@RNAiAnalyst
@rnaianalyst.bsky.social
aka Dirk Haussecker. Biotech enthusiast with expertise in sequence-directed therapeutics (RNAi, ASO, ADAR, CRISPR...) enjoying drug development and trying to make a buck along the way.
#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.
The opposite is the case.
June 2, 2025 at 6:09 PM
#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.
The opposite is the case.
Impressive #obesity data from #Regeneron: anti-myostatin antibody + Glp-1 leads to less lean body mass loss, while preserving overall weight loss.
Does a matching deal for Glp1/GIP dual with #China Hansoh.
investor.regeneron.com/news-release...
investor.regeneron.com/news-release...
Does a matching deal for Glp1/GIP dual with #China Hansoh.
investor.regeneron.com/news-release...
investor.regeneron.com/news-release...
June 2, 2025 at 11:15 AM
Impressive #obesity data from #Regeneron: anti-myostatin antibody + Glp-1 leads to less lean body mass loss, while preserving overall weight loss.
Does a matching deal for Glp1/GIP dual with #China Hansoh.
investor.regeneron.com/news-release...
investor.regeneron.com/news-release...
Does a matching deal for Glp1/GIP dual with #China Hansoh.
investor.regeneron.com/news-release...
investor.regeneron.com/news-release...
#ANGPTL3 it may be time for #ArrowheadTherapeutics and #VerveTherapeutics to reposition their ANGPTL3-targeting agents from currently only homoFH and potentially ASCVD...to much larger #MASH opportunity.
See also #EliLilly's solbinsiran and Arrowhead's zodasiran ANGPTL3 results:
See also #EliLilly's solbinsiran and Arrowhead's zodasiran ANGPTL3 results:
May 31, 2025 at 10:40 AM
#ANGPTL3 it may be time for #ArrowheadTherapeutics and #VerveTherapeutics to reposition their ANGPTL3-targeting agents from currently only homoFH and potentially ASCVD...to much larger #MASH opportunity.
See also #EliLilly's solbinsiran and Arrowhead's zodasiran ANGPTL3 results:
See also #EliLilly's solbinsiran and Arrowhead's zodasiran ANGPTL3 results:
#VerveTherapeutics the oral #PCSK9 AZD0780 discussion part sets out by stating that only 10-30% (!) reach LDLc goal.
Yeah right, adding a 10th daily oral LDLc option will change that:
Yeah right, adding a 10th daily oral LDLc option will change that:
May 28, 2025 at 6:58 AM
#VerveTherapeutics the oral #PCSK9 AZD0780 discussion part sets out by stating that only 10-30% (!) reach LDLc goal.
Yeah right, adding a 10th daily oral LDLc option will change that:
Yeah right, adding a 10th daily oral LDLc option will change that:
One good thing in the 'One Big Beautiful Bill' is that drugs with more than 1 #orphan indications will be excluded from IRA negotiations. This is relevant for #RNAi #RNAediting and #CRISPR targeting things like TTR, LDL-R, ApoB.
May 22, 2025 at 8:30 PM
One good thing in the 'One Big Beautiful Bill' is that drugs with more than 1 #orphan indications will be excluded from IRA negotiations. This is relevant for #RNAi #RNAediting and #CRISPR targeting things like TTR, LDL-R, ApoB.
#VinayPrasad wants to increase confidence in #vaccines to encourage uptake of proven vaccines.
He's a rational guy and he, too, will come around to understanding transformative value of #CRISPR programs like #VERVE-102 with help of experts.
He's a rational guy and he, too, will come around to understanding transformative value of #CRISPR programs like #VERVE-102 with help of experts.
May 21, 2025 at 9:02 AM
#VinayPrasad wants to increase confidence in #vaccines to encourage uptake of proven vaccines.
He's a rational guy and he, too, will come around to understanding transformative value of #CRISPR programs like #VERVE-102 with help of experts.
He's a rational guy and he, too, will come around to understanding transformative value of #CRISPR programs like #VERVE-102 with help of experts.
