Specialist
Professor at UC Davis School of Medicine
Agenda
- CRISPR technology market update and competitive landscape developments, highlighting Editas (NASDAQ: EDIT), Intellia (NASDAQ: NTLA), CRISPR Therapeutics (NASDAQ: CRSP) and Beam (NASDAQ: BEAM)
- Safety and mechanism – main points of differentiation and avenues for growth or improvement
- Q4 2021 outlook and broader CRISPR pipeline
Questions
1.
In our previous Interview [see CRISPR Technology – Editas, Intellia & CRISPR Therapeutics – 18 March 2020], we discussed the concept of CRISPR treatment being inserted directly into the body. Since then, this has reached additional milestones and become more of a reality. How have these dynamics played out over the past year or so and could you break down some of the biggest achievements?
2.
How does the progress in in vivo CRISPR compare to where we are with ex vivo? Which companies do you believe are leading the way with in vivo and ex vivo?
3.
In June 2021, Intellia Therapeutics and its partner Regeneron announced positive in-term clinical data from a phase 1 trial evaluating NTLA-2001, which is a potentially curative therapy in patients with ATTR [transthyretin amyloidosis] and polyneuropathy. What do these results mean for in vivo CRISPR genome editing in humans, as well as for Intellia? What kind of opportunity do you think exists for this candidate given the data so far?
4.
What are some notable manufacturing considerations around the use of lipid nanoparticles? How do they compare to the manufacture of viral vectors?
5.
What more needs to be done to make the switch over to lipid nanoparticles a reality, given AAV [adeno associated virus] vectors’ toxicity risks and production challenges? Over what timeline would you expect this to take place?
6.
What should we monitor from the next set of data to come out of Intellia’s NTLA-2001 trial? What does this candidate need to prove to be successful? What questions would you like to see answered?
7.
What challenges might Intellia experience as it aims to bring its NTLA-2001 candidate to market? Could you discuss the conversations we’ve heard around pricing, how high it could be and how it could pose a challenge for the company?
8.
How are you assessing the work Intellia is doing on the ex vivo side and how does it compare to competitors? Would you like to highlight anything in particular about its efforts here?
9.
How are you assessing Editas’s efforts in LCA [Leber congenital amaurosis]? Do you believe there’s room for the company’s candidate in the market, especially given the disappointing sales of Spark Therapeutics’ FDA-approved product, Luxturna?
10.
How are you assessing the players working towards sickle cell disease gene therapies? I understand the market is quite crowded, so how do players such as CRISPR Therapeutics and Editas approach it? How would you expect their data to potentially differ?
11.
Which of the players we’ve discussed are you most excited about?
Gain access to Premium Content
Submit your details to access up to 5 Forum Transcripts or to request a complimentary 48 hour week trial
The information, material and content contained in this transcript (“Content”) is for information purposes only and does not constitute advice of any type or a trade recommendation and should not form the basis of any investment decision.This transcript has been edited by Third Bridge for ease of reading. Third Bridge Group Limited and its affiliates (together “Third Bridge”) make no representation and accept no liability for the Contentor for any errors, omissions or inaccuracies in respect of it. The views of the specialist expressed in the Content are those of the specialist and they are not endorsed by, nor do they represent the opinion of, Third Bridge. Third Bridge reserves all copyright, intellectual and other property rights in the Content. Any modification, reformatting, copying, displaying, distributing, transmitting, publishing, licensing, creating derivative works from, transferring or selling any Content is strictly prohibited
