@RNAiAnalyst
@rnaianalyst.bsky.social
aka Dirk Haussecker. Biotech enthusiast with expertise in sequence-directed therapeutics (RNAi, ASO, ADAR, CRISPR...) enjoying drug development and trying to make a buck along the way.
#markets these should be 2 quite volatile trading weeks with 2-week-Trump having to decide on Iran and tariffs.
Then we could have a little FDA OTP Verdun-firing scare in CGT space on top, or maybe investors have gotten used to it by now.
Then we could have a little FDA OTP Verdun-firing scare in CGT space on top, or maybe investors have gotten used to it by now.
June 19, 2025 at 7:38 PM
#markets these should be 2 quite volatile trading weeks with 2-week-Trump having to decide on Iran and tariffs.
Then we could have a little FDA OTP Verdun-firing scare in CGT space on top, or maybe investors have gotten used to it by now.
Then we could have a little FDA OTP Verdun-firing scare in CGT space on top, or maybe investors have gotten used to it by now.
Eli Lilly acquires $VERV- official now. finance.yahoo.com/news/lilly-a...
Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk
Eli Lilly and Company (NYSE: LLY) and Verve Therapeutics, Inc. (Nasdaq: VERV), a Boston-based clinical-stage company developing genetic medicines for cardiovascular disease, today announced a definiti...
finance.yahoo.com
June 17, 2025 at 10:56 AM
Eli Lilly acquires $VERV- official now. finance.yahoo.com/news/lilly-a...
#CRISPR Stocks in Wake of $VERV Acquisition by Eli Lilly (blog): rnaitherapeutics.blogspot.com/2025/06/cris...
$CRSP $LLY $NTLA $BEAM $PRME $MGX $IONS
$CRSP $LLY $NTLA $BEAM $PRME $MGX $IONS
CRISPR Stocks in Wake of Verve Therapeutics Acquisition by Eli Lilly
Last night, news broke that pharma giant Eli Lilly was in talks to acquire Verve Therapeutics. After a 3-year lull in major CRISPR deal...
rnaitherapeutics.blogspot.com
June 17, 2025 at 10:47 AM
#CRISPR Stocks in Wake of $VERV Acquisition by Eli Lilly (blog): rnaitherapeutics.blogspot.com/2025/06/cris...
$CRSP $LLY $NTLA $BEAM $PRME $MGX $IONS
$CRSP $LLY $NTLA $BEAM $PRME $MGX $IONS
#VerveTherapeutics CEO did what was best for the medicine, but also good for shareholders.
Though Eli Lilly will get to buy them for too cheap, investors can reinvest quickly into $NTLA $PRME and maybe $MGX for hopefully another easy double. #CRISPR
Though Eli Lilly will get to buy them for too cheap, investors can reinvest quickly into $NTLA $PRME and maybe $MGX for hopefully another easy double. #CRISPR
June 17, 2025 at 2:15 AM
#VerveTherapeutics CEO did what was best for the medicine, but also good for shareholders.
Though Eli Lilly will get to buy them for too cheap, investors can reinvest quickly into $NTLA $PRME and maybe $MGX for hopefully another easy double. #CRISPR
Though Eli Lilly will get to buy them for too cheap, investors can reinvest quickly into $NTLA $PRME and maybe $MGX for hopefully another easy double. #CRISPR
#DyneTherapeutics myotonic dystrophy type I regulatory update likely coming tomorrow.
These pathway updates have been major biotech stock movers recently. If no big move on day 1, following days usually strong upward momentum.
investors.dyne-tx.com/news-release...
These pathway updates have been major biotech stock movers recently. If no big move on day 1, following days usually strong upward momentum.
investors.dyne-tx.com/news-release...
investors.dyne-tx.com
June 16, 2025 at 9:45 PM
#DyneTherapeutics myotonic dystrophy type I regulatory update likely coming tomorrow.
These pathway updates have been major biotech stock movers recently. If no big move on day 1, following days usually strong upward momentum.
investors.dyne-tx.com/news-release...
These pathway updates have been major biotech stock movers recently. If no big move on day 1, following days usually strong upward momentum.
investors.dyne-tx.com/news-release...
