SMID Biotech & Gene Editing 🧬 Hype welcome, receipts mandatory. Primary sources. Long some of what I post. Opinions, not 💵 advice.
Joined November 2011
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Welcome to the receipts desk 🧬
SMID biotech & gene editing. Numbers first, caveat second — and only with receipts.
House rules:
1. Hype is welcome. Receipts are mandatory. FDA letters, 8-Ks, trial registries. Not "people familiar with the matter."
2. A press release is the trailer. The filing is the movie.
3. "Up to $2B" is not $2B. Milestones are wishes with a spreadsheet attached.
4. When I'm wrong, I'll say it here. No quiet deletes.
5. Sarcasm is free. Facts aren't negotiable.
Moonshot calls are fine. Moonshot calls with zero receipts get roasted.
Follow if you want the numbers and the caveat.
🤖 Made with AI
Sources:
Precision late-breaker investor.precisionbioscience…
Vir SOLSTICE nasdaq.com/press-release/vir…
Altimmune IMPACT nasdaq.com/press-release/alt…
~110 Dravet patients are past the week 28 primary endpoint.
$STOK's zorevunersen, Phase 3 EMPEROR: ~20 past week 52, all in open label. Readout Q3 2027, NDA planned 2H27, cash $358.8M into early 2028.
Cognition is a key secondary. Can it move behavior, not just seizures?
Source: nasdaq.com/press-release/sto…
One drug: 0 of 14 major responses. Add a second: 4 of 14 colon tumors had no viable cancer at surgery.
$AGEN's botensilimab plus balstilimab, given before surgery in MSS colon cancer. No combo patient relapsed at 19.2 months median.
14 per arm, not randomized. Will it hold?
Source: nature.com/articles/s41591-0…
Casgevy Q3: $99.2M, up 30% from Q2's $76.4M and nearly 6x Q3 2025. 750+ patients initiated, about 200 infused since launch, a fourth straight quarter of 100+ starts. $CRSP splits Casgevy profits 40/60 with Vertex. Vertex flags Q4 seasonality in infusion timing.
Sources:
Nature Medicine STOP-V: nature.com/articles/s41591-0…
Krystal Q3: ir.krystalbio.com/news-relea…
Kidney function fell 0.6 a year on drug, 5.7 on placebo.
$VOR's telitacicept (dual BAFF/APRIL blocker) in IgA nephropathy, 2 year Phase 3 TELIGAN: eGFR slope −0.6 vs −5.7, p<0.001. Run only in China by partner RemeGen.
Does China kidney data carry any US weight?
Source: globenewswire.com/news-relea…
8 deaths now reported to FDA's adverse event database in Prader Willi patients on $NBIX Vykat XR since its March 2025 approval, with swelling and hyperglycemia listed again. Caveat: spontaneous reports have no denominator and can't prove cause. How many patients are on drug?
Another Prader Willi patient prescribed Neurocrine's $NBIX Vykat has died, according to the FDA's AEMS database.
That's the eighth patient death reported since the drug was approved. Like the other cases, "swelling" and hyperglycemia were listed as contributing factors.
Source: FDA FAERS public dashboard fda.gov/drugs/fdas-adverse-e…
Oct 2023 I asked which CRISPR name gets bought first.
Answer: $VERV. Lilly closed it in July 2025 at $10.50 a share plus a CVR, up to about $1.3B. $GRPH never got bought; it reverse merged into $LENZ.
Small sample. Who is next on the list?
The #Biotech merger & acquistion hype is back 😆
🤔I wonder which public #CRISPR company will be first on the hit list for Big Biotech/Pharma to gobble up 😋
>$1 Billion Market Cap
$NTLA
$CRSP
$BEAM
<$1 Billion Market Cap
$CRBU
$PRME
$SANA
$VERV
$GRPH
Place your bets 🏇
Some recent non-CRISPR examples below from 2023 so far…
$182M in one quarter, a year into launch.
$PTCT's oral PKU drug Sephience grew 20% vs Q2, with 2,000+ patients on it. Product sales guidance is now $960M to $990M. Next: a Q4 BLA for ST-920, a one dose Fabry gene therapy.
Ex-US was only $37M. How much room is left abroad?
ALT $PTCT Sephience Q3 2026 sales card: $182M quarter, 20% growth vs Q2, 2,000+ patients, guidance $960M to $990M
One base editing shot cut triglycerides 75%. In mice.
$CLLS's .HEAL-101 (ESGCT): ~65% editing of human APOC3 in humanized liver mice, liver only, no double strand break.
Preclinical. First human data 2H27 from a China investigator trial. Can one edit replace repeat APOC3 shots?
Source: nasdaq.com/press-release/cel…
