CRISPR Therapeutics AG
http://www.crisprtx.com/
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Latest From CRISPR Therapeutics AG
Arrowhead’s Focus On Plozasiran Intensifies Competition With Ionis
Arrowhead’s in-house, late-stage cardiovascular disease programs will center on triglyceride-lowering plozasiran while it seeks a partner to fund Phase III for cholesterol-reducing zodasiran.
‘Threading The Needle:’ Finding A Regulatory Foundation For Ultra Rare Disease Development
Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.
EHA Preview: Agios Takes Spotlight In Thalassemia
The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.
Gene Therapy Q1 Roundup: The Struggle Is Real
Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.
Company Information
- Industry
- Pharmaceuticals
-
Pharmaceuticals
- Drug Delivery
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Biotechnology
- Gene Therapy, Cell Therapy
- Large Molecule
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Drug Discovery Tools
- Genomics-Proteomics
- Other Names / Subsidiaries
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- Casebia Therapeutics
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