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Krystal Biotech receives FDA approval for DEB topical gene therapy

Pharmaceutical Technology

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. Topic sponsors are not involved in the creation of editorial content.

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STAT+: FDA approves first treatment for skin condition that causes persistent wounds, a redosable gene therapy

STAT

The therapy, known as Vyjuvek and made by Krystal Biotech, will soon be available for patients with dystrophic epidermolysis bullosa, a genetic disease that causes painful blisters and persistent wounds. Patients with the condition live covered in bandages to try to stop new blisters from forming and to dress their wounds.

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Test – STAT+ Only virtual event

STAT

STAT+ subscribers get access to exclusive virtual pop-up conversations with STAT reporters on key stories of the moment, and biotech, pharma and health tech leaders on breakthroughs.   Please note: Speakers and topics are subject to change! Register below for the upcoming conversation.

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Listen: Whither PhRMA, Alzheimer’s treatment data revealed, and the first fecal microbiome drug approval

STAT

We cover all these topics and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. The first FDA-approved fecal microbiome drug. And a stinging defeat for Big Pharma’s lobbying arm.

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PHARMAP 2023 to explore latest pharma trends

Express Pharma

Continuous manufacturing The topic of continuous manufacturing is also discussed in frames of PHARMAP 2023. Pharma biotech Pharma biotech is a rapidly expanding field focusing on the production of biopharmaceuticals that could lead to cures for many diseases. “I I think there are various different ways of development.

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First redosable gene therapy approved

European Pharmaceutical Review

The gene therapy is a topical gel that restores functional copies of the COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing. DEB affects the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene, according to Krystal Biotech.

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Oculis enters merger deal with European Biotech Acquisition

Pharmaceutical Technology

Oculis has signed a definitive business combination agreement with special purpose acquisition company (SPAC) European Biotech Acquisition (EBAC). OCS-02 is a topical anti-TNF alpha monoclonal antibody fragment, while OCS-05 is an activator of serum-glucocorticoid kinase 2.