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Gene therapy faces fresh uncertainty as two more top FDA officials depart

Bio Pharma Dive

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.

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Entering the era of prenatal gene therapies 

Pharmaceutical Technology

As scientists explore treating genetic disorders before birth, the promise of foetal gene therapy is growingbut there are hurdles to overcome.

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Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

XTalks

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency.

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FDA to use new review tool on Sarepta’s gene therapy work

Bio Pharma Dive

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.

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Making the Case for Case Management for Cell and Gene Therapies

As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements.

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Prasad’s FDA appointment pressures cell and gene therapy stocks

Bio Pharma Dive

A vocal opponent of his predecessor Peter Marks, Vinay Prasad will now lead the office tasked with reviewing some genetic medicines, adding more uncertainty to an already struggling field of research.

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SpliceBio secures $135m for gene therapy development

Pharmaceutical Technology

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007, its gene therapy candidate for Stargardt disease. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Credit: Hryshchyshen Serhii/Shutterstock.