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To set the rules of the road, albeit one less traveled and full of uncertainty, US FDA, released in July 2018, a draft guidance for the development of such therapies.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


Sameera Singh

Delivery Lead, Corporates & Consulting

Sameera Singh is life sciences practice lead within the corporates and consulting vertical at Acuity Knowledge Partners. She has over seven years of work experience in life-sciences consulting, business research, and marketing. She has hands-on experience in strategic projects, such as therapeutic area assessment, competitive intelligence, disease insights, and brand management. Sameera is an MBA (Pharmaceutical Management) gold medalist and holds a bachelor’s degree in Pharmaceutical Sciences.

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

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