HAE
Haemonetics Corporation
stock NYSE

Problem with HAE is it is extremely crowded in terms of treatment options and there will be countless obstacles for NTLA to commercialize Lonzo when it comparable efficacy to other options.

I sadly didn’t find out about the IONS news until the stock price was around $13… I was pretty gutted when I read it. I maybe sold 20% - 25% of my stock in the $$15-$17 range… My average isn’t bad though at $9.9
I do think regardless of dollar amount HAE brings in it at least proves the technology that in vivo gene editing works (if approved). That alone is worth a lot if NTLA were to ever get bought out.
Yeahh TTR is definitely a weird one, the trials for ions and ntla are a mess as phase 1 and 2 didn’t have anyone on stabilizers but by the time phase 3 came around pfizer had their drug on the market which was now the placebo arm for phase 3. There is no true head on comparison trial, which I get why but it absolutely sucks trying to figure out a drugs true effect. There might not be much room left in treating ttr other than trying to diagnose it earlier.
On the other hand 90% reduced ttr leaves you with 10% left in the body compared to 20% left in the body with an 80% reduction. Thats a 100% difference in remaining TTR left in the body.

I was in complete agreement with you so I left NTLA at 15.63 after the Ionis news. However the more I researched it there is still a good chance on TTR. It's just going to take a while before anything materializes here. Stabilizers aren't great for the heart and if you can get off stabilizers with a silencer, people should theoretically live longer and at less costs to insurers.
The HAE program alone would be put the market cap of this company to \~$4B with the platform and data they have. Revenue from treating 500 patients a year at $2m per patient would be a huge savings, and countries with universal healthcare will jump on the chance to save money by curing people rather than treating the disease. The current market cap at $1.7B when companies like CRSP which are behind them with in-vivo being valued at $5B+ makes no sense.

I posted this on stocktwits and i’m copying my message to post here. This was in response to our drop from $18 to low $10’s recently.
Don’t get me wrong in bullish on NTLA but I looked into the IONS trial results and it may be why we are absolutely cratering. IONS drug reduced TTR by 80% and that showed no improvement in survival rate for patients already on modern TTR stabilizers… Our treatment works by turning off the gene that produces TTR and phase 1 showed roughly a 91% in overall TTR reduction.
l strong believe the HAE program but the IONS trial failure really made me loose faith in the TTR program.

Problem with HAE is it is extremely crowded in terms of treatment options and there will be countless obstacles for NTLA to commercialize Lonzo when it comparable efficacy to other options.

I sadly didn’t find out about the IONS news until the stock price was around $13… I was pretty gutted when I read it. I maybe sold 20% - 25% of my stock in the $$15-$17 range… My average isn’t bad though at $9.9
I do think regardless of dollar amount HAE brings in it at least proves the technology that in vivo gene editing works (if approved). That alone is worth a lot if NTLA were to ever get bought out.
Yeahh TTR is definitely a weird one, the trials for ions and ntla are a mess as phase 1 and 2 didn’t have anyone on stabilizers but by the time phase 3 came around pfizer had their drug on the market which was now the placebo arm for phase 3. There is no true head on comparison trial, which I get why but it absolutely sucks trying to figure out a drugs true effect. There might not be much room left in treating ttr other than trying to diagnose it earlier.
On the other hand 90% reduced ttr leaves you with 10% left in the body compared to 20% left in the body with an 80% reduction. Thats a 100% difference in remaining TTR left in the body.

I was in complete agreement with you so I left NTLA at 15.63 after the Ionis news. However the more I researched it there is still a good chance on TTR. It's just going to take a while before anything materializes here. Stabilizers aren't great for the heart and if you can get off stabilizers with a silencer, people should theoretically live longer and at less costs to insurers.
The HAE program alone would be put the market cap of this company to \~$4B with the platform and data they have. Revenue from treating 500 patients a year at $2m per patient would be a huge savings, and countries with universal healthcare will jump on the chance to save money by curing people rather than treating the disease. The current market cap at $1.7B when companies like CRSP which are behind them with in-vivo being valued at $5B+ makes no sense.

I posted this on stocktwits and i’m copying my message to post here. This was in response to our drop from $18 to low $10’s recently.
Don’t get me wrong in bullish on NTLA but I looked into the IONS trial results and it may be why we are absolutely cratering. IONS drug reduced TTR by 80% and that showed no improvement in survival rate for patients already on modern TTR stabilizers… Our treatment works by turning off the gene that produces TTR and phase 1 showed roughly a 91% in overall TTR reduction.
l strong believe the HAE program but the IONS trial failure really made me loose faith in the TTR program.

I have a slightly different take. To me CRSP is a short, as casgevy is somewhat of a commercial failure due to the cumbersome nature of the treatment. When I had last checked they didn’t have anything else even in phase 3. In my mind, NTLA has picked the perfect diseases for people to want to try editing. Both HAE and ATTR are some combination of terrifying and deadly. I can certainly imagine wanting a one and done therapy for those types of conditions. That fact combined with NTLA being the first in vivo treatment, makes me think this could be where CRISPR breaks through