Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model
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Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model

According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…

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Idiopathic Pulmonary Fibrosis Treatment Does Well In Preclinical Trial
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Idiopathic Pulmonary Fibrosis Treatment Does Well In Preclinical Trial

Cynata Therapeutics Limited has recently announced the results from their preclinical trial of induced pluripotent stem cell (iPSC)-derived Cymerus™ mesenchymal stem cells (MSCs), according to BioSpace. They are testing these…

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Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria: Marketing Applications Submitted
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Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria: Marketing Applications Submitted

  Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…

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New Reduced-Intensity Conditioning for HSCT Found Beneficial for 20 Rare Diseases

A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…

Continue Reading New Reduced-Intensity Conditioning for HSCT Found Beneficial for 20 Rare Diseases
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