Sentiment Analysis of the earnings transcript to help figure out if there are any bullish or bearish sentiments that could be gathered from it. We're doing ML and AI based analysis on the earnings call to get some more insights.
Please consider a small donation if you think this website provides you with relevant information
| Statement |
|---|
| But again, we're in a very good position to adapt and frankly, to use and benefit from both the infrastructure that's being built as well as the experiences that the others have |
| Because as I say our technology is very powerful, our IP is very strong and knowhow is very strong |
| So that was in a very good position to actually also learn benefit from the significant infrastructure build that Vertex and Bluebird will be carrying out, as well as actually learning from as the payer landscape evolves and develops and obviously as we have our own interactions |
| We have to keep driving on that, and I'm happy to say, and I should have said it in the strengths that we have a very strong leadership team now with some very seasoned veterans who know how to develop, get across regulatory approval and commercialized medicines, including our Chief Commercial Officer, Caren Deardorf |
| So we actually feel very good about the progress we're making in getting ready for BLA and commercial launch |
| From a point of view of the strengths of the company, we have very strong science |
| We also have very strong chemistry around guide RNA and obviously a robust CMC organization with a particular focus, currently on bringing reni-cel and getting ready for commercialization |
| So in fact, notwithstanding sort of that holiday, the end of the year, we really have done, executed very well with regard to dosing and recruitment |
| This patient's doing very well |
| Can you discuss your funding, today? How much cash did you have as of year-end and how long will that last you? Erick Lucera We think we're in a really strong financial position, not only with the $427 million we ended the year at which gets us into 2026, but also the ability to monetize that IP portfolio as a source of non-dilutive capital in multiple ways |
| And so I think we're very happy with the progress we've made there |
| And actually, I should say we're very happy with the enthusiasm we have patients lining up |
| And I would finally say that with a head-to-head of our gamma globin target versus a BCL11a target we see, we expected to see and in the non-clinical or preclinical side, we saw that not only did we get very robust fetal hemoglobin expression and it was durable, but we actually saw better red cell output, better red cell longevity |
| I mean, we have -- we're in a great position, we're a fast follower with a potentially differentiated product, potentially be best in class with that |
| And in fact, activations are going very well for sites |
| We will upregulate fetal hemoglobin and we're actually seeing that very robustly |
| We are progressing the program nicely and actually very happy with the data we've seen to date |
| So now we're not just building the numbers of patients, but we're actually building the duration of follow-up, which actually again will continue to reinforce our confidence in the durability of the therapy |
| Where are you in your manufacturing work in terms of the commercial process as well as scale it? Gilmore O'Neill So we're actually in a very good place right now |
| Just a little over a year ago, we outlined a new strategy with three pillars, advancing our lead asset reni-cel and that has been going very well on the execution side with some regulatory alignment, et cetera, as we drive towards BLA and commercialization |
| So as that evolves and collects, I would anticipate that we're actually going to have more confidence, certainly at the robustness of both the fetal hemoglobin, but actually the overall, well the total fetal hemoglobin expression |
| From the point of the state of the company, I think things are actually are moving very well |
| I think it shows a lot of enthusiasm on the part of investigators, patients and frankly the institutions where the research is going on |
| So we actually had -- we were very happy with the conversation with the FDA and let me preface it by saying that that's against the background of receiving RMAT last October, which enables us to have more frequent meetings with shorter timelines between true requests and meeting and to meet actually with senior officials within the agency |
| Any update on that patient? Gilmore O'Neill Yes, so that patient is actually doing very well |
| And from my standpoint, as a CFO, that is a key reason why I came to the company was to have that extra ability to raise money that nobody else really has |
| We don't want to sort of pile into very competitive spaces with a lot of other players, but we actually also want to ensure that we maximize the property of technical, clinical and regulatory success as we build a pipeline that is going to be both meaningful for patients and commercially viable |
| I think we have an opportunity over the next year or so, or next year or so to actually see how it evolves and really adapt |
| The second pillar is around building and focusing our pipeline efforts on in vivo, and we are happy to be on track for a preclinical in vivo proof-of-concept this year |
| And we have increased enrollment from 27 at the end of last year to 40 |
| Statement |
|---|
| The challenges really are around helping the world recognize the true transformative value of gene editing |
| So where they were anemic before, they're no longer anemic |
| But I think your hypothesis is plausible |
| Forgive me for misrepresenting you, Phil |
Please consider a small donation if you think this website provides you with relevant information