December will serve as a milestone month for gene therapies.
The Gene Therapy Wave Is Here
The long-talked about wave of gene therapy approvals is beginning to reveal itself.
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The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
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Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
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The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
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PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.
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PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.
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Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.
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A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.