Gene delivery

A New Single-Cell Sequencing and Analysis Platform to Accelerate Cell and Gene Therapy Pipelines, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Tuesday, May 18, 2021

Many of these therapies involve the genetic engineering of cells in order to insert exogenous sequences or alter existing genes.

Key Points: 
  • Many of these therapies involve the genetic engineering of cells in order to insert exogenous sequences or alter existing genes.
  • After the cells are altered, they are introduced into the patient where they either replace dysfunctional cells or provide a desired immune response.
  • For instance, there may be cell-to-cell variability in transduction efficiency and copy number (for viruses), or for on- and off-target editing efficiencies (for gene editors).
  • Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.\nSydney Perelmutter, Xtalks, +1 (416) 977-6555 x 352, [email protected]\n'

Novocure Announces FDA Approval of IDE Supplement for Phase 3 Pivotal LUNAR Trial of Tumor Treating Fields in Non-Small Cell Lung Cancer

Retrieved on: 
Tuesday, May 18, 2021

After review of the interim analysis report earlier this year, the DMC concluded that the LUNAR trial should continue with no evidence of increased systemic toxicity.

Key Points: 
  • After review of the interim analysis report earlier this year, the DMC concluded that the LUNAR trial should continue with no evidence of increased systemic toxicity.
  • It is estimated that approximately 46,000 patients receive second-line treatment for stage 4 NSCLC each year in the U.S.
  • Electrically charged proteins within the cell are critical for cell division, making the rapidly dividing cancer cells vulnerable to electrical interference.
  • TTFields, however, have a unique frequency range, between 100 to 500 kHz, enabling the electric fields to penetrate the cancer cell membrane.

Charles River Laboratories to Acquire Vigene Biosciences to Enhance Gene Therapy Capabilities

Retrieved on: 
Monday, May 17, 2021

b"Charles River Laboratories International, Inc. (NYSE: CRL) announced today that it has signed a definitive agreement to acquire Vigene Biosciences, Inc. , a premier, U.S.-based gene therapy contract development and manufacturing organization (CDMO) providing viral vector-based gene delivery solutions.

Key Points: 
  • b"Charles River Laboratories International, Inc. (NYSE: CRL) announced today that it has signed a definitive agreement to acquire Vigene Biosciences, Inc. , a premier, U.S.-based gene therapy contract development and manufacturing organization (CDMO) providing viral vector-based gene delivery solutions.
  • We look forward to welcoming Vigene\xe2\x80\x99s dedicated employees to the Charles River family.\xe2\x80\x9d\nThe acquisition of Vigene Biosciences will enhance Charles River\xe2\x80\x99s gene therapy capabilities in the high-growth, value-added cell and gene therapy CDMO sector.\nExpands Charles River\xe2\x80\x99s Gene Therapy CDMO Capabilities for Viral Vectors and Plasmid DNA \xe2\x80\x93 Vigene offers its clients contract manufacturing solutions across several key gene therapy platforms, enhancing Charles River\xe2\x80\x99s ability to meet its clients\xe2\x80\x99 evolving scientific needs.
  • Its primary area of expertise is CGMP viral vector manufacturing, which is used for gene therapies and gene-modified cell therapies.
  • Vigene will also support Charles River\xe2\x80\x99s existing, U.S.-based cell therapy production capabilities and establish an end-to-end, gene-modified cell therapy solution.

Global and Chinese Viral Vectors and Plasmid DNA Manufacturing Markets, 2016-2020 & 2021-2026 - ResearchAndMarkets.com

Retrieved on: 
Monday, May 17, 2021

b'The "Global and Chinese Viral Vectors and Plasmid Dna Manufacturing Industry, 2021 Market Research Report" report has been added to ResearchAndMarkets.com\'s offering.\n\'Global and Chinese Viral Vectors and Plasmid DNA Manufacturing Industry, 2021 Market Research Report\' is a professional and in-depth study on the current state of the global Viral Vectors and Plasmid DNA Manufacturing industry with a focus on the Chinese market.\nThe report provides key statistics on the market status of the Viral Vectors and Plasmid DNA Manufacturing manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry.\nFirstly, the report provides a basic overview of the industry including its definition, applications and manufacturing technology.

Key Points: 
  • b'The "Global and Chinese Viral Vectors and Plasmid Dna Manufacturing Industry, 2021 Market Research Report" report has been added to ResearchAndMarkets.com\'s offering.\n\'Global and Chinese Viral Vectors and Plasmid DNA Manufacturing Industry, 2021 Market Research Report\' is a professional and in-depth study on the current state of the global Viral Vectors and Plasmid DNA Manufacturing industry with a focus on the Chinese market.\nThe report provides key statistics on the market status of the Viral Vectors and Plasmid DNA Manufacturing manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry.\nFirstly, the report provides a basic overview of the industry including its definition, applications and manufacturing technology.
  • In this part, the report presents the company profile, product specifications, capacity, production value, and 2016-2021 market shares for each company.\nThrough statistical analysis, the report depicts the global and Chinese total market of Viral Vectors and Plasmid DNA Manufacturing industry including capacity, production, production value, cost/profit, supply/demand and Chinese import/export.
  • The total market is further divided by company, by country, and by application/type for the competitive landscape analysis.\nThe report then estimates 2021-2026 market development trends of the Viral Vectors and Plasmid DNA Manufacturing industry.
  • In the end, the report makes some important proposals for a new project of the Viral Vectors and Plasmid DNA Manufacturing Industry before evaluating its feasibility.\nOverall, the report provides an in-depth insight of the 2021 global and Chinese Viral Vectors and Plasmid DNA Manufacturing industry covering all important parameters.\n'

Otonomy and AGTC Present Preclinical Proof-of-Concept Results for OTO-825 Gene Therapy at ASGCT Annual Meeting

Retrieved on: 
Friday, May 14, 2021

b'SAN DIEGO, May 14, 2021 (GLOBE NEWSWIRE) -- Otonomy, Inc.\xc2\xa0(Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical proof-of-concept results for OTO-825 presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.

