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:
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…
No efficacy numbers, just a stop.
$GERN ended Phase 3 IMpactMF (imetelstat, JAK-relapsed myelofibrosis, n=327) after the DMC said overall survival was unlikely to win. No new safety signals.
Rytelo's approved MDS label is unaffected; Q2 sales were $57.5M.
The long arc of biotech. I predicted the failure of $GERN imetelstat myelofibrosis study effort 8 years ago...
Ahead of Geron blowup, warning signs were visible for those willing to see them statnews.com/2018/09/27/gero…
87% fewer HAE attacks after one infusion.
Since this March 2025 post, $NTLA's lonvo-z hit every key Phase 3 HAELO endpoint (n=80), and FDA set a Mar 10 2027 PDUFA with no AdCom planned.
Still not approved. Does the first in vivo CRISPR label reprice the group?
🐻The CRISPR bear market from 2021-2025 🪦 A picture is worth a thousand words (see screenshots below for market caps comparisons 2021 vs. 2025) 😱 $CRBU $NTLA $CRSP $BEAM $SANA $PRME $EDIT $VERV $MGX $IPSC $ARKG
🔵Total Market Cap of Key CRISPR Publicly Traded Companies (2021) ≈ $26.74 billion USD 🚀 🌕 🫧
🆚
Comparison of this CRISPR basket to 2025 💀 ⬇️
🔵2025 Total (March 26) ≈ $8.69 billion
📉Decline -67.5% decrease in total market cap 🤯
‼️This means that the CRISPR 🧬 ✂️Gene Editing sector has lost nearly $18 billion in market value since 🎅 EOY 2021, largely due to broader biotech downturns, Trump uncertainty, rising interest rates, investor sentiment shifts, impatience & commercial challenges.
It’s even worse if you compare to ATHs earlier in 2021 😂
The Compound Annual Growth Rate (CAGR) of the CRISPR gene-editing market from 2021 to 2030 is projected to be between approximately 14.73% and 30%, according to various industry reports.
Have we bottomed yet❓
The 99% is T cell responses, not patients helped.
$ELTX's KRAS vaccine camupepimut missed its Phase 2 primary in resected pancreatic cancer: DFS 23.8 vs 21.2 months, HR 0.85, p=0.53, n=144. The ctDNA negative subgroup had HR 0.51.
Can a ctDNA enriched Phase 3 confirm that?
🧬 A New Pancreatic Cancer Vaccine Activated the Immune System in Nearly 99% of Patients
What if the immune system could be trained to recognize one of the deadliest cancers?
Scientists have developed an experimental vaccine called camupepimut that teaches immune cells to recognize specific genetic mutations linked to pancreatic cancer.
In a phase 2 clinical trial involving 144 patients, the results caught researchers' attention. Among 90 vaccinated patients whose immune responses could be measured, 89 (98.9%) developed stronger T-cell responses against cancer-related KRAS mutations.
These immune cells are important because they can recognize and attack abnormal cells. Researchers also observed that patients with stronger immune responses tended to remain cancer-free for longer.
But there is an important detail: the vaccine has not yet been proven to prevent cancer from returning or extend survival. The trial did not show a statistically significant improvement in disease-free survival across the entire group. A larger Phase 3 trial is needed.
Still, teaching the immune system to recognize cancer-related mutations in almost every patient is an encouraging step toward new pancreatic cancer treatments.
Source:
Lymph node-targeted, mKRAS-specific amphiphile vaccine in pancreatic cancer: The randomized phase 2 AMPLIFY-7P trial. (2026). Nature Medicine.
ALT 🧬 **A New Pancreatic Cancer Vaccine Activated the Immune System in Nearly 99% of Patients** What if the immune system could be trained to recognize one of the deadliest cancers? Scientists have developed an experimental vaccine called **camupepimut** that teaches immune cells to recognize specific genetic mutations linked to pancreatic cancer. In a phase 2 clinical trial involving 144 patients, the results caught researchers' attention. Among 90 vaccinated patients whose immune responses could be measured, **89 (98.9%) developed stronger T-cell responses** against cancer-related KRAS mutations. These immune cells are important because they can recognize and attack abnormal cells. Researchers also observed that patients with stronger immune responses tended to remain cancer-free for longer. But there is an important detail: the vaccine has not yet been proven to prevent cancer from returning or extend survival.
Source: nature.com/articles/s41591-0…
REGAL hit death number 80. Now comes the unblinding.
$SLS's Phase 3 tests galinpepimut-S as AML maintenance in second remission; overall survival is the primary endpoint. Topline is promised this quarter.
An event count says nothing about efficacy. What HR would matter here?
Same pill, twice the survival in one subgroup.
$ZNTL's WEE1 pill azenosertib, platinum-resistant ovarian cancer (ESMO): median OS 15.9 mo if Cyclin E1-positive vs 7.5 mo if negative. ORR 37.2%.
Single arm; 43% needed dose cuts. Is Cyclin E1 predictive, or just prognostic?
ALT $ZNTL azenosertib DENALI Cyclin E1 OS charcoal card
7 of 26 needed zero surgeries for a year. That earned the first FDA approval for a DNA medicine.
$INO's VOCERYQ (INO-3107), accelerated approval Oct 30 for adult RRP, an HPV airway disease. Single arm, 32 treated. Confirmatory protocol due by end of 2026. Durable past year one?
$31M more Attruby than last quarter. BridgeBio now frames $25M to $30M a quarter as the near term pace.
$BBIO US Attruby sales hit $253.2M in Q3. The small $11.6M operating loss leaned on a single $75M Bayer milestone.
How steep can the climb stay in a crowded ATTR-CM market?
When I saw the Attruby numbers last night, I thought that this drug is doing best in the very competitive aTTR market, but I also think the intense competition is hurting everyone in this space overall. I do not think Attruby will be as big as many think. I always have my numbers lower than everyone else. With that said, I am a buyer on weakness as it has 3 more drugs going commercial which have far less competition.