#ODAC meeting vote strongly against sponsor (Genentech) because trial involved largely non-US cancer patients and outcomes seem to differ in US-exUS.
A sign of 'America First' hurting the industry? What does this mean for other drug trials?
A sign of 'America First' hurting the industry? What does this mean for other drug trials?
May 20, 2025 at 4:41 PM
#ODAC meeting vote strongly against sponsor (Genentech) because trial involved largely non-US cancer patients and outcomes seem to differ in US-exUS.
A sign of 'America First' hurting the industry? What does this mean for other drug trials?
A sign of 'America First' hurting the industry? What does this mean for other drug trials?
OMG... #Intellia's CRISPR blows RNAi+ASO competition out of water in #ATTR-PN at 2 years. 14 out of 18, including 5/6 Patisiran progressors, continue to IMPROVE in mNIS+7, QoL, weight.
RNAi/ASO 'just' stabilizes/
ir.intelliatx.com/news-release...
RNAi/ASO 'just' stabilizes/
ir.intelliatx.com/news-release...
May 18, 2025 at 1:26 PM
OMG... #Intellia's CRISPR blows RNAi+ASO competition out of water in #ATTR-PN at 2 years. 14 out of 18, including 5/6 Patisiran progressors, continue to IMPROVE in mNIS+7, QoL, weight.
RNAi/ASO 'just' stabilizes/
ir.intelliatx.com/news-release...
RNAi/ASO 'just' stabilizes/
ir.intelliatx.com/news-release...
#ATTR-CM for the #RNAediting and #genomeediting aficionados, worth noting that the most common form of vATTR-CM in US+UK is one that can be fixed by A-->I/G editing.
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
May 17, 2025 at 1:50 PM
#ATTR-CM for the #RNAediting and #genomeediting aficionados, worth noting that the most common form of vATTR-CM in US+UK is one that can be fixed by A-->I/G editing.
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
p.V142I caused by c424G>A
No vitamin A supplementation, no daily pills. Would be a cure. @proqr.bsky.social
#IntelliaTherapeutics with impressive #ATTR-CM data showing that HEREDITARY ATTR-CM patients not just stabilized by #CRISPR NTLA-2001 nex-z, but actually IMPROVE on it.
Never seen before improvement e.g. in walk distance.
Cannot wait for longer-term data.
www.intelliatx.com/wp-content/u...
Never seen before improvement e.g. in walk distance.
Cannot wait for longer-term data.
www.intelliatx.com/wp-content/u...
May 17, 2025 at 1:47 PM
#IntelliaTherapeutics with impressive #ATTR-CM data showing that HEREDITARY ATTR-CM patients not just stabilized by #CRISPR NTLA-2001 nex-z, but actually IMPROVE on it.
Never seen before improvement e.g. in walk distance.
Cannot wait for longer-term data.
www.intelliatx.com/wp-content/u...
Never seen before improvement e.g. in walk distance.
Cannot wait for longer-term data.
www.intelliatx.com/wp-content/u...
#SilenceTherapeutics trades like it will be acquired. Nice steady climb over recent weeks, no matter political upheavals.
Lots of dealmaking in oligo space. #Regulus acquisition, #EliLilly trans-splicing deal, #Abbvie #RNAi deal...
Lots of dealmaking in oligo space. #Regulus acquisition, #EliLilly trans-splicing deal, #Abbvie #RNAi deal...
May 17, 2025 at 9:58 AM
#SilenceTherapeutics trades like it will be acquired. Nice steady climb over recent weeks, no matter political upheavals.
Lots of dealmaking in oligo space. #Regulus acquisition, #EliLilly trans-splicing deal, #Abbvie #RNAi deal...
Lots of dealmaking in oligo space. #Regulus acquisition, #EliLilly trans-splicing deal, #Abbvie #RNAi deal...
