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Lijkt me ook een vreemde tweet. Rare gozer, anti vaxer en gelooft niet in de klimaatverandering door industrie. Niet te verifiëren wat hij zegt, is gewoon een hersenspinsel van zichzelf wat hij verkondigt.
Onze BRieder, Heeft me geband op Twitter ;-) Doet hij wel met meerderen en dan post hij het wel even op DC ,anders heeft hij geen publiek meer. Met momenten zijn zijn bijdrages erg zinvol maar ooit schreef ik hem dat hij een kleuter was die teveel kleur podloodjes in bezit had gehouden. Hij is ook moderator op DC ! Daar wou hij me ook bannen maar kreeg flink lik op stuk van BBS, en de twee opstarters. Rare kwast ,net als ikzelf.
Death in CRISPR gene therapy study sparks search for answersapnews.com/article/science-technology... Although little is known about how he died, his death occurred during one of the first studies to test a gene editing treatment built for one person. It’s raising questions about the overall prospect of such therapies, which have buoyed hopes among many families facing rare and devastating diseases. “We know that CRISPR can miss its target. We know that CRISPR can be partially effective. And we also know that there may be issues with … viral vectors” that deliver the therapy into the body, he said. “Red flags are flying here. We’ve got to make sure that they get addressed very, very quickly.” Safety issues have arisen in gene therapy studies before. Late last year, Pfizer reported the death of a patient in its early-stage trial for a different Duchenne muscular dystrophy gene therapy. And in a major earlier setback for the gene therapy field, 18-year-old Jesse Gelsinger died in 1999 during a study that involved placing healthy genes into his liver to combat a rare metabolic disease. Scientists later learned that his immune system overreacted to the virus used to deliver the therapy. Of het nu wel of niet door CRISPR komt, het is in elk geval geen goed nieuws voor gene editing. Crispr -4% nu. De markt lijkt nog geen conclusies te hebben getrokken.
Volgende week is belangrijk voor Arrowhead. Benieuwd! Wat de koers doet is wat bijzaak. Al mag het eens wat meer zijn.
Piddybull. schreef op 4 november 2022 21:04 :
Volgende week is belangrijk voor Arrowhead.
Benieuwd!
Wat de koers doet is wat bijzaak.
Al mag het eens wat meer zijn.
Uuh, oneens. De koers is voor mij hoofdzaak!
de tuinman schreef op 5 november 2022 09:27 :
twitter.com/BioBoyScout/status/158869... Bbs
Hij haalt er wel precies de quotes uit die CRISPR in een kwaad daglicht stellen, haha, terwijl het nog allerminst zeker is of het tragische overlijden ook maar iets met de therapie te maken heeft. Maar goed, wij varen er wel bij. ;-)
