Applied genetics

Seeding a Life-Changing International Genomic Insight Alliance to Treat Pediatric Epilepsy, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Thursday, March 11, 2021
Epilepsy, Pediatrics, Health, Medical specialties, Clinical medicine, Applied genetics

As a community, it is our responsibility to ensure access and equitability of knowledge and to continue to drive understanding and precision treatments for pediatric epilepsy.

Key Points: 
  • As a community, it is our responsibility to ensure access and equitability of knowledge and to continue to drive understanding and precision treatments for pediatric epilepsy.
  • As epilepsy can appear similar across individuals, the use of not only clinical differentiation but also genetic sequencing can aid in determining cause and treatment regimen.
  • In partnership, Sanford Health and Congenica are working to drive forward genomic insights for pediatric epilepsy to address the existing challenges.
  • For more information, or to register for this event, visit Seeding a Life-Changing International Genomic Insight Alliance to Treat Pediatric Epilepsy.

MeiraGTx Reports Fourth Quarter and Full Year 2020 Financial and Operational Results

Retrieved on: 
Thursday, March 11, 2021
Branches of biology, Biology, Applied genetics, Gene delivery, Emerging technologies, Medical genetics, Molecular biology, Xerostomia, Gene therapy, AAV

LONDONandNEW YORK, March 11, 2021 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent progress.

Key Points: 
  • LONDONandNEW YORK, March 11, 2021 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent progress.
  • AAV-AQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia:
    MeiraGTx reported preliminary data from the Phase 1 AQUAx clinical trial in December 2020.
  • In 2020, MeiraGTx and Janssen were granted Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations for AAV-RPGR.
  • Research and development expenses for the years ended December 31, 2020 and 2019 were as follows (in millions):

Sio Gene Therapies to Participate in Upcoming Investor & Scientific Conferences

Retrieved on: 
Thursday, March 11, 2021
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Congenital disorders, Applied genetics, Sandhoff disease, Tay–Sachs disease, Gene therapy, Therapy

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Taysha Gene Therapies Announces Participation in Upcoming March Investor Healthcare Conferences

Retrieved on: 
Thursday, March 11, 2021
Biotechnology, General Health, Health, Genetics, Pharmaceutical, Branches of biology, Life sciences, Biology, Biotechnology, Gene delivery, Applied genetics, Molecular biology, Emerging technologies, Therapy, Gene therapy, Monogenic

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, 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) in both rare and large patient populations, today announced its participation in virtual fireside chats for the Oppenheimer 31st Annual Healthcare Conference and Stifels 3rd Annual CNS Day.

Key Points: 
  • Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, 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) in both rare and large patient populations, today announced its participation in virtual fireside chats for the Oppenheimer 31st Annual Healthcare Conference and Stifels 3rd Annual CNS Day.
  • Audio webcasts for these conferences will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .
  • Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.
  • We have combined our teams 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.

Taysha Gene Therapies Announces Collaboration to Advance Next-Generation Mini-Gene Payloads for an AAV Gene Therapy for the Treatment of Neurodevelopmental Disorders

Retrieved on: 
Tuesday, March 9, 2021
Health, Genetics, Other Health, Research, Science, Pharmaceutical, Biotechnology, Branches of biology, Biology, Life sciences, Gene delivery, Biotechnology, Genetic engineering, Applied genetics, Molecular genetics, Viral vector, Gene therapy, Mental disorder, University of Texas Southwestern Medical Center

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, 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) in both rare and large patient populations, today announced a multi-year collaboration to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of neurodevelopmental disorders.

Key Points: 
  • Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, 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) in both rare and large patient populations, today announced a multi-year collaboration to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of neurodevelopmental disorders.
  • Taysha will have an exclusive option on new payloads, constructs, and intellectual property associated with, and arising from, the research conducted under this agreement.
  • Under the terms of this most recent agreement, a team of researchers from Yale University will create mini-gene payloads designed to treat neurodevelopmental disorders including intellectual disability.
  • UTSW will produce viral vector constructs that incorporate the mini-gene payloads and evaluate the constructs in in vivo and in vitro efficacy models.

Biogen Announces Plans to Build a New, State-of-the-Art Gene Therapy Manufacturing Facility in Research Triangle Park, North Carolina

Retrieved on: 
Thursday, March 4, 2021
Life sciences, Branches of biology, Biology, Biotechnology, Medical genetics, Biogen, Spinal muscular atrophy, Applied genetics, Bioethics, Therapy, Gene therapy

CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced its plans to build a new gene therapy manufacturing facility at its Research Triangle Park (RTP) manufacturing campuses in North Carolina to support its growing gene therapy pipeline across multiple therapeutic areas.

