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A Visual Guide to Gene Delivery

Codon

Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream.

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Synedgen Names Dr. Laura Saward CEO, Dr. Kenton Gregory CMO

The Pharma Data

Her deep experience in managing government contracts, navigating regulatory pathways, and driving product development to FDA approval makes her an ideal leader. government agencies, Synedgen is well-positioned to become a leading force in both biodefense and commercial healthcare innovation.

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A Temporary Extension for the Rare Pediatric Disease Priority Review Voucher with a Longer-Term Extension in Sight?

FDA Law Blog: Biosimilars

A very important aspect of the program is that it costs FDA, and the government, no additional dollars, though we do not wish to minimize the additional resources a priority review demands over the standard review that such an application might otherwise receive.

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Educating Patients about Generic Drugs: Strategies for Success

Drug Patent Watch

Explain the FDA Approval Process Many patients are unaware of the rigorous approval process generic drugs must undergo. Educate them about the FDA’s role in ensuring the safety and efficacy of generic medications. These anecdotes can help alleviate fears and build confidence in generic medications.

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Even in “Unprecedented” Times, There is Precedent

FDA Law Blog: Biosimilars

The HHS Secretary intervened in an FDA approval decision. The court ruled the agency’s action was arbitrary and capricious and entered a mandatory injunction ordering FDA to make the drug available. Those cases evaluate when state and local governments can act in ways that may be different from the federal government.

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Vertex taking ‘flexible’ approach to pricing talks for experimental CRISPR therapy

BioPharma Drive: Drug Pricing

The company is discussing various payment models for its sickle cell treatment with government and commercial insurers ahead of an FDA approval decision later this year.

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Vanda-lay Litigation Industries, Inc.: Taking Stock of Vanda Pharmaceuticals, Inc.’s Big Bets on Petitioning and Litigation Against FDA and the Federal Government

FDA Law Blog: Biosimilars

District Court for the District of Columbia (“DDC”) against FDA alleging that the Agency’s structure of NDA review is unconstitutional (by our count the 31st Vanda litigation against FDA or another government entity in the last five years, including appeals). In January 2024, the U.S. VANDA PHARMACEUTICALS, INC.