CBER and CDER Directors Offer Insights on Overcoming Hurdles to Biomanufacturing Innovation at NIIMBL Annual Meeting

Among the ways that public/private collaborations could play a critical role in advancing cell and gene therapy (CGT) manufacturing would be helping create a non-proprietary set of adeno-associated virus (AAV) vectors, CBER Director Peter Marks proposed at the third annual meeting of the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), held in Crystal City, Virginia in late June.

Addressing the current challenges and opportunities in CGT product development in his introductory remarks at an expert panel session, Marks noted that their great promise depends on the ability to manufacture them.

A big hurdle, he pointed out, is that academic and research organizations are currently “starting from scratch” in developing unique viral or non-viral vectors for each new therapy. This lack of standardization creates significant inefficiencies – not only for the developers, who are “reinventing the wheel” each time, but also in the tech transfer process, and for regulators who then have to evaluate each new individual vector.

Noting that the AAV vector has become a “workhorse” in the gene therapy context, Marks suggested that, with a non-proprietary AAV vector, “we could make the technology more accessible to academics and innovators, streamlining the pre-clinical and manufacturing information needed.” Most important, he noted, would be the savings in time required to bring new, life-saving therapies to the patients who desperately need them.

The CBER director began his remarks by highlighting the “wave of transformative therapies” that is building, with four having now been approved by FDA.

In 2018, investigational new drug applications (INDs) to CBER for gene therapies almost doubled from the previous year – from 106 to 206 – and the projection is that 2019 will exceed the 2018 number by another 50%. In June 2019, CBER had over 800 active gene therapy INDs in its purview.

Marks then explained: ● the viral and non-viral vectors that are coming into play in these therapies ● the manufacturing challenges their deployment presents in the context of the disparate diseases and the capacities of the research community, and ● the role NIIMBL can play in addressing the challenges.

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