Key Points: 
  • CAMBRIDGE, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced its plans to build a new gene therapy manufacturing facility at its Research Triangle Park (RTP) manufacturing campuses in North Carolina to support its growing gene therapy pipeline across multiple therapeutic areas.
  • With this new facility, Biogen is investing in robust and scalable gene therapy manufacturing with the goal of ensuring reliable supply to patients worldwide.
  • Gene therapy is an emerging therapeutic modality that may be suitable for a growing list of genetically validated targets in neuroscience.
  • We plan to build differentiated, sustainable and advanced manufacturing capabilities to support our gene therapy programs and collaborations, said Nicole Murphy, Senior Vice President, Global Manufacturing and Technical Operations.

Matica Biotechnology, Inc. Announces Master Research Agreement with Texas A&M

Retrieved on: 
Wednesday, March 3, 2021
Branches of biology, Biology, Life sciences, Gene delivery, Virotherapy, Applied genetics, Genetic engineering, Medical genetics, Viral vector, Gene therapy, Lentivirus

The agreement covers joint research and development projects for plasmid, protein and viral vector products in compliance with FDA regulations.

Key Points: 
  • The agreement covers joint research and development projects for plasmid, protein and viral vector products in compliance with FDA regulations.
  • "Texas A&M University is internationally renowned for its research programs developing novel vaccines and therapies to improve public health," stated Dr. Byung Se So, CEO of Matica Bio.
  • "Matica Bio is excited to partner with the TAMHSCCIADM staff as we strive to innovate manufacturing solutions for our clients' novel cell and gene therapy products.
  • Matica Bio's Texas facility is scheduled to open in Q3 2021 utilizing a bioreactor-based platform for the production of Lentivirus and AAV vectors.

Global Biobanks Markets Report 2021-2028: Biobanks Continue to Evolve with New Technologies Such as NGS and Increased Focus on Genomic Medicine

Retrieved on: 
Tuesday, March 2, 2021
Applied genetics, Biological databases, Biobanks, Branches of biology, Biological specimen, Centers for Disease Control and Prevention, Applied sciences, Academic disciplines

The global biobanks market size is expected to reach USD 106.9 billion by 2028 and is expected to expand at a CAGR of 6.2% from 2021 to 2028.

Key Points: 
  • The global biobanks market size is expected to reach USD 106.9 billion by 2028 and is expected to expand at a CAGR of 6.2% from 2021 to 2028.
  • Biobanks continue to evolve with the introduction of new technologies such as NGS and increased focus on genomic medicine.
  • The quality of biospecimens can significantly influence the disease testing as well as preclinical and clinical research.
  • For instance, the U.S. Centers for Disease Control and Prevention (CDC) released guidelines to minimize human hazards while handling COVID-19 samples.

Freeline to Present at Upcoming Investor Conferences

Retrieved on: 
Tuesday, March 2, 2021
Branches of biology, Applied genetics, Parvovirinae, Genetics, Adeno-associated virus, Parvoviruses, Biology, AAV, Gene therapy

ET

Key Points: 
  • ET
    The Company presentations from both conferences will be available on the investor relations section of the Freeline website at Freeline.life .
  • Senior management will also participate in virtual one-on-one meetings with investors at these conferences.
  • Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies.
  • The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases.

Biobanks (Virtual, Physical) Market Size, Share & Trends Analysis Report 2021 - Global Forecast to 2028 - ResearchAndMarkets.com

Retrieved on: 
Friday, February 26, 2021
Health, Biotechnology, Biobanks, Biological databases, Applied genetics, Branches of biology, Biological specimen, Applied sciences, Academic disciplines

The "Biobanks Market Size, Share & Trends Analysis Report by Product, by Service (Cold Chain, Lab Processing), by Biospecimen Type, by Biobank Type (Virtual, Physical), by Application, by Region, and Segment Forecasts, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Biobanks Market Size, Share & Trends Analysis Report by Product, by Service (Cold Chain, Lab Processing), by Biospecimen Type, by Biobank Type (Virtual, Physical), by Application, by Region, and Segment Forecasts, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.
  • The global biobanks market size is expected to reach USD 106.9 billion by 2028 and is expected to expand at a CAGR of 6.2% from 2021 to 2028.
  • Biobanks continue to evolve with the introduction of new technologies such as NGS and increased focus on genomic medicine.
  • The availability of several types of biospecimens to cater to multiple domains including drug discovery, diagnostics, and others has accelerated the diversification of biorepositories, thereby driving the market.