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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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US FDA approves Krystal Biotech’s Vyjuvek for DEB

Pharmaceutical Technology

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. We offer our sincere gratitude to DEB patients, caregivers, investigators, US regulators and our employees who made this approval possible. “For

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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FDA approves first gene therapies for sickle cell disease (with a warning)

Drug Discovery World

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Filsuvez Topical Gel Gets FDA Approval for Epidermolysis Bullosa

XTalks

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. The clinical study also highlighted the favorable tolerability profile of Filsuvez. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector. Specifically, 41.3 percent of cases.

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Wainua (Eplontersen) Now FDA-Approved for Rare Disease ATTRv-PN

XTalks

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Polydefkis, professor of Neurology at Johns Hopkins University School of Medicine and an investigator in the Phase III NEURO-TTRansform study on which Wainua’s FDA approval was grounded on, in the news release.

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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.