Key Points: 
  • b'SAN DIEGO, May 14, 2021 (GLOBE NEWSWIRE) -- Otonomy, Inc.\xc2\xa0(Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical proof-of-concept results for OTO-825 presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.
  • OTO-825 is an AAV-mediated gene therapy targeting the gap junction beta-2 (GJB2) gene developed under the company\xe2\x80\x99s collaboration with Applied Genetic Technologies Corporation (Nasdaq: AGTC).
  • The forward-looking statements in this press release are based on information available to Otonomy as of the date hereof.
  • Otonomy disclaims any obligation to update any forward-looking statements, except as required by law.\n'

Luye Life Sciences Unveils Cutting-edge Advances in Gene and Cell Therapy at ASGCT 2021

Retrieved on: 
Friday, May 14, 2021

b'BOSTON, May13, 2021 /PRNewswire/ -- The Boston R&D Center and GeneLeap Biotech, two subsidiaries of Luye Life Sciences Group, tookpart in the 24th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, presenting the progress of four of the company\'s latest preclinical developments in gene and cell therapy to the global biological community.\nASGCT is one of the world\'s most influential academic conferences in the area of gene and cell therapy.

Key Points: 
  • b'BOSTON, May13, 2021 /PRNewswire/ -- The Boston R&D Center and GeneLeap Biotech, two subsidiaries of Luye Life Sciences Group, tookpart in the 24th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, presenting the progress of four of the company\'s latest preclinical developments in gene and cell therapy to the global biological community.\nASGCT is one of the world\'s most influential academic conferences in the area of gene and cell therapy.
  • Sean Fu, President of Luye Life Sciences Boston R&D Center, and CEO of GeneLeap Biotech, said, "This is the first time that we have presented information on the progress of our scientific research in gene and cell therapy at the ASGCT annual meeting.
  • "\nThe R&D center at Boston, Woburn, MA is a subsidiary of Luye Life Sciences, developing novel drugs for cancer and genetic disorders, including gene therapy, immune checkpoint inhibitory antibodies, CAR-T cells therapy, and novel drug delivery mechanisms.
  • At present, the AAV platform for gene therapy program developed by the Boston R&D center has several candidates in the pipeline.\nGeneLeap Bio., a subsidiary of Luye Life Sciences, specializes in developing gene therapy drugs for cancer and severe infectious diseases, including mRNA and oligonucleotides.

Taysha Gene Therapies Expands Leadership Team with the Appointment of Chief Development Officer

Retrieved on: 
Thursday, May 13, 2021

b'Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer.

Key Points: 
  • b'Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-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) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer.
  • Her decades of experience in all key aspects of drug development, particularly in gene therapy and rare disease, will be invaluable as we continue to grow our business.\xe2\x80\x9d\nMs.
  • Newman joins Taysha with more than 30 years of experience in regulatory affairs and research and development within the biotechnology industry, focusing on rare diseases.
  • \xe2\x80\x9cTaysha is well-positioned to be a leader in developing disease-modifying gene therapies for patients with monogenic CNS diseases.

Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs at 24th Annual Meeting of the American Society of Gene and Cell Therapy

Retrieved on: 
Thursday, May 13, 2021

All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.

Key Points: 
  • All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.
  • Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia.
  • Rocket\xe2\x80\x99s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Pregene and Dr. Reddy's Announce License Agreement for Anti-BCMA CAR-T PRG1801 in India

Retrieved on: 
Thursday, May 13, 2021

Reddy's acquires the exclusive rights to commercialize PRG1801 in India, and will be responsible for future development including clinical trials, in India.

Key Points: 
  • Reddy's acquires the exclusive rights to commercialize PRG1801 in India, and will be responsible for future development including clinical trials, in India.
  • Pregene will be the exclusive supplier of the core material - lentiviral vectors for manufacturing PRG1801.
  • The lentivirus vectors are produced by Pregene using a proprietary serum free suspension production system with gene-therapy-grade quality and a high transduction unit yield.
  • Pregene's product pipeline includes various cell therapy programs under development for the treatment of hematological malignancies, solid tumors and bone defects.\nDr.

Taysha Gene Therapies to Participate in Upcoming May Investor Healthcare Conferences

Retrieved on: 
Wednesday, May 12, 2021

b'Webcasts for these conferences will be available in the \xe2\x80\x9cEvents & Media\xe2\x80\x9d section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .

Key Points: 
  • b'Webcasts for these conferences will be available in the \xe2\x80\x9cEvents & Media\xe2\x80\x9d section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .
  • Archived versions of the webcasts will be available on the website for 60 days.\nTaysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.
  • With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside.
  • We have combined our team\xe2\x80\x99s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications.