@RNAiAnalyst

#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.

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...

Eli Lilly acquires $VERV- official now. finance.yahoo.com/news/lilly-a...

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...

#CRISPR Stocks in Wake of $VERV Acquisition by Eli Lilly (blog): rnaitherapeutics.blogspot.com/2025/06/cris...

$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

#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...

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 ...

$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

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 ...

#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...

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

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.

Thanks!

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 💊

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.

Breaking...2nd Patient with Chronic Granulomatous Disease Successfully Treated with #PrimeEditing (blog): rnaitherapeutics.blogspot.com/2025/06/2nd-...

$PRME $BEAM #CRISPR

On the topic of the AATD-related arbitration with #BeamTherapeutics, #PrimeMedicine says AATD development candidate will include 'intentional' non-transition mutation, hence it does not infringe Beam's field which is transition-'only'.

Sounds reasonable.

#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.

#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.

Wonderful #CBER listening session with FDA commish, HHS secretary and Dr OZ. It was literally #CRISPR, #CRISPR, #CRISPR and supporting US biotech.

Now Urnov talking to HHS/FDA leadership about miracles of #CRISPR and gene therapy.

A real biotech, and esp #CRISPR-rare disease lovefest going on today at #CBER.

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...

#FDA HHS secretary Kennedy loves #CRISPR and wants US to be competitive!

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...

Updated my #IntelliaTherapeutics blog to include recent disclosures on toxicity event: delayed liver enzyme elevations and prior paracetamol use.

rnaitherapeutics.blogspot.com/2025/05/grad...

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

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/...

#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.

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...

#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...

Bestuur biotechbedrijf Proqr doorstaat aanval Van Herk De aandeelhouders zijn akkoord gegaan met de herbenoeming van ceo Daniel de Boer en president-commissaris James Shannon.

You can't make this up.

van Herk was not allowed (11%) cause of recent letter, and according to US securities laws, there has to be a 10-day cooling off period after activist actions: fd.nl/financiele-m...

Anyway, I give @proqr.bsky.social until end of month to meet guidance. Urgency needed.

#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...

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

#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...

No, unfortunately. We should see the voting results soon. No filings yet. The message should be clear: more urgency, plus I want to see the NTCP preclinical package when #ProQR announce CTA.

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.

#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...

#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.