was led by Recipharm A/S with additional funding from the Company’s co-founders, Igor Fish and Nicolas Mermod.
  • “Current gene and cell therapies primarily focus on rare diseases.

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.

Agathos Biologics to present data on cell lines for viral vector production

Retrieved on: 
Wednesday, May 3, 2023
Research, Genetics, Clinical Trials, Health Technology, Other Health, Biotechnology, Radiology, Health, Science, Other Science, Immortalised cell line, Gene, Multimedia, Sale, HeLa, Patient, Rita Raave, Cell, Society, Vaccine, Pharmaceutical industry

The data show rAAV production with this cell line, Agathos BHK-[wt E1], using a standard triple transfection method.

Key Points: 
  • The data show rAAV production with this cell line, Agathos BHK-[wt E1], using a standard triple transfection method.
  • The BHK-[wt E1] data will be presented in a poster titled “E1-transformed BHK Cell Lines for Helper Virus-Free AAV Production Using Triple Transfection”, number 1221 , and the GH329 data will be presented in a poster titled “Use of the GH329 Cell Line for Adeno-Associated Virus Production”, number 397 .
  • (Photo: Business Wire)
    “We are excited to present our data that show rAAV production in Agathos BHK-[wt E1] and GH329,” said James Brown, CEO and co-founder of Agathos Biologics.
  • Agathos Biologics is a biotechnology company pursuing transformational science in biomanufacturing, biologic payload delivery, and cell and gene therapy.

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.

Avirmax CMC Inc. Launches AAV Production with Sf Rhabdovirus-free Cell Lines (Sf-RVF™)

Retrieved on: 
Thursday, March 23, 2023
Immortalised cell line, Vaccine, Sf9, SF, Research, RNA, CDMO, Rita Raave, ACI, A-DNA, DVM

However, Sf9 cell lines persistently carries Sf rhabdovirus (Sf-RV), an enveloped, negative sense and single stranded RNA (-ssRNA) virus.

Key Points: 
  • However, Sf9 cell lines persistently carries Sf rhabdovirus (Sf-RV), an enveloped, negative sense and single stranded RNA (-ssRNA) virus.
  • When using serum-free suspension culture, Avirmax CMC's Sf-RVF™ cell lines have been shown to be comparable with respect to rAAV yields and quality, relative to rAAV produced using Sf-RV parental cell lines.
  • Avirmax CMC Inc. is now using the Sf-RVF™ cell lines in rAAV manufacturing for research and clinical development purposes.
  • Avirmax CMC Inc. is also expanding the Sf-RVF™ cell lines to the production of other modalities including vaccines and protein drugs.

rAAV Manufacturing -- Enzymatic DNA for Achieving a Robust & High-Quality DNA Supply, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Thursday, March 16, 2023
Quality control, DNA, ITRS, AAV, TAAV, Disease, Transfection, Rita Raave, R, Vaccine

TORONTO, March 16, 2023 /PRNewswire-PRWeb/ -- The gene therapy clinical pipeline continues to be in a growth phase, with over 2,000 therapies in development globally, with recombinant adeno-associated viral vectors (rAAV) as one of the most powerful tools for gene delivery to treat human disease, leading the way.

Key Points: 
  • In this free webinar, learn about achieving a robust, scalable and high-quality DNA supply in recombinant adeno-associated viral vector (rAAV) gene therapy manufacturing.
  • Attendees will learn about a synthetically manufactured DNA to produce double stranded linear molecules with covalently closed ends.
  • Attendees will learn about the use of this enzymatic DNA technology in Phase I/II.
  • Join the featured experts to learn more about achieving a robust, scalable and high-quality DNA supply in recombinant adeno-associated viral vector gene therapy manufacturing.

Avirmax Biopharma Inc. Will Present at Glaucoma 360 New Horizons Forum 2023

Retrieved on: 
Friday, February 3, 2023
Rat, Detection, DARC, Excitotoxicity, Mouse, Therapy, Glaucoma, Westin St. Francis, Rita Raave, ABI, NMDA, Patient, Chief scientific officer, CMC, Research, IOP, Death, FDA, RGC, Paratriathlon, Pont, Pharmaceutical industry

HAYWARD, Calif., Feb. 3, 2023 /PRNewswire/ -- Avirmax Biopharma Inc. (ABI), Chief Executive and Scientific Officer, Shengjiang Shawn Liu will present the Avirmax BioPharma Inc (ABI)'s progress in the innovation and development of genetic medicines for glaucoma treatment at Glaucoma 360 New Horizons Forum at The Westin St. Francis in San Francisco, Feb 03, 2023.

Key Points: 
  • HAYWARD, Calif., Feb. 3, 2023 /PRNewswire/ -- Avirmax Biopharma Inc. (ABI), Chief Executive and Scientific Officer, Shengjiang Shawn Liu will present the Avirmax BioPharma Inc (ABI)'s progress in the innovation and development of genetic medicines for glaucoma treatment at Glaucoma 360 New Horizons Forum at The Westin St. Francis in San Francisco, Feb 03, 2023.
  • Since its founding, Avirmax Biopharma Inc (ABI) has been focusing on developing an effective gene therapy product, ABI-902 for glaucoma treatment.
  • ABI-902 is manufactured by Avirmax CMC Inc.
    Now in its 12th year, the Glaucoma 360 New Horizons Forum unites key clinical, industrial, financial, and FDA leaders in a unique exchange on research innovation and advances in glaucoma treatment.
  • Avirmax Biopharma Inc. (ABI) is a subsidiary of Avirmax Inc., a San Francisco Bay Area based company working on genetic medicine.

Exegenesis Bio Announces FDA Clearance of Investigational New Drug (IND) Application for EXG102-031; A Novel Gene Therapy for the Treatment of neovascular Age-Related Macular Degeneration (nAMD)

Retrieved on: 
Wednesday, January 18, 2023
Health, FDA, Hospitals, Genetics, Clinical Trials, Pharmaceutical, Optical, IND, Severe cognitive impairment, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Macular degeneration, Food, Patient, NAMD, Rita Raave, CMC, Pharmaceutical industry

Exegenesis Bio, a rapidly growing global gene therapy company is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for EXG102-031, a recombinant adeno-associated virus (rAAV) based gene therapy that is being studied for the treatment of neovascular Age Related Macular Degeneration (nAMD), the leading cause of severe vision loss and irreversible blindness worldwide.

Key Points: 
  • Exegenesis Bio, a rapidly growing global gene therapy company is pleased to announce that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for EXG102-031, a recombinant adeno-associated virus (rAAV) based gene therapy that is being studied for the treatment of neovascular Age Related Macular Degeneration (nAMD), the leading cause of severe vision loss and irreversible blindness worldwide.
  • “We are excited by the progress that our company has made and pleased to have reached this critical milestone; this is our second IND approval and the first in North America since the inception of Exegenesis Bio 3 years ago,” stated Zhenhua Wu, CEO of Exegenesis Bio.
  • “This is a strong validation of the world-class R&D, CMC, quality and regulatory capabilities that we have built.
  • We look forward to accelerating development of our innovative gene therapy pipeline in areas with high unmet medical needs and bringing these innovative treatments to patients worldwide.”

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.