therapeutics notes

NYu
4 min readFeb 11, 2020

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(filtered which names in this list is worth reading, deleted all the weak charts)
author’s opinions only

study why it went 10–170

48–108

1.72–72–42

8–84–32–50

1–32–15–19

1.72–120–70–105

13–44–20–34

5–125–90

Intellia Therapeutics

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products

  • On track to submit IND application for lead candidate, NTLA-2001 for transthyretin amyloidosis, in mid-2020 and dose first patients in 2H 2020
  • Nominated NTLA-5001 for the treatment of acute myeloid leukemia as first WT1-TCR-directed engineered cell therapy development candidate; plan to submit IND in 1H 2021
  • Announced third development program, which will be for treatment of hereditary angioedema; expects to nominate a development candidate in 1H 2020
  • Ended 2019 with strong cash position of $284 million; cash runway extended through YE 2021
  • 2020 will be a significant year for Intellia, as we execute on our full-spectrum strategy. With milestones anticipated across our pipeline, we are making important progress towards the development of curative treatments for severe diseases. In particular, we expect to dose ATTR patients with the first-ever systemically delivered CRISPR/Cas9-based therapy this year, and we are beginning IND-enabling activities for our newly announced development candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy, for treatment of AML,” said Intellia President and Chief Executive Officer, John Leonard, M.D. “We are focused on developing a robust platform with modular genome editing capabilities that enable a fast and reproducible path to development. Today’s update reflects this strategy, and it also features the announcement of our third development program, an in vivo knockout approach for HAE. Importantly, this program leverages the infrastructure and insights from NTLA-2001 and underscores our ability to produce a rapid succession of new clinical candidates. We are excited by the strong momentum across our diverse pipeline and look forward to providing updates on our development programs in the upcoming year.”
  • ( https://finance.yahoo.com/news/intellia-therapeutics-highlights-recent-progress-123010208.html)

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NYu
NYu

Written by NYu

I’ve been trading stocks for awhile but understandably I’m likely to trade or invest for the rest of my life. Here’s my way of thinking about things

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