$NTLA-2002 More and More Looks Like a Cure for HAE (blog): rnaitherapeutics.blogspot.com/2025/06/ntla...
Jaw-dropping efficacy/safety. Could totally disrupt HAE market, including on-demand.
#HAE $IONS $KALV
Jaw-dropping efficacy/safety. Could totally disrupt HAE market, including on-demand.
#HAE $IONS $KALV
NTLA-2002 More and More Looks Like a Cure for HAE
When Intellia Therapeutics set out to develop a CRISPR-based prekallikrein knockout for hereditary angioedema (HAE), it was as a compelling ...
rnaitherapeutics.blogspot.com
June 15, 2025 at 4:56 PM
$NTLA-2002 More and More Looks Like a Cure for HAE (blog): rnaitherapeutics.blogspot.com/2025/06/ntla...
Jaw-dropping efficacy/safety. Could totally disrupt HAE market, including on-demand.
#HAE $IONS $KALV
Jaw-dropping efficacy/safety. Could totally disrupt HAE market, including on-demand.
#HAE $IONS $KALV
#uniQure hires away Kylie O’Keefe from #Huntington's competitor #PTCTherapeutics, planning for AMT-130 launch.
Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon.
So do I.
finance.yahoo.com/news/uniqure...
Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon.
So do I.
finance.yahoo.com/news/uniqure...
uniQure Announces Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer
~ Proven biotech executive to lead commercialization of AMT-130 in Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, June 11, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene ...
finance.yahoo.com
June 11, 2025 at 2:21 PM
#uniQure hires away Kylie O’Keefe from #Huntington's competitor #PTCTherapeutics, planning for AMT-130 launch.
Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon.
So do I.
finance.yahoo.com/news/uniqure...
Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon.
So do I.
finance.yahoo.com/news/uniqure...
What we've learned since #IntelliaTherapeutics liver enzyme disclosure 2 weeks ago:
1) late response (week 4-5)...unlikely LNP tox
2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis)
3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
1) late response (week 4-5)...unlikely LNP tox
2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis)
3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
June 10, 2025 at 2:37 PM
What we've learned since #IntelliaTherapeutics liver enzyme disclosure 2 weeks ago:
1) late response (week 4-5)...unlikely LNP tox
2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis)
3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
1) late response (week 4-5)...unlikely LNP tox
2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis)
3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
Worth repeating:
It is unfair to blame the #pharmaceutical industry for the poor #health of US population. It actually makes sense: more $ spent when people are sick.
What needs to be addressed is what makes them sick in 1st place: poor nutrition, having to take a car to get to cross a road.
It is unfair to blame the #pharmaceutical industry for the poor #health of US population. It actually makes sense: more $ spent when people are sick.
What needs to be addressed is what makes them sick in 1st place: poor nutrition, having to take a car to get to cross a road.
June 10, 2025 at 9:14 AM
Worth repeating:
It is unfair to blame the #pharmaceutical industry for the poor #health of US population. It actually makes sense: more $ spent when people are sick.
What needs to be addressed is what makes them sick in 1st place: poor nutrition, having to take a car to get to cross a road.
It is unfair to blame the #pharmaceutical industry for the poor #health of US population. It actually makes sense: more $ spent when people are sick.
What needs to be addressed is what makes them sick in 1st place: poor nutrition, having to take a car to get to cross a road.
MAHA Vows to Disrupt Sick-Care Model in Favor of Cures (blog): rnaitherapeutics.blogspot.com/2025/06/maha...
#CRISPR #cures #genetherapy #FDA $NTLA $VERV $BBIO 💊
#CRISPR #cures #genetherapy #FDA $NTLA $VERV $BBIO 💊
MAHA Vows to Disrupt Sick-Care Model in Favor of Cures
There is now overwhelming evidence that the new US healthcare administration is serious in its mission of making its citizens inherently hea...
rnaitherapeutics.blogspot.com
June 9, 2025 at 6:43 PM
MAHA Vows to Disrupt Sick-Care Model in Favor of Cures (blog): rnaitherapeutics.blogspot.com/2025/06/maha...
#CRISPR #cures #genetherapy #FDA $NTLA $VERV $BBIO 💊
#CRISPR #cures #genetherapy #FDA $NTLA $VERV $BBIO 💊
I bought a German military stock 3 months ago for my kids' future-oriented #portfolio. I checked today: that stock is 80% up.
#biotech investing is hard.
#biotech investing is hard.
June 6, 2025 at 12:56 PM
I bought a German military stock 3 months ago for my kids' future-oriented #portfolio. I checked today: that stock is 80% up.
#biotech investing is hard.
#biotech investing is hard.
Breaking...2nd Patient with Chronic Granulomatous Disease Successfully Treated with #PrimeEditing (blog): rnaitherapeutics.blogspot.com/2025/06/2nd-...
$PRME $BEAM #CRISPR
$PRME $BEAM #CRISPR
2nd Patient with Chronic Granulomatous Disease Successfully Treated with Prime Editing
Prime editing researchers are so excited about the technology that sponsor Prime Medicine is unable to control the newsflow on their CGD tri...
rnaitherapeutics.blogspot.com
June 6, 2025 at 9:19 AM
Breaking...2nd Patient with Chronic Granulomatous Disease Successfully Treated with #PrimeEditing (blog): rnaitherapeutics.blogspot.com/2025/06/2nd-...
$PRME $BEAM #CRISPR
$PRME $BEAM #CRISPR
#PrimeMedicine CEO said they'll complete dosing of cohort 1 in CGD trial (2-3 patients).
I believe this could be enough to get approval under new #FDA. That would be worth ~$150M currently.
I believe this could be enough to get approval under new #FDA. That would be worth ~$150M currently.
June 5, 2025 at 8:45 PM
#PrimeMedicine CEO said they'll complete dosing of cohort 1 in CGD trial (2-3 patients).
I believe this could be enough to get approval under new #FDA. That would be worth ~$150M currently.
I believe this could be enough to get approval under new #FDA. That would be worth ~$150M currently.
#FDA Dr OZ at #CBER listening session had interesting idea wrt how to pay for $2M Casgevy-type drugs.... #reinsurance, socialize cost so that individual insurance companies do not feel disadvantaged having rare disease patients.
June 5, 2025 at 8:06 PM
#FDA Dr OZ at #CBER listening session had interesting idea wrt how to pay for $2M Casgevy-type drugs.... #reinsurance, socialize cost so that individual insurance companies do not feel disadvantaged having rare disease patients.
#FDA HHS secretary Kennedy loves #CRISPR and wants US to be competitive!
www.youtube.com/watch?v=x6Lz...
www.youtube.com/watch?v=x6Lz...
Grade 4 Liver Enzyme Elevation in Intellia’s Phase 3 ATTR Amyloidosis Trial
Last night, Intellia filed a material event report ( 8-k ) with the SEC. In there, they revealed a case of very high, grade 4 liver enzyme...
rnaitherapeutics.blogspot.com
June 5, 2025 at 3:44 PM
#FDA HHS secretary Kennedy loves #CRISPR and wants US to be competitive!
www.youtube.com/watch?v=x6Lz...
www.youtube.com/watch?v=x6Lz...
Another must-watch podcast from the 'new' #FDA. Anybody still in camp that Makary and Prasad are bad for #innovation in drug development seriously needs to re-think. In addition to words, now plenty of action to support:
x.com/DrMakaryFDA/...
x.com/DrMakaryFDA/...
Dr. Martin Makary on X: "The physician patient relationship is sacred and when the government tries to become your doctor, we don’t have a great track record. Our job at the FDA is to review data and decide whether products are safe and effective. The FDA is not your doctor. https://t.co/odWpWU7ngh" / X
The physician patient relationship is sacred and when the government tries to become your doctor, we don’t have a great track record. Our job at the FDA is to review data and decide whether products are safe and effective. The FDA is not your doctor. https://t.co/odWpWU7ngh
x.com
June 5, 2025 at 6:48 AM
Another must-watch podcast from the 'new' #FDA. Anybody still in camp that Makary and Prasad are bad for #innovation in drug development seriously needs to re-think. In addition to words, now plenty of action to support:
x.com/DrMakaryFDA/...
x.com/DrMakaryFDA/...