Nice 80% #RNAediting efficiency in patient-derived cells at therapeutically relevant #oligonucleotide concentrations. If #ProQR can take advantage of #FDA platform pathway, #Rett opportunity very exciting.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
May 17, 2025 at 9:52 AM
Nice 80% #RNAediting efficiency in patient-derived cells at therapeutically relevant #oligonucleotide concentrations. If #ProQR can take advantage of #FDA platform pathway, #Rett opportunity very exciting.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
#WaveLifeSciences, once saying they are out of correcting mutations, are also on it.
#ProQR had hoped to see NHP editing efficiencies for new AX-0810 #cholestatic disease candidate, but largely preclinical data with older (less potent candidate).
Guidance for CTA remains Q2 (though I am skeptical they can meet it) and topline data in Q4.
www.proqr.com/files/2025-0...
Guidance for CTA remains Q2 (though I am skeptical they can meet it) and topline data in Q4.
www.proqr.com/files/2025-0...
May 16, 2025 at 9:58 PM
#ProQR had hoped to see NHP editing efficiencies for new AX-0810 #cholestatic disease candidate, but largely preclinical data with older (less potent candidate).
Guidance for CTA remains Q2 (though I am skeptical they can meet it) and topline data in Q4.
www.proqr.com/files/2025-0...
Guidance for CTA remains Q2 (though I am skeptical they can meet it) and topline data in Q4.
www.proqr.com/files/2025-0...
#affimed's last hurray. Close to 1 BILLION shares traded, 50x outstanding shares traded, up to 500%+ earlier today.
May 16, 2025 at 6:06 PM
#affimed's last hurray. Close to 1 BILLION shares traded, 50x outstanding shares traded, up to 500%+ earlier today.
#FDA Peter Marks sees it the same way. This is a big deal, doubt 'new FDA' will backtrack on #platform approach.
Beneficiaries, #PrimeMedicine, to some degree #Beam (when certain single nucleotide mutations involved).
Beneficiaries, #PrimeMedicine, to some degree #Beam (when certain single nucleotide mutations involved).
May 15, 2025 at 6:37 PM
#FDA Peter Marks sees it the same way. This is a big deal, doubt 'new FDA' will backtrack on #platform approach.
Beneficiaries, #PrimeMedicine, to some degree #Beam (when certain single nucleotide mutations involved).
Beneficiaries, #PrimeMedicine, to some degree #Beam (when certain single nucleotide mutations involved).
#EditasMedicine #CRISPR achieving apparent 40-50%* editing in hematopoietic stem cells after just a SINGLE LNP admin in non-human primates is impressive.
In vivo sickle cell CRISPR cannot come fast enough. Tech getting close.
*still increasing at 3 months
In vivo sickle cell CRISPR cannot come fast enough. Tech getting close.
*still increasing at 3 months
May 15, 2025 at 5:35 PM
#EditasMedicine #CRISPR achieving apparent 40-50%* editing in hematopoietic stem cells after just a SINGLE LNP admin in non-human primates is impressive.
In vivo sickle cell CRISPR cannot come fast enough. Tech getting close.
*still increasing at 3 months
In vivo sickle cell CRISPR cannot come fast enough. Tech getting close.
*still increasing at 3 months
#EliLilly buys into #CRISPR for one-time future of cardiovascular disease treatment by accepting #VERVE-301 as candidate for #Lpa.
Triggered $20M milestone (why so modest, Verve, and not disclose it in PR?)
Increases likelihood they'll opt into #PCSK9 later this year.
Triggered $20M milestone (why so modest, Verve, and not disclose it in PR?)
Increases likelihood they'll opt into #PCSK9 later this year.
May 14, 2025 at 1:00 PM
#EliLilly buys into #CRISPR for one-time future of cardiovascular disease treatment by accepting #VERVE-301 as candidate for #Lpa.
Triggered $20M milestone (why so modest, Verve, and not disclose it in PR?)
Increases likelihood they'll opt into #PCSK9 later this year.
Triggered $20M milestone (why so modest, Verve, and not disclose it in PR?)
