Medical genetics

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Retrieved on: 
Monday, October 5, 2020

FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.

Key Points: 
  • FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.
  • This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts.
  • Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene.
  • Fulcrum is also advancing FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa

Retrieved on: 
Monday, October 5, 2020

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa.

Key Points: 
  • CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa.
  • The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com .
  • Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery.
  • Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases.

AVROBIO Expands Lentiviral Gene Therapy Pipeline with Program for Hunter Syndrome

Retrieved on: 
Monday, October 5, 2020

We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function.

Key Points: 
  • We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function.
  • The program was developed by Brian Bigger, Ph.D., a professor of cell and gene therapy at the University of Manchester, U.K. Prof.
  • Both these factors make AVROBIO an excellent partner for our investigational Hunter syndrome gene therapy.
  • AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide.

Global Predictive and Presymptomatic Testing Industry (2020 to 2027) - Market Trajectory & Analytics

Retrieved on: 
Friday, October 2, 2020

DUBLIN, Oct. 2, 2020 /PRNewswire/ -- The "Predictive and Presymptomatic Testing - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, Oct. 2, 2020 /PRNewswire/ -- The "Predictive and Presymptomatic Testing - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.
  • The 142-page report presents concise insights into how the pandemic has impacted production and the buy side for 2020 and 2021.
  • Amid the COVID-19 crisis, the global market for Predictive and Presymptomatic Testing estimated at US$4.1 Billion in the year 2020, is projected to reach a revised size of US$7.7 Billion by 2027, growing at a CAGR of 9.6% over the period 2020-2027.
  • The U.S. Market is Estimated at $1.1 Billion, While China is Forecast to Grow at 12.7% CAGR
    The Predictive and Presymptomatic Testing market in the U.S. is estimated at US$1.1 Billion in the year 2020.

New Real-World Evidence Further Demonstrates the Clinical Utility of the Oncotype DX Breast Recurrence Score® Test, Beyond Clinical Risk Factors, in Guiding Chemotherapy Treatment Decisions in Patients with Early-stage Breast Cancer

Retrieved on: 
Friday, October 2, 2020

In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer.

Key Points: 
  • In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer.
  • Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS.
  • NOTE: Oncotype DX, Breast Recurrence Score, and Recurrence Score are trademarks or registered trademarks of Genomic Health, Inc.
  • Distribution of the 21-gene breast recurrence score in patients with primary breast cancer in germany.

Co-Diagnostics, Inc. Logix Smart Kit to be Used with Newly Authorized Saline Oral Rinse Collection

Retrieved on: 
Friday, October 2, 2020

Dwight Egan, CEO of Co-Diagnostics, remarked: "We are pleased that Co-Diagnostics technology is being used in the first FDA EUA for a test using a simple saline 30 second swish and gargle collection.

Key Points: 
  • Dwight Egan, CEO of Co-Diagnostics, remarked: "We are pleased that Co-Diagnostics technology is being used in the first FDA EUA for a test using a simple saline 30 second swish and gargle collection.
  • Co-Diagnostics, Inc., a Utah corporation, is a molecular diagnostics company that develops, manufactures and markets a state-of-the-art diagnostics technology.
  • The Company's technology is utilized for tests that are designed using the detection and/or analysis of nucleic acid molecules (DNA or RNA).
  • Readers of this press release are cautioned not to place undue reliance on any forward-looking statements.

New Real-World Evidence Further Demonstrates the Clinical Utility of the Oncotype DX Breast Recurrence Score® Test, Beyond Clinical Risk Factors, in Guiding Chemotherapy Treatment Decisions in Patients with Early-stage Breast Cancer

Retrieved on: 
Friday, October 2, 2020

In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer.

Key Points: 
  • In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer.
  • Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS.
  • NOTE: Oncotype DX, Breast Recurrence Score, and Recurrence Score are trademarks or registered trademarks of Genomic Health, Inc.
  • Distribution of the 21-gene breast recurrence score in patients with primary breast cancer in germany.

Global Gene Therapy Technologies, Markets & Companies Report 2020: Profiles of 202 Companies Presented with 266 Collaborations

Retrieved on: 
Thursday, October 1, 2020

Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described.

Key Points: 
  • Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described.
  • The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including gene editing and germline gene therapy.
  • The markets for gene therapy have been difficult to estimate as there only a few approved gene therapy products Gene therapy markets are estimated for the years 2019-2029.
  • Profiles of 202 companies involved in developing gene therapy are presented along with 266 collaborations.

Global Molecular Diagnostics Partnering Deal Terms and Agreements 2014-2020: Access to Headline, Upfront, Milestone and Royalty Data

Retrieved on: 
Thursday, October 1, 2020

DUBLIN, Oct. 1, 2020 /PRNewswire/ -- The "Global Molecular Diagnostics Partnering Terms and Agreements 2014 to 2020" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, Oct. 1, 2020 /PRNewswire/ -- The "Global Molecular Diagnostics Partnering Terms and Agreements 2014 to 2020" report has been added to ResearchAndMarkets.com's offering.
  • The Global Molecular Diagnostics Partnering Terms and Agreements 2014-2020 report provides a detailed understanding and analysis of how and why companies enter molecular diagnostics partnering deals.
  • The report takes the reader through a comprehensive review Molecular Diagnostics deal trends, key players, top deal values, as well as deal financials, allowing the understanding of how, why and under what terms, companies are entering Molecular Diagnostics partnering deals.
  • Global Molecular Diagnostics Partnering Terms and Agreements includes:
    Available deals are listed by:

Konica Minolta Precision Medicine (KMPM) Company Ambry Genetics Announces Comprehensive COVID-19 Screening, Management, and Testing Program To Help Schools and Businesses to Safely Reopen

Retrieved on: 
Thursday, October 1, 2020

The Ambry Genetics Comprehensive, Assessment, Risk, and Education (CARE) for COVID Program is designed to help identify and test individuals in need of coronavirus testing.

Key Points: 
  • The Ambry Genetics Comprehensive, Assessment, Risk, and Education (CARE) for COVID Program is designed to help identify and test individuals in need of coronavirus testing.
  • The Western Springs School District, located in a suburb of Chicago, Illinois, serves students from kindergarten to eighth grade.
  • The Green Alliance International will be using the CARE for COVID Program's screening and exposure questionnaire as part of their Gateway Entry Systems program.
  • For more information on risk factors for hereditary cancer, please visit cancer.gov's fact sheet on hereditary cancer and genetic testing.