Kaay Raav Tumhi

AskBio Contribution to Advancing Gene Therapy Highlighted at ESGCT 30th Congress

Retrieved on: 
Monday, October 23, 2023
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Includes data from AskBio Wholly Owned Manufacturing Subsidiaries, TAAV and Viralgen

Key Points: 
  • Includes data from AskBio Wholly Owned Manufacturing Subsidiaries, TAAV and Viralgen
    RESEARCH TRIANGLE PARK, N.C., Oct. 23, 2023 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that 20 company abstracts will be presented at the European Society for Gene and Cell Therapy (ESGCT) 30th Congress, which is being held in Brussels, Belgium, from October 24 to 27, 2023.
  • The full company presence highlights the breadth of the AskBio contribution to advancing gene therapy and includes an oral presentation of the company's work on immune modulation and the role of genetic diversity in translational animal models of AAV gene therapy, as well as a poster presentation on rational design of regulatable expression cassettes in non-human primates.
  • "With 20 presentations at this year's ESGCT, including data from our wholly owned subsidiaries, TAAV and Viralgen, AskBio's ambition is clear when it comes to advancing the science and driving innovation in gene therapy," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.
  • "ESGCT provides us with an important platform to share our work with the broader scientific community and demonstrate the transformative power of AAV technology and gene therapy."

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.

Adverum Biotechnologies Introduces an Intravitreal Gene Therapy Program for Geographic Atrophy and Presents Data on Its Ocular Gene Therapy Platform

Retrieved on: 
Thursday, May 18, 2023
Injection, CFI, Vance DeGeneres, Gene, MOI, AMD, CMC, Optogenetics, Research, GA, Publication, AAV, Geographic atrophy, IVT, Protein, Patient, Complement factor I, Kaay Raav Tumhi, Annual general meeting, Cell, Retinal pigment epithelium, Sf9 (cells), Poster, RPE, Kinetics, Society, Disease, Vaccine, Chemistry, Adverum Biotechnologies

REDWOOD CITY, Calif., May 18, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today presented new nonclinical data on an IVT gene therapy for the treatment of GA secondary to dry age-related macular degeneration (dry AMD) via expression of CFI at the American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting, in Los Angeles, California. Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.

Key Points: 
  • Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.
  • “The ability to deliver both CFI and an engineered melanopsin leveraging our proprietary capsids via IVT administration demonstrates the strength of our platform at Adverum,” commented Brigit Riley, Ph.D., chief scientific officer of Adverum Biotechnologies.
  • “Similar to wet AMD, patients with GA are forced either to receive frequent, often monthly, injections or to experience faster lesion growth and vision deterioration.
  • The ASGCT poster and oral presentations will be made available on the Publications page of the Adverum website.

Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting

Retrieved on: 
Monday, May 8, 2023
Immortalised cell line, Propionic acidemia, Promise, Gene, Challenge, Safety, PCCB, Efficacy, Institutional review board, CDKL5, Fibroblast, Los Angeles Convention Center, AAV, Downstream, CDKL5 deficiency disorder, Immunosuppression, Kaay Raav Tumhi, OTC, Cell, Glycogen storage disease, PCCA, CDD, Abstract, Society, Rare, Pharmaceutical industry, Medical device, Vaccine, Antibiotics, Pinnacle

The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.

Key Points: 
  • The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.
  • 1424)
    Poster presentation: Development of UX055 AAV Gene Therapy for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD), a Rare Neurological Disorder (Board No.
  • 1340)
    Poster presentation: Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy (Board No.
  • 1578)
    In addition to the data presentations, Ultragenyx will present at one pre-meeting workshop on Tuesday, May 16:

Skyline Therapeutics to Present at the ASGCT 26th Annual Meeting

Retrieved on: 
Wednesday, May 3, 2023
SMN, SMA, RNV, Gene, Anti-VEGF, CNV, NAMD, Clinical trial, SMN1, Toxicity, AAV, CNS, DSM-IV codes, IVT, VEGF, Neuromuscular junction, Patient, Kaay Raav Tumhi, Annual general meeting, Cell, NMJ, American Society of Gene and Cell Therapy, Liver, NHP, Therapy, Society, Human, A-DNA, Choroidal neovascularization, Pharmaceutical industry, Vaccine

SHANGHAI, May 3, 2023 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases, today announced multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles, CA, May 16-20, 2023.

Key Points: 
  • SHANGHAI, May 3, 2023 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases, today announced multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles, CA, May 16-20, 2023.
  • SKG0106 is a proprietary AAV vector carrying a transgene genome encoding a novel anti-VEGF protein.
  • Upon delivery, SKG0106 effectively and specifically suppresses the action of human VEGF, a key factor contributing to the pathological angiogenesis in the eyes of nAMD patients.
  • SKG0201 is a novel AAV-mediated gene replacement therapy comprising an optimized human SMN1 cDNA under the control of a unique promoter.

Agathos Biologics Launches Analytical Services

Retrieved on: 
Wednesday, April 12, 2023
Health, Health Technology, Genetics, Radiology, Research, Science, Biotechnology, QIAGEN, Multimedia, Mycoplasma, Kaay Raav Tumhi, Nested polymerase chain reaction, Biology, Immunohistochemistry, PCR, Microscope, Vaccine, Agatha (given name)

Agathos Biologics, a leading biotechnology company, is proud to offer advanced analytical testing services for life science researchers.

Key Points: 
  • Agathos Biologics, a leading biotechnology company, is proud to offer advanced analytical testing services for life science researchers.
  • Agathos Biologics uses the latest digital PCR (dPCR) technology, delivering unparalleled sensitivity and precision in nucleic acid quantification.
  • These services represent the first step in our strategy to create breakthrough products and services within a strong ethical and moral framework that benefit everyone.” For more information on high-content imaging and dPCR, visit Agathos Services on our web site.
  • Agathos Biologics is a biotechnology company pursuing transformational science in biomanufacturing, biologic payload delivery, and cell and gene therapy.

Codexis Announces Several Key Presentations from its Gene Therapy Programs at 19th Annual WORLDSymposium™

Retrieved on: 
Wednesday, February 22, 2023
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REDWOOD CITY, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company, today highlights several key presentations from its gene therapy programs from 19 th Annual WORLDSymposium™ , taking place from February 22-26, 2023, in Orlando, Florida.

Key Points: 
  • REDWOOD CITY, Calif., Feb. 22, 2023 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company, today highlights several key presentations from its gene therapy programs from 19 th Annual WORLDSymposium™ , taking place from February 22-26, 2023, in Orlando, Florida.
  • Takeda is combining these improved transgenes with its gene therapy capabilities to develop novel product candidates for the treatment of rare genetic disorders.
  • Takeda’s poster presentation, titled, “Preventing Fabry disease progression in a symptomatic mouse model with a recombinant adeno-associated virus (rAAV) based gene therapy,” highlights its rAAV-based gene therapy candidate for the potential treatment of Fabry disease.
  • Codexis is also presenting two abstracts highlighting its gene therapy program in GM1 Gangliosidosis (GM1) at WORLDSymposium™.

Recombinant Adeno-Associated Virus (rAAV) Technology Pioneered by AskBio's Dr. Jude Samulski is Key Component of All FDA Approved AAV Gene Therapeutics

Retrieved on: 
Tuesday, December 20, 2022
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"The progress made in gene therapy to date is remarkable," said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer.

Key Points: 
  • "The progress made in gene therapy to date is remarkable," said Jude Samulski, AskBio Co-Founder, President and Chief Scientific Officer.
  • My ultimate vision, shared by all of us at AskBio, is to make gene therapy more accessible for everyone in need around the world.
  • Dr. Samulski led the UNC Gene Therapy Center from 1993 to 2016 and, in 2001, co-founded AskBio, where many advances in recombinant AAV (rAAV) and underlying AAV vector technology were, and are being, made.
  • Since 2017, three AAV gene therapies have been approved for use by the FDA.

AskBio Co-Founder Jude Samulski Receives Inaugural European Society of Gene and Cell Therapy Founders Award

Retrieved on: 
Friday, October 28, 2022
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RESEARCH TRIANGLE PARK, N.C., Oct. 28, 2022 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., AskBio President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29th Congress.

Key Points: 
  • "ESGCT created the Founders Award this year to mark the Society's 30th anniversary," said Professor Hildegard Bning, ESGCT Board President.
  • "The award highlights benchmark milestones in gene and cell therapy.
  • The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982.
  • In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award.

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.