Genetic Disease Life Science RNA 
BioPharma Reporter
OCTOBER 17, 2024
Wave Life Sciences reports that it has successfully carried out RNA editing in two patients with alpha-1 antitrypsin deficiency, the first time this kind of clinical RNA editing has been completed in humans according to the company.
XTalks
DECEMBER 22, 2021
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.
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XTalks
JANUARY 5, 2022
Ayesha also discussed some of the predicted trends in the life sciences for 2022. These include the continued promise of RNA-based vaccines as well as therapeutics based on RNA editing approaches for the treatment of various conditions including rare genetic diseases.
XTalks
SEPTEMBER 13, 2021
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
Pharmaceutical Technology
JANUARY 12, 2023
The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
The Pharma Data
DECEMBER 8, 2020
December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”
XTalks
MAY 4, 2021
Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing.
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