#genetherapy

  1. Intellia Up on Upbeat Long-Term Angioedema Study Data

    By Zacks Intellia Therapeutics ' (NASDAQ: NTLA ) shares gained 10% on Jun 3 after the company reported encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for treating hereditary angioedema. Shares of NTLA continued to gain anothe
  2. Diabetic Neuropathy Market Size, Segmentation, Top Manufacturers and Forecast to 2031

    The global diabetic neuropathy market is on track for significant expansion in the coming years, fueled by the rising prevalence of diabetes and the need for effective management strategies. According to a recent industry report, the market, valued at USD 5.25 billion in 2022, is
  3. South Africa’s Afrigen targets local diseases with mRNA vaccines

    By Alexis Akwagyiram The Scoop KIGALI, Rwanda — South African biotech company Afrigen is collaborating with a Nobel Prize-winning scientist to develop groundbreaking gene therapy treatments that target diseases that have plagued Africa for decades. The Cape Town-based company is wor
  4. Merck KGaA Reports the Acquisition of Mirus Bio, Strengthening Upstream Portfolio

    By PharmaShots Shots: Merck KGaA has entered into a definitive agreement to acquire Mirus Bio (part of Gamma Biosciences) for an aggregate of $600M to strengthen its upstream portfolio. The transaction is expected to conclude in Q3’24 The acquisition adds Mirus Bio’s transfection
  5. Fractyl Health Highlights the Preclinical Data of its Pancreatic Gene Therapy at DDW 2024

    By PharmaShots Shots: Fractyl highlighted preclinical data of Rejuva pancreatic gene therapy candidate under the title, ‘Single-Dose GLP-1-Based Pancreatic Gene Therapy Maintains Weight Loss After Semaglutide Withdrawal and Reduces Hepatic Triglycerides in a Murine Model of Obesit
  6. Deaf Baby Girl Hears for First Time in 'Mind-Blowing' Gene Therapy Trial

    By Jesus Mesa Eighteen-month-old Opal Sandy was born completely deaf due to a rare congenital defect. No matter how loud or close a sound, she did not respond. Today, Opal's able to hear — unaided — after receiving a single dose of a groundbreaking gene therapy as part of an ongoi
  7. "In a country defined by irrational, runaway drug spending, many gene therapies live on a rare island of semi-rationality: They cost a fortune, but the return, sometimes, is babies who get to have childhoods, grow up and become adults." 🥹 @statnews 's J
  8. 6 Deaf Children Can Now Hear After a Single Injection

    Several gene therapies aim to restore a protein necessary for transmitting sound signals from the ear to the brain. By Emily Mullin Born deaf, the 1-year-old boy had never responded to sound or speech before. But after receiving an experimental treatment injected into one of his ea
  9. 2nd CRISPR Treatment Approved

    🧬FDA approves CRISPR-based Casgevy for beta thalassemia, expanding gene therapy options beyond sickle cell disease. A step forward in precision medicine! #CRISPR #GeneTherapy #HealthcareInnovation 🩸🔬🧪
  10. In a World First, a Patient’s Antibody Cells Were Just Genetically Engineered

    B cells are prolific producers of antibodies, but for the first time, scientists have modified them to make other proteins to counteract a serious genetic disease. By Emily Mullin Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins