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Drug Development

A total of 168 posts are filed under Drug Development
New Bluebird Bio Data Shows Gene Therapy Promising for Thalessemia
Rare Daily Staff Bluebird Bio said data from its phase 1/2 Northstar study of its experimental LentiGlobin gene therapy in adolescents and… Continue Reading
Experimental Drug for “Elephant Man” Disease May Benefit Certain Cancers Too
Joseph Merrick, who was known as the Elephant Man, suffered from Proteus syndrome, a rare disorder that causes abnormal and disproportionate… Continue Reading
BridgeBio Pharma Launches CoA Therapeutics to Target Coenzyme-A for Rare Developmental Disorders
Rare Daily Staff BridgeBio Pharma said it launched CoA Therapeutics, a biopharmaceutical company that will develop small-molecules designed… Continue Reading
FDA Grants Rare Pediatric Disease Designation to Albireo’s Candidate for Rare Liver Disease
Rare Daily Staff The U.S. Food and Drug Administration granted Albireo Pharma’s lead candidate A4250 Rare Pediatric Disease designation… Continue Reading
BridgeBio Pharma Acquires Late-Stage Therapy for Ultra-Rare Disorder from Alexion
Rare Daily Staff BridgeBio Pharma said it has entered into an agreement with Alexion Pharmaceuticals to acquire ALXN1101, a synthetic… Continue Reading
FDA Approves Genentech’s Rituxan for Rare Autoimmune Condition
Rare Daily Staff The U.S. Food and Drug Administration approved Genentech’s Rituxan for adults with moderate to severe pemphigus vulgaris,… Continue Reading
Programming Stem Cells to Protect Neurons in ALS
BrainStorm Cell Therapeutics is developing innovative, autologous stem cell therapies for debilitating neurodegenerative diseases such as… Continue Reading
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