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- Spruce’s successful FDA talks plant seeds for Q4 filing of ultra-rare disease drugSpruce Biosciences has taken another step toward filing for FDA approval of its ultra-rare disease drug candidate, ticking off some of the manufacturing tasks needed to submit the long-awaited appl…Fierce Biotech · 10h ago↗ source
- Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findingsThe FDA has put a clinical hold on Regenxbio’s RGX-121 over asymptomatic spine MRI findings, derailing the biotech’s plans to refile for approval of the once-rejected gene therapy candidate.Fierce Biotech · 12h ago↗ source
- Ambros Therapeutics merges with Werewolf, raises $150M to fund phase 3 trial of rare disease drugAmbros Therapeutics announced an all‑stock reverse merger with Werewolf Therapeutics, accompanied by a $150 million private placement. The transaction will see the combined company operate under th…