Applied genetics

Orchard Therapeutics and Pharming Group Announce Collaboration to Develop and Commercialize ex vivo autologous HSC Gene Therapy for Hereditary Angioedema

Retrieved on: 
Thursday, July 1, 2021
Branches of biology, Life sciences, Biology, Biotechnology, Medical genetics, Emerging technologies, Applied genetics, Therapy, Gene therapy, Angioedema, Stem cell, Metachromatic leukodystrophy

Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with angioedema attacks.

Key Points: 
  • Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with angioedema attacks.
  • Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies.
  • Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration.
  • In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

Neurogene Announces EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy 

Retrieved on: 
Tuesday, June 29, 2021
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Gene therapy, Emerging technologies, Medical genetics, Adeno-associated virus, Batten disease, Therapy, Gene therapy of the human retina, Orphan Drug Designation, CLN5, a subtype of Batten disease, Genetic Testing, Neurogene Inc., ORPHAN DRUG DESIGNATION, CLN5, A SUBTYPE OF BATTEN DISEASE, GENETIC TESTING, NEUROGENE INC.

Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.

Key Points: 
  • Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.
  • Applications for orphan designation are examined by the EMAs Committee for Orphan Medicinal Products (COMP), using the Committees network of experts.
  • Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.
  • Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.

ImmunityBio and NantOmics Announce Clinical Validation of a Proprietary Method to Identify Unique Targets for Immunotherapy in Individual Breast Cancer Patients

Retrieved on: 
Monday, June 28, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Branches of biology, Medical specialties, Clinical medicine, Applied genetics, Biotechnology, Cancer vaccines, Genomics, Medical genetics, ImmunityBio, Neoepitope, Cancer immunotherapy, Patrick Soon-Shiong

The pipeline is described in Identification and validation of expressed HLA-binding breast cancer neoepitopes for potential use in individualized cancer therapy, which recently published in the Journal for Immunotherapy of Cancer.

Key Points: 
  • The pipeline is described in Identification and validation of expressed HLA-binding breast cancer neoepitopes for potential use in individualized cancer therapy, which recently published in the Journal for Immunotherapy of Cancer.
  • In clinical use, this neoepitope discovery system supports the targeted delivery of antigens with ImmunityBios second-generation Adeno platform.
  • The future of immunotherapy is in a personalized approach, said Dr. Patrick Soon-Shiong, Founder and Executive Chairman of ImmunityBio.
  • ImmunityBio cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof.

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update

Retrieved on: 
Tuesday, June 22, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Biotechnology, Bioethics, Gene therapy, Therapy, Adeno-associated virus, Spark Therapeutics, Jeanne Lusher

and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.

Key Points: 
  • and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.
  • These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date.
  • All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec.
  • The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec.

Industry Leaders Join 23andMe Board of Directors

Applied genetics, 23andMe, Branches of biology, Academic disciplines, California

Valerie and Peter are both strong, impactful leaders who bring diverse perspectives.

Key Points: 
  • Valerie and Peter are both strong, impactful leaders who bring diverse perspectives.
  • 23andMe is uniquely positioned to empower and engage with individuals around their health and well-being, said Mr. Taylor, president of ECMC Foundation.
  • 23andMe has pioneered direct access to genetic information as the only company with multiple FDA authorizations for genetic health risk reports.
  • The 23andMe research platform has generated more than 180 publications on the genetic underpinnings of a wide range of diseases, conditions, and traits.

Vedere Bio II Announces Appointment of Gabor Veres, Ph.D., as Chief Scientific Officer

Retrieved on: 
Tuesday, June 22, 2021
Life sciences, Branches of biology, Health sciences, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, Applied genetics, BioMarin Pharmaceutical, Therapy, Gene therapy

CAMBRIDGE, Mass., June 22, 2021 /PRNewswire/ --Vedere Bio II, Inc., a company developing next-generation ocular gene therapies for vision restoration and preservation in patients with vision loss due to photoreceptor death, today announced the appointment of Gabor Veres, Ph.D., as Chief Scientific Officer.

Key Points: 
  • CAMBRIDGE, Mass., June 22, 2021 /PRNewswire/ --Vedere Bio II, Inc., a company developing next-generation ocular gene therapies for vision restoration and preservation in patients with vision loss due to photoreceptor death, today announced the appointment of Gabor Veres, Ph.D., as Chief Scientific Officer.
  • Dr. Veres joins Vedere Bio II from BioMarin, where he served as Vice President, Head of Gene Therapy Research, focusing on AAV platform discovery and identifying new therapeutic indications in gene therapy.
  • "Vedere's approach to vision restoration holds great potential to treat vision loss regardless of underlying genetic cause or disease stage, and I'm looking forward to helping build out and progress the company's unique platform and product pipeline," said Gabor Veres, Chief Scientific Officer at Vedere Bio II.
  • For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn .

