Gene Editing is the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. It demonstrates great potential in gene research, gene therapy and genetic improvement. The Gene Editing concept includes major US-listed companies whose businesses are involved in gene editing.
Fabry gene therapy has almost no side effects and requires no steroids or immunosuppressive therapy at all so far.
This is rare in gene therapy.
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Company Overview
• Founded: 2013
• Founder: Dr. Jonathan Rothberg, a serial entrepreneur also known for inventing next-generation DNA sequencing (NGS).
• Headquarters: Guilford, Connecticut, USA
• ...
bluebird bio Presents Positive Long-Term Data On LYFGENIA™ (lovotobegligene autotemcel) Gene Therapy for Sickle Cell Disease at 66th American Society of Hematology (ASH) Annual Meeting and Exposition
Positive
94.7% of evaluable patients achieved complete resolution of severe VOEs
86.8% of evaluable patients achieved complete resolution of VOEs
100% success rate in pediatric patients (10/10) for VOE resolution
No stroke recurrence in patients with prior history through 9 y...
Beam Therapeutics Presents New Non-human Primate (NHP) Data Demonstrating Proof-of-concept for ESCAPE, a Non-genotoxic, Antibody-based Conditioning Approach to Treating Sickle Cell Disease, at American Society of Hematology (ASH) Annual Meeting
Beam Therapeutics (NASDAQ: BEAM) presented new data for its ESCAPE conditioning platform at the ASH Annual Meeting. The data demonstrated successful proof-of-concept in non-human primates (NHPs) for a non-genotoxic conditionin...
bluebird bio, Inc. (NASDAQ:BLUE) today announced updated data from patients with beta-thalassemia who require regular blood transfusions treated with be...
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