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Revolo advances plans to tackle eosinophilic esophagitis with orphan drug tag 

Pharmaceutical Technology

The FDA has granted an orphan drug designation to Revolo’s ‘1104, with plans for a Phase IIb study later this year.

Drugs 130
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With European approval secured, BioMarin puts roughly $1.5M price tag on hemophilia gene therapy

Bio Pharma Dive

On a conference call, BioMarin executives revealed the anticipated price for Roctavian and the company's initial launch plans, which include pay-for-performance deals customized to different markets.

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Novartis-linked startup launches with technology designed to remove ‘destroy’ tags on helpful proteins

Bio Pharma Dive

Vicinitas and its protein-stabilizing research have attracted $65 million from a group of investors that includes a16z, Deerfield and GV.

Protein 258
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FDA approves Duchenne gene therapy with hefty $3.2m price tag

Pharmaceutical Technology

Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.

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#news #biotech Bluefin tuna tagged for the first time in UK waters with acoustic ‘residency’ tags

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Bluefin tuna tagged for the first time in UK waters with acoustic ‘residency’ tags.Bluefin tuna have been tagged with state-of-the-art acoustic tracking tags for the first time … Continue reading →

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Genprex’s Reqorsa gene therapy picks up orphan drug designation from FDA for SCLC

Pharmaceutical Technology

The latest tag adds to three fast track designations for Reqorsa, with the company initiating a Phase I/II trial in Q4 2023.

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#news #biotech Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug.Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug … Continue reading →

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