Veritas Intercontinental: Genetics makes it possible to identify cardiovascular genetic risk and prevent cardiac accidents such as those that have been in the news in recent days

Retrieved on: 
Tuesday, June 22, 2021
Branches of biology, Biology, Medical genetics, Applied genetics, Medical tests, Genetics, Human genetics, Genetic testing, Exome sequencing, Genetic counseling, Exome, Pathology

- Genetic diagnosis allows the patient to be referred to a specialist of the cardiological pathology detected, allowing appropriate management of the patient and genetic counseling for family members.

Key Points: 
  • - Genetic diagnosis allows the patient to be referred to a specialist of the cardiological pathology detected, allowing appropriate management of the patient and genetic counseling for family members.
  • MADRID, June 22, 2021 /PRNewswire/ -- We have recently witnessed, once again, a professional athlete suffering a cardiovascular attack during a match.
  • This type of incidence and the possible fatal consequences result from an individual's genetic makeup.
  • Whole exome sequencing (WES) is the most appropriate tool to address the genetic heterogeneity present in inherited cardiovascular disease.

Best Prime Day DNA Test Deals 2021: Early 23andMe, AncestryDNA & More DNA Test Kit Sales Rated by Consumer Articles

Retrieved on: 
Sunday, June 20, 2021
MEN, Online Retail, Family, Discount, Variety, Specialty, Consumer, Genetics, Teens, Retail, Women, Seniors, Health, Pets, Retailing, Retailers, Applied genetics, Branches of biology, Ancestry.com, 23andMe, Walmart, E-commerce, Advertising

Click here to access the entire selection of deals at Walmarts Deals for Days sale and click here to see all of Amazons deals.

Key Points: 
  • Click here to access the entire selection of deals at Walmarts Deals for Days sale and click here to see all of Amazons deals.
  • Consumer Articles earns commissions from purchases made using the links provided.
  • One of the best ways to get to know yourself better is by taking a DNA Test (AncestryDNA, 23andMe).
  • About Consumer Articles: Consumer Articles shares informative e-commerce news.

DNA Test Prime Day Deals 2021: Early 23andMe & AncestryDNA Test Kit Deals Highlighted by Deal Tomato

Retrieved on: 
Saturday, June 19, 2021
Retail, Genetics, Health, Other Retail, Applied genetics, Branches of biology, Genetics, Retailers, Ancestry.com, Genealogy software, Lehi, Utah, 23andMe, Tomato, Amazons, Walmart

Click here to enjoy the entire range of deals at Walmarts Deals for Days sale (June 20 23) and click here to browse Amazons active and upcoming deals.

Key Points: 
  • Click here to enjoy the entire range of deals at Walmarts Deals for Days sale (June 20 23) and click here to browse Amazons active and upcoming deals.
  • Shoppers who want to find out more about themselves can always take a DNA test (23andMe, AncestryDNA).
  • With AncestryDNA, you can find out exactly where your family is from and even discover your origins, ethnicity, and DNA matches.
  • About Deal Tomato: Deal Tomato reports on popular sales events.

New Cell and Gene Therapy Business Outlook Newsletter Launched

Retrieved on: 
Friday, June 18, 2021
Branches of biology, Life sciences, Health sciences, Applied genetics, Bioethics, Gene therapy, Molecular biology, Therapy

ARLINGTON, Va., June 18, 2021 /PRNewswire/ --Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, announces the publication of Cell and Gene Therapy Business Outlook.

Key Points: 
  • ARLINGTON, Va., June 18, 2021 /PRNewswire/ --Science and Medicine Group, the company behind Instrument Business Outlook, Kalorama Information, SDi and other publications, announces the publication of Cell and Gene Therapy Business Outlook.
  • This new twice-monthly publication dedicated to cell and gene therapy, Cell and Gene Therapy Business Outlook offers the following:
    Cell and Gene Therapy Tools, CMOs, Manufacturing Developments
    Unique features of this new newsletter include market analysis in each issue and a table that tracks important deals in the cell and gene industry.
  • Edited by Blake Middleton, a professional CGT researcher and former Staff Research Associate at UCLA Department of Pharmacology, Cell and Gene Therapy Business Outlook is designed to provide the most relevant news.
  • Cell and Gene Therapy Business Outlook also explains the relevant science behind partnerships, product launches and business deals.