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Rare Daily Staff Rare disease drug developer Acer Therapeutics said it is raising $2.675 million in a […]
Read moreQuince Gets Unsolicited Acquisition Offer from Echo Lake Capital
Rare Daily Staff Rare disease therapeutics developer Quince Therapeutics confirmed that it received an unsolicited offer to […]
Read moreNovartis Reports Long-Term Efficacy of Zolgensma
Rare Daily Staff Novartis presented new data from two-long terms studies of Zolgensma, its one-time gene therapy […]
Read moreFDA’s Marks Calls for Providing Accelerated Approval Path for Gene Therapies
Rare Daily Staff Peter Marks, director of the U.S. Food and Drug Administration’s Center for Biologics Evaluation […]
Read moreEurodis, Ahead of EC Release of Revisions to Orphan Regulations, Comments on Shortcoming of Leaked Draft
Rare Daily Staff The European rare disease organization Eurodis, responding to a leaked draft of soon to […]
Read moreGeneDx Reports Progress on Study to End Rare Disease Diagnostic Odyssey
Rare Daily Staff Initial data and analysis from GUARDIAN, a research study recently launched by Columbia University […]
Read moreFDA Grants RMAT Designation to Intellia’s Experimental Treatment for Hereditary Angioedema
Rare Daily Staff The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Intellia […]
Read moreNovartis and Biogen Terminate Collaborations with Sangamo for Neurodevelopmental Disorders
Rare Daily Staff Sangamo Therapeutics said that Novartis and Biogen both have terminated collaboration and licensing agreements […]
Read moreArrowhead Receives FDA Fast Track Designation for ARO-APOC3 as a Treatment for FCS
Rare Daly Staff The U.S. Food and Drug Administration has granted Fast Track designation to Arrowhead Pharmaceuticals’ […]
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