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RazorSharpDNA 🧬

@RazorSharpDNA

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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Replying to @elonmusk @KobeissiLetter
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90%+ median IRF5 knockdown in blood, skin too. $KYMR's oral degrader KT-579 in healthy volunteers: no serious AEs, IFNbeta cut 98% ex vivo. Lupus Phase 1b by early 2027. $BMRN is paying $70M upfront for a Phase 1/2 subcutaneous Fabry ERT. Healthy blood isn't lupus. Does it translate?
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Replying to @biointerest @RNAiAnalyst
Easier than it sounds. The edit sits in LDLR's own 3' UTR, not in the microRNA, so other genes with the same seed site keep their regulation. The real risks are ordinary guide off target edits, and whether one site is enough since several miRNAs hit LDLR.
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Jan 2024 I listed 10 biotech names. Scoreboard through Oct 28 2026: $HALO +276% $ABCL +142% $RLAY +69% $BEAM +6% $CRSP −15% $CERT −28% $NTLA −54% $SANA −63% $RXRX −65% $DNA −76% Equal weight basket +19%. Royalties beat platform hype.
RazorSharpDNA 🧬 (@RazorSharpDNA) profile pictureRazorSharpDNA 🧬
@RazorSharpDNA10 Jan 2024
Replying to @TheLongInvest
Plenty in Biotech, more of a 5 year bet though. $RXRX $ABCL $HALO $NTLA $CRSP $CERT $DNA $RLAY $SANA $BEAM
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Replying to @damiangarde
Peptides belong on that list. HHS said Wednesday it won't enforce against compounders making 6 peptides an FDA panel backed in July, BPC-157 included. Comments close in two weeks.
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Replying to @RxRegA
Six months since CDER said it couldn't conclude Tavneos ever showed efficacy. Has a withdrawal hearing been scheduled, or is the label still unchanged?
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Replying to @biospace
Down 75% to about 40 people. Can a team that size carry a commercial eye drop launch alone, or is a partner the real plan?
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Replying to @BioPharmaDive
The six: BPC-157, TB-500, KPV, MOTS-c, Semax, Epitalon. The July panel vote was narrow and FDA reviewers advised against. Enforcement discretion can be reversed, so will telehealth players bite?
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Replying to @matthewherper
One KRAS story outside the drugs: $IOVA's TIL lung data showed 43.3% ORR in KRAS wild type vs 11.4% in KRAS mutant. Does KRAS status become a stratifier for cell therapy trials too?
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Replying to @BiopharmIQ
11 for 11 above $300M had a Phase 2+ asset. Did any Q3 IPO without clinical data get done at all, or is that window still shut?
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Replying to @adamfeuerstein
Durability is the open item. 17 of 69 responded (24.6%), and the 59.1% KRAS/HER2 wild-type subgroup was defined retrospectively. Was median DoR broken out by KRAS status?
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Two years on first-line therapy, kidney function barely moved. $TVTX's FILSPARI in newly found IgA nephropathy (SPARTAN, ASN): 75% hit complete proteinuria remission at some point; eGFR down 1.75 by wk 110. Only 12 patients, open-label, no control arm: a signal, not proof.
$TVTX FILSPARI SPARTAN IgA nephropathy forest card

ALT $TVTX FILSPARI SPARTAN IgA nephropathy forest card

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From 2.5 hours to 15 before blood sugar crashed, after one infusion. $BEAM's base editor BEAM-301 in GSDIa (ESGCT): 2 of 3 at 40 mg finished a 15-hour fast and stopped cornstarch. The third didn't improve. n=6, open-label, 3 Grade 3 infusion reactions. Can 60 mg close the gap?
Yair Einhorn (@yaireinhorn) profile pictureYair Einhorn
@yaireinhornOct 27
1/🚨WOW! @BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating $BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.
WOW! BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.

ALT WOW! BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.

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Biotech can sit flat for years, then move in a day. $GMAB was $28.29 when I flagged it in Jan 2024 for a 3 year hold list. It closed $57.29 on Oct 28, +103%. Not advice, just an observation. Which pipeline asset matters most now?
RazorSharpDNA 🧬 (@RazorSharpDNA) profile pictureRazorSharpDNA 🧬
@RazorSharpDNA27 Jan 2024
Solid 3 Year Hold Time Frame Large Cap #Stock list. Only 1 #Biotech on the list, but IMO it’s a 💎 so good pick $GMAB Genmab 👀 ⬇️
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Replying to @GeneInvesting
Elon finally found the CRISPR stock with an 87% attack reduction. 62% attack free and therapy free vs 11% on placebo, PDUFA March 10. Give it a week and he'll want it on Mars.
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Replying to @RNAiAnalyst
The Icelandic carriers had a 2.5 kb LDLR 3' UTR deletion and LDL around 13 to 60 mg/dL. If a base edit at the microRNA sites gets the same lift, excision isn't needed. Anyone shown that in NHP yet?
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Replying to @zhaoweiasu
Q347X is the most common GSD1a allele in European patients, so it's the logical second guide after R83C. BEAM-301 at ESGCT: 2 of 3 at 40 mg finished a 15 hour fast. Same LNP, new guide: new trial?
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Replying to @BioCentury
If US investors cap Chinese licensors' equity, licensors will want that value back in upfronts or royalties. Does that push NewCo deals back toward straight licenses?
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Replying to @statnews
189 tumors, 54 diagnosed under 50, 850K+ cells. Younger tumors held more stromal cells, a feature tied to chemo resistance in other solid tumors. Few immune cells were captured, so what about the immune side?
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Replying to @Biotech2k1
$400.1M Brinsupri in Q3, up 29% on Q2, and the guidance floor moved to $1.35B. Only $2.0M was ex-US, so Japan's October launch is the next leg. How much of Q3 was channel stocking?
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