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“My father-in-law, Ben – or as Pam calls him, Benny – lost his battle with AML far too soon, which makes me especially proud to work for a company that is striving to provide curative treatments for that disease, among others.”

Stacey
Associate Director, Finance
Pam, whose husband passed away from acute myeloid leukemia

“Benny had a way of making everything OK. I miss his smile.”

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The 2020 Nobel Prize in Chemistry has been awarded “for the development of a method for genome editing.”

Jennifer Doudna, Ph.D.
Professor of Chemistry and Molecular and Cell Biology, University of California; Chemistry Nobel Laureate, 2020; Co-founder, Intellia Therapeutics

“Have you ever been in an environment full of intellectual people? Now combine that with CRISPR/Cas9. Greatness at full potential.”

Denisse
Sr. Associate Scientist, Mass Spectrometry

“UKATPA’s objective is to raise awareness and give support to patients, caregivers and health care professionals.”

Vince
UKATPA Treasurer

Vince and David, friends and colleagues living with ATTR

“The biggest surprise to us was how many people have amyloidosis and don’t have anyone to talk to.”
– David, UKATPA Secretary

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Each one of us together for patients.

Using a modular approach, we optimize the necessary therapeutic components so they can be used and combined in our in vivo and ex vivo programs to develop product candidates addressing a broad range of diseases. As exemplified by our current programs, this approach will allow us and our partners to build a full-spectrum genome editing company, with a pipeline of products that can treat a broad range of genetic and oncological diseases.

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Overview

As a pioneer in the development of CRISPR/Cas9-based therapies, we are rapidly moving our development candidates towards the clinic so we can make them available to patients suffering severe and life-threatening disorders.

Our company’s unique strengths include our CRISPR/Cas9 system improvements, modular lipid nanoparticle delivery system and our determined focus on product development.

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