Increases likelihood they'll opt into #PCSK9 later this year.
Very satisfying to see progress on #RNAediting efficiencies. Also >50% in #RettSyndrome program. Just 2 years ago, 1-3% were top hits without ADAR overexpression.
#ProQR
#ProQR
May 13, 2025 at 10:00 AM
Very satisfying to see progress on #RNAediting efficiencies. Also >50% in #RettSyndrome program. Just 2 years ago, 1-3% were top hits without ADAR overexpression.
#ProQR
#ProQR
#ProQR impressive preclinical AX-0810 cholestatic disease #ASGCT25 abstract. Clinical candidate shows >50% #RNAediting.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
May 13, 2025 at 9:49 AM
#ProQR impressive preclinical AX-0810 cholestatic disease #ASGCT25 abstract. Clinical candidate shows >50% #RNAediting.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
Robust changes in biomarkers (serum bile acids; conjugated/unconjugated ratio) already at less editing.
$CRSP out with #CTX310 #ANGPTL3 CRISPR in hypercholesterolemia/mixed dyslipidemia.
Highest dose (n=1) w/ substantial efficacy...but 0.8mg/kg 'lean body weight-based dose' sounds rather high for an LNP. Imp to learn more about liver tox, esp that n=1 subject.
www.globenewswire.com/news-release...
Highest dose (n=1) w/ substantial efficacy...but 0.8mg/kg 'lean body weight-based dose' sounds rather high for an LNP. Imp to learn more about liver tox, esp that n=1 subject.
www.globenewswire.com/news-release...
May 6, 2025 at 8:35 PM
$CRSP out with #CTX310 #ANGPTL3 CRISPR in hypercholesterolemia/mixed dyslipidemia.
Highest dose (n=1) w/ substantial efficacy...but 0.8mg/kg 'lean body weight-based dose' sounds rather high for an LNP. Imp to learn more about liver tox, esp that n=1 subject.
www.globenewswire.com/news-release...
Highest dose (n=1) w/ substantial efficacy...but 0.8mg/kg 'lean body weight-based dose' sounds rather high for an LNP. Imp to learn more about liver tox, esp that n=1 subject.
www.globenewswire.com/news-release...
#PTCTherapeutics claims win on 'primary endpoint', huntingtin-lowering IN BLOOD! But look at more relevant CSF measurement.
#UniQure
#UniQure
May 5, 2025 at 12:19 PM
#PTCTherapeutics claims win on 'primary endpoint', huntingtin-lowering IN BLOOD! But look at more relevant CSF measurement.
#UniQure
#UniQure
I have a feeling that the #pharmaceutical industry is getting a big boost by (fascist) Trump regime...unless you are in the generics or vaccine business that is.
May 3, 2025 at 6:10 PM
I have a feeling that the #pharmaceutical industry is getting a big boost by (fascist) Trump regime...unless you are in the generics or vaccine business that is.
The #PCSK9 cardiovascular category will probably make $6B in 2025 with semi-annual subQ #Leqvio (Alnylam/Novartis) doing remarkably well against the MAbs despite not having CVOT data and slightly lower LDLc efficacy.
It's clear what patients prefer for chronic disease: less admin burden. $VERV
It's clear what patients prefer for chronic disease: less admin burden. $VERV
May 3, 2025 at 10:23 AM
Every single #BigPharma boss has now publicly kissed Trump's shoes promising billions in investments in the US and that all drugs sold there will also be made there.
Fast forward 4 years (when Trump out of office)...little of that will have materialized. Politics...
Fast forward 4 years (when Trump out of office)...little of that will have materialized. Politics...
April 29, 2025 at 9:09 AM
Every single #BigPharma boss has now publicly kissed Trump's shoes promising billions in investments in the US and that all drugs sold there will also be made there.
Fast forward 4 years (when Trump out of office)...little of that will have materialized. Politics...
Fast forward 4 years (when Trump out of office)...little of that will have materialized. Politics...