#biotech shorts and those that missed the boat by not buying Vinay Prasad appointment are becoming louder...which means we are on the right track.
CBER transparency day tomorrow. Important.
CBER transparency day tomorrow. Important.
June 4, 2025 at 7:33 PM
#biotech shorts and those that missed the boat by not buying Vinay Prasad appointment are becoming louder...which means we are on the right track.
CBER transparency day tomorrow. Important.
CBER transparency day tomorrow. Important.
#FDA Sarepta's #rAAVrh74 viral vector in the limb girdle muscular dystrophy program receives #platform designation.
This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...
This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...
U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4
CAMBRIDGE, Mass., June 04, 2025--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector used in the invest...
finance.yahoo.com
June 4, 2025 at 7:30 PM
#FDA Sarepta's #rAAVrh74 viral vector in the limb girdle muscular dystrophy program receives #platform designation.
This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...
This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...
#ProQR votes are out. deBoer (86.3%) + Shannon (80.7%) re-appointed as Directors. Should encourage corp governance discussion.
Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business?
www.sec.gov/Archives/edg...
Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business?
www.sec.gov/Archives/edg...
www.sec.gov
June 3, 2025 at 8:37 PM
#ProQR votes are out. deBoer (86.3%) + Shannon (80.7%) re-appointed as Directors. Should encourage corp governance discussion.
Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business?
www.sec.gov/Archives/edg...
Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business?
www.sec.gov/Archives/edg...
#FDA must-read: CBER chief Vinay Prasad wants to speed rare disease patient access to drugs that show any promise, also using surrogate endpoints. Vows not to interfere in review process. My man.
endpoints.news/fdas-vinay-p...
endpoints.news/fdas-vinay-p...
New CBER chief Prasad promises to 'rapidly' push even small advances for rare disease drugs
FDA biologics chief Prasad pledges flexibility on rare disease drug data, citing Gleevec as model, promises faster reviews & use of real-world data while maintaining postmarket tracking
endpoints.news
June 3, 2025 at 8:30 PM
#FDA must-read: CBER chief Vinay Prasad wants to speed rare disease patient access to drugs that show any promise, also using surrogate endpoints. Vows not to interfere in review process. My man.
endpoints.news/fdas-vinay-p...
endpoints.news/fdas-vinay-p...
#FDA lots of positive things from Prasad and Makary over last few days. Patient access to medical options is top of agenda.
On Thursday, we will hear directly from Prasad. Buckle in.
www.fda.gov/vaccines-blo...
On Thursday, we will hear directly from Prasad. Buckle in.
www.fda.gov/vaccines-blo...
CBER Roundtable on Cell and Gene Therapy
The Roundtable will provide you with an opportunity to share your thoughts on advancing the field of cell and gene therapy.
www.fda.gov
June 3, 2025 at 4:12 PM
#FDA lots of positive things from Prasad and Makary over last few days. Patient access to medical options is top of agenda.
On Thursday, we will hear directly from Prasad. Buckle in.
www.fda.gov/vaccines-blo...
On Thursday, we will hear directly from Prasad. Buckle in.
www.fda.gov/vaccines-blo...
#uniQure this AMT-260 epilepsy n=1 data looks very interesting. If it were an administration procedure artefact, I would have expected the most pronounced effect directly following admin.
The opposite is the case.
The opposite is the case.
June 2, 2025 at 6:09 PM
#uniQure this AMT-260 epilepsy n=1 data looks very interesting. If it were an administration procedure artefact, I would have expected the most pronounced effect directly following admin.
The opposite is the case.
The opposite is the case.
#Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
June 2, 2025 at 3:04 PM
#Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
Real #biotech feasts all over the place. More days like that. And the big news that too few are talking about is #uniQure's regulatory alignment. Vinay Prasad is human!
June 2, 2025 at 2:59 PM
Real #biotech feasts all over the place. More days like that. And the big news that too few are talking about is #uniQure's regulatory alignment. Vinay Prasad is human!