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The pharmaceutical industry has largely endorsed the US Food and Drug Administration's (FDA) guidance on next-generation sequencing (NGS) for assessing the safety of human gene therapy products. However, they have requested clarification on several points, including off-target data, nomination methods, and additional information on how to assess these products following any manufacturing changes.

The US Food and Drug Administration (FDA) roadmap to reduce animal testing as a requirement for drug development has already made an impact after its first year, but the initiative faces future challenges in scaling and international harmonization, a group of experts wrote in a recent paper published in Stem Cell Research & Therapy.

As cell and gene therapies (CGTs) continue to advance, they have unique developmental features that may require innovation in a clinical trial setting, the authors of a recent paper explained.

The US Food and Drug Administration (FDA) has proposed guidance on the types of scientifically valid prior knowledge sponsors should consider when developing certain gene therapies (GT). The agency said the recommendations in the guidance are intended to improve review efficiency and help accelerate product development.

The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on chemistry, manufacturing, and controls (CMC) flexibilities for cell and gene therapy (CGT) products developed for biologics license applications (BLAs).

The US pharmaceutical industry expressed support for the US Food and Drug Administration’s (FDA) proposed framework for increasing access to treatments for ultra-rare diseases yet raised questions concerning various aspects of the program.

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing (NGS) methods in nonclinical studies to assess the safety risks associated with gene therapy products. This guidance aims to support the submission of investigational new drug applications (INDs) and biologics license applications (BLAs).

US Food and Drug Administration (FDA) officials credited recent advancements in regulatory science and flexibilities with enabling growth in the cell and gene therapy sector during a meeting on Thursday; however, a cell therapy expert said there is still reluctance from FDA and industry to enroll pediatric patients in clinical trials for such treatments.