AAV

REGENXBIO Highlights AAV Pipeline with Interim Results from Retinal and Duchenne Programs at its Virtual Investor Day on July 11, 2023

Retrieved on: 
Tuesday, July 11, 2023
Interim, Myopathy, AMD, Cohort, ABBV, Eye, Quality of life, American Academy, DRSS, NAV, American Academy of Ophthalmology, RNA, Inflammation, AAV, Facial muscles, Duchenne muscular dystrophy, VEGF, Investigational New Drug, DMD, CGMP, Patient, AbbVie, Webcast, Diabetic retinopathy, Ageing, Phase, FDA, IND, Safety, Dietary supplement, Pharmaceutical industry, Cryptocurrency, Medical device, Vaccine, Duchenne, Ophthalmology

ROCKVILLE, Md., July 11, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) will announce today new interim data from the Phase II AAVIATE® and ALTITUDE® trials of ABBV-RGX-314 suprachoroidal delivery in wet AMD and diabetic retinopathy (DR). The company will also provide a comprehensive Duchenne program overview including initial safety data for RGX-202 and a new exon 53 program.

Key Points: 
  • REGENXBIO will host a virtual Investor Day today at 8:30 a.m.
  • The webcast and slides of the presentation can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com .
  • "Today, we are pleased to share an update on REGENXBIO's pipeline of AAV-based gene therapies with a focus on our retinal and Duchenne programs.
  • The virtual event will take place on Tuesday, July 11, 2023, at 8:30 a.m. Eastern Time.

Astellas and 4D Molecular Therapeutics (4DMT) Enter into License Agreement to Use 4DMT's Proprietary Intravitreal R100 Vector for Rare Ophthalmic Targets

Retrieved on: 
Tuesday, July 11, 2023
Retina, IgG4-related ophthalmic disease, Therapy, Visual impairment, Regeneration, Research, Paratriathlon, Diabetic retinopathy, AAV, MD, Patient, R100, Entrepreneurship, Russian–Ukrainian Friendship Treaty, TSE, Pharmaceutical industry, Vaccine, Astellas Pharma

R100 is an adeno-associated virus (AAV) vector invented by 4DMT for intravitreal delivery.

Key Points: 
  • R100 is an adeno-associated virus (AAV) vector invented by 4DMT for intravitreal delivery.
  • It has the ability to penetrate the internal limiting membrane barrier and to efficiently transduce the entire retina, resulting in robust transgene expression within retinal cells.
  • All three 4DMT clinical-stage ophthalmic product candidates utilize the R100 vector, including 4D-150 for wet age-related macular degeneration and diabetic macular edema.
  • Under the terms of the agreement, 4DMT will provide its proprietary R100 vector technology to Astellas to deliver Astellas' unique genetic payloads for the treatment of rare monogenic diseases.

Novel gene delivery technology published and licensed exclusively to Cellinfinity Bio

Retrieved on: 
Monday, July 10, 2023
Oncology, Health, Genetics, Research, Science, Biotechnology, Nature Biomedical Engineering, CRISPR, Electroporation, AAV, Messenger, Associate, Cell, Monocyte, CD8, MAJESTIC, CD4, Gene, Genome, Vaccine, Yale University

This technology is published in Nature Biomedical Engineering on July 10.

Key Points: 
  • This technology is published in Nature Biomedical Engineering on July 10.
  • Cellinfinity Bio has an exclusive license of the published MAJESTIC technology from Yale University and Professor Sidi Chen’s lab, Founder of Cellinfinity Bio and Associate Professor of Genetics, Yale University.
  • “We intend to employ MAJESTIC in multiple cell types, allowing development of next generation cell therapy against broad ailments.” Cellinfinity holds exclusive rights to the MAJESTIC technology.
  • In addition, the company has licensed other novel cell evolution technologies from Yale, that allow whole-genome gain-of-function and loss-of-function gene screens from the Chen lab.

Sensorion Submits Clinical Trial Application for Lead Gene Therapy Candidate OTOF-GT in the UK

Retrieved on: 
Monday, July 10, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, US Foods, Audiogenic, Dental implant, Congenital hearing loss, Health care, European Medicines Agency, Cochlea, Clinical trial, Quality of life, Mutation, CTA, AAV, IHC, EMA, Patient, Hearing, Approved Drug Products with Therapeutic Equivalence Evaluations, Medicines and Healthcare products Regulatory Agency, Orphan, Ageing, FDA, Safety, Food, MHRA, Pharmaceutical industry, Europe 1

The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.

Key Points: 
  • The phase 1/2 clinical trial (Audiogene), aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged up to 31 months.
  • The CTA submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial.
  • Sensorion’s OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss.
  • Nawal Ouzren, Chief Executive Officer of Sensorion, commented: “This first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion’s broader gene therapy franchise.

Skyline Therapeutics Receives FDA Clearance of IND for SKG0106, a Novel Intravitreally Delivered AAV Gene Therapy Candidate for Neovascular Age-related Macular Degeneration

Retrieved on: 
Monday, July 3, 2023
Immortalised cell line, GMP, Capsid, NAMD, AAV, Kaay Raav Tumhi, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Vaccine

Global Phase I clinical trial will be initiated soon.

Key Points: 
  • Global Phase I clinical trial will be initiated soon.
  • SKG0106 is a distinctively designed recombinant adeno-associated virus (rAAV)-mediated gene therapy for ocular disease.
  • It encompasses a proprietary novel AAV capsid and a uniquely configured transgene genome encoding anti-VEGF protein.
  • In comprehensive preclinical studies, SKG0106 has demonstrated potent efficacy of inhibition of intraocular neovascularization, a favorable safety profile, and long-lasting durability following a single intravitreal administration.

Enhancing AAV Full Capsid Enrichment with an Optimized AEX Platform: A Case Study, Upcoming Webinar Hosted by Xtalks

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Friday, June 30, 2023
Acceleration, Partnership, Workflow, Clinical Case Studies, CMO, Doctor of Philosophy, AAV, Research and development, CDMO, AEX, Synthetic biology, Fine chemical, Vaccine, Research

TORONTO, June 30, 2023 /PRNewswire-PRWeb/ -- In the ever-evolving field of gene therapy, a remarkable webinar is set to shed light on significant advancements in the ability to improve AAV purification and separation during downstream processing. A free webinar titled "Enhancing AAV Full Capsid Enrichment with an Optimized AEX Platform: A Case Study" is scheduled to take place, presenting the findings from a partnership between two key industry players: Teknova and Sartorius BIA Separations.

Key Points: 
  • A free webinar titled "Enhancing AAV Full Capsid Enrichment with an Optimized AEX Platform: A Case Study" is scheduled to take place, presenting the findings from a partnership between two key industry players: Teknova and Sartorius BIA Separations .
  • The webinar will focus on the successful separation of AAV empty and full capsids using anion exchange chromatography (AEX).
  • It will also demonstrate the critical parameters that affect the full capsid enrichment step in AAV purification.
  • The free webinar , "Enhancing AAV Full Capsid Enrichment with an Optimized AEX Platform: A Case Study," is scheduled for Monday, July 17, 2023, at 1pm EDT (10am PDT).

EXPRESSION SYSTEMS LAUNCHES RHABDOVIRUS-FREE CELL LINE FOR DEVELOPMENT OF ADVANCED THERAPIES

Retrieved on: 
Wednesday, June 28, 2023
Immortalised cell line, GMP, ANGUS, Sf9, Gene, Cell bank, Research, AAV, Zenith Contract Services, Documentation, Cell, ESF, VLP, Growth, A-DNA, Vaccine

BUFFALO GROVE, Ill. and DAVIS, Calif., June 28, 2023 /PRNewswire/ -- Expression Systems, an ANGUS company, and a leading global provider of specialty cell culture media formulations, cell lines, and contract services for the baculovirus expression platform, today announced early-access availability of its new rhabdovirus-free cell line for manufacturing advanced therapies.  

Key Points: 
  • BUFFALO GROVE, Ill. and DAVIS, Calif., June 28, 2023 /PRNewswire/ -- Expression Systems, an ANGUS company, and a leading global provider of specialty cell culture media formulations, cell lines, and contract services for the baculovirus expression platform, today announced early-access availability of its new rhabdovirus-free cell line for manufacturing advanced therapies.
  • The Sf9 RV-Free Insect Cell Line is a proven rhabdovirus-negative Sf9 cell line grown in suspension culture and adapted for optimized growth in Expression Systems' new ESF AdvanCD Chemically Defined Insect Cell Culture Medium.
  • "We are thrilled with the exceptional growth characteristics of Expression Systems' Sf9 RV-Free Insect Cells with our new ESF AdvanCD Chemically Defined Insect Cell Culture Medium," said Thera Mulvania, President, Expression Systems.
  • The Sf9 RV-Free Cell Line is available exclusively from Expression Systems for research use only under a Limited Use Label License ("LULL").

Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at R&D Day

Retrieved on: 
Wednesday, June 28, 2023
Logarithm, Central nervous system, Natural history, Probability, Neurodegenerative disease, IDMC, Food and Drug Administration, Skin, MECP2, Rett syndrome, CTA, SNAP, AAV, CNS, Compound muscle action potential, U.S. FDA, CHU, Regeneration, Data analysis, Visual acuity, GAN, DPM, HIV disease progression rates, Patient, CMAP, Child, FDA, UK, IND, LogMAR chart, Food, Safety, Disease, Peripheral nervous system, Pharmaceutical industry, Medical imaging, DALLAS, List of things named after Thomas Bayes

DALLAS, June 28, 2023 (GLOBE NEWSWIRE) --  Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), announced new data analyses for TSHA-120 in GAN and initial clinical observations for TSHA-102 in Rett syndrome. Taysha will host a virtual R&D Day today at 10:00 AM ET to discuss these updates. The webcast link can be accessed on the Events and Presentations section of Taysha’s website.

Key Points: 
  • Taysha will host a virtual R&D Day today at 10:00 AM ET to discuss these updates.
  • The webcast link can be accessed on the Events and Presentations section of Taysha’s website.
  • We’ve requested a formal FDA meeting to discuss these new developments to support a potential regulatory path forward for TSHA-120.
  • TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression.

New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease

Retrieved on: 
Wednesday, June 28, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Ignition, Partnership, GM2, Glycogen storage disease, Hope, Multimedia, AAV, Forge, Tay–Sachs disease, CDMO, CGMP, Patient, Entrepreneurship, HEK293, Pharmaceutical industry, Hearth

View the full release here: https://www.businesswire.com/news/home/20230628556153/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230628556153/en/
    “We are proud to be the CDMO partner for New Hope Research Foundation, an organization that is significantly advancing a gene therapy approach to provide hope for patients suffering from Tay-Sachs disease,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics.
  • “At Forge, we are ready to partner with companies, nonprofit organizations, and gene therapy developers around the world, working towards the mission we share to bring potentially life-saving gene therapies to patients.”
    Through this partnership, Forge will provide adeno-associated virus (AAV) process development, analytical services, and cGMP manufacturing to New Hope Research Foundation.
  • The Foundation will leverage Forge’s platform processes, including its proprietary HEK293 suspension Ignition Cells™, to accelerate the initial production.
  • All development and AAV manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy facility in Columbus, Ohio.

Neuronascent to Present New Parkinson’s Model Data at AAIC 2023 for the Alzheimer’s Disease Investigational Therapy, NNI-362

Retrieved on: 
Wednesday, June 28, 2023
Learning, Neuron, Substantia nigra, NIA, Ageing, Brain, AAV, Regeneration, AAIC, Research, Alpha-synuclein, Memory, Conference, Hippocampus, Disease, Pharmaceutical industry, Vaccine, Takele Uma Banti

The main intent of this Parkinson’s model work was to assess the capacity of NNI-362, to regenerate neurons and ameliorate behavioral deficits for age-related neurodegenerative disorders in those regions of the brain where disease is present.

Key Points: 
  • The main intent of this Parkinson’s model work was to assess the capacity of NNI-362, to regenerate neurons and ameliorate behavioral deficits for age-related neurodegenerative disorders in those regions of the brain where disease is present.
  • The Phase 1a trials were successfully completed in a healthy aged population in 2021, for Alzheimer’s disease, and Neuronascent aims to continue toward Phase 2 proof-of-concept trials for more than one age-related, neurodegenerative indication.
  • To determine whether NNI-362 could support disease-specific regeneration of neurons outside of the hippocampus, an AAV-alpha synuclein model of Parkinson’s disease was run at Motac, France, under the guidance of Dr. Erwan Bezard.
  • This work aims to assess whether NNI-362 is disease-agnostic, such that in aging the neurodegeneration can be reversed no matter the triggers of disease neuron degeneration.