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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Hair counts kept climbing after month 6 on a pill.
$MANE VDPHL01 (oral minoxidil) Study 302 Part B, men with pattern hair loss: +43.9 hairs/cm² at month 12 twice daily, up from +33.0 at month 6; once daily held at +29.7. No treatment related SAEs.
Will men stay on it for years?
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25.8% vs 0% on the strictest liver composite at 48 weeks.
$ALT pemvidutide, Phase 2b IMPACT, F2/F3 MASH (n=212): fat, ALT and stiffness improved together in 42.9% on 1.8 mg vs 3.3% placebo. ELF plus stiffness: 32.4% vs 3.2%.
Noninvasive tests. Will biopsy agree?
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RazorSharpDNA 🧬 retweeted
The bottom is in.
View from THE bottom?
Over 100 trading days through November 5th $XLK underperformed $SPY by -12.7 pts, the most since at least 2010.
When Tech has lagged the S&P by 2 standard deviations in the past, the downside has been limited, often marking the bottom.
$ES_F $SPX $MSFT $MU $AVGO $QQQ $NDX $SPCX $AAPL $NVDA $MAGS $SMH
46.5% mean splicing correction after four monthly doses, vs 6.8% on placebo.
$PEPG PGN-EDODM1, Phase 2 FREEDOM2 in DM1 (10 mg/kg, n=6 vs 2 placebo): hand opening time 3.9s faster at week 16, grip up 6.3 PPN. No SAEs.
Six patients, 16 weeks. Does function hold at 6 months?
$PEPG Biotech investors often see glass half empty and miss out on doubles and triples. This applies to the 5mg/kg outlier who dominated share reaction back then.
Of course, the 10mg/kg was not just a mean reversion story...it also met the IMO necessary criteria of finally showing functional benefits with this compound. Hard task for sure in this highly variable patient population and after only 3 months.
Monthly shots, and hepatitis delta virus went undetectable in 79% by week 48.
$VIR elebsiran + tobevibart, Phase 3 ECLIPSE 1 (n=124): 44% met the primary (undetectable HDV RNA plus normal ALT) vs 0% delayed arm, p<0.0001.
Open label. Does it hold vs bulevirtide?
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5 of 5 biopsied lupus nephritis patients reached DORIS remission off all immunomodulators, after one CAR T infusion.
$CABA rese-cel at ACR: 6 of 6 dermatomyositis held responses at one year; 5 of 7 in SSc hit rCRISS-50.
Caveat: open label, single arm, optional biopsies.
Editas can now dose people with its in vivo LDL editor.
$EDIT EDIT-401 cleared Australian ethics review for the Phase 1/2 Strive study in HeFH. In monkeys, LDL-C stayed down 90%+ for 10 months.
First human safety data Q1 2027. Will primate durability hold in people? 🤷♂️
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MACE was 1 on cell therapy vs 8 on surgery alone (p=0.043).
$LGVN laromestrocel in 40 infants with HLHS (ELPIS II, AHA Sunday): right ventricle pump function no different vs surgery alone (p=0.68, CMR in 28). No treatment related SAEs.
Can a secondary endpoint carry this?
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$222.7M of Yutrepia in Q3, up 31% vs Q2 and from $51.7M a year ago.
$LQDA: 1,300 prescribers through October, referral to start conversion above 87%, October shipments a record.
Caveat: a $389.7M UTHR settlement charge made it a $236.3M net loss quarter.
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12+ straight months free of severe VOCs, independently adjudicated: $BEAM says base edited risto-cel met that pivotal BEACON primary endpoint in sickle cell, plus all primary safety and secondary endpoints.
Topline at an interim; busulfan still required. What share hit the bar?
$BEAM risto-cel in sickle cell disease.
investors.beamtx.com/news-re…
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