Fostering Gene Therapy Development [Part 1]: A Snapshot of FDA Guidance 2018

Gene therapy could well become the ‘new biotech’ in the life sciences industry, known to drive innovation and provide new, potent ways to treat complex diseases.
2017 was a progressive year with new gene therapies entering the US market. Trailblazing comes with its own set of advantages and limitations. To help understand the implications of developing such therapies from a regulatory, clinical, and commercial standpoint, a ‘Blueprint of Gene Therapies’ would be presented in PART 2 of the insights piece.
To set the rules of the road, albeit one less traveled and full of uncertainty, US FDA, released in July 2018, a draft guidance for the development of such therapies. This insight piece captures a snapshot of critical clinical data requirements for such therapies that would help mitigate risks at the time of drug approvals.

Key Takeaways

• Gene therapy development trends in the US and Europe.
• A snapshot of key clinical considerations for development of therapies for rare diseases, hemophilia, and retinal dystrophy.

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