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Rebecca Ahrens Nicklas Shares FDA Interactions on Interventional Genetics with The Rare Disease Community
Rebecca Ahrens Nicklas/LinkedIn
Nov 4, 2025, 08:53

Rebecca Ahrens Nicklas Shares FDA Interactions on Interventional Genetics with The Rare Disease Community

Rebecca Ahrens Nicklas, Rare disease physician-scientist at the Children’s Hospital of Philadelphia, shared on LinkedIn:

”Excited to share our vision for how we scale personalized gene editing therapies.

We need formal clinical trials to critically evaluate these novel treatments and (hopefully) obtain formal approvals.

One major step to make this happen is regulatory innovation.

We thought it was essential that we share our FDA interactions with the rare disease community so that we can all learn together about how we will build the field of interventional genetics.”

Read the full article in AJHG.

Article: How to create personalized gene editing platforms: Next steps toward interventional genetics

Authors: Rebecca Ahrens Nicklas, Kiran Musunuru

Rebecca Ahrens Nicklas Shares FDA Interactions on Interventional Genetics with The Rare Disease Community

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AJHG bleeding disorders FDA gene editing platforms Health Hematology Hemostasis Hemostasis Today interventional genetics Kiran Musunuru Medicine Rare Diseases Rebecca Ahrens-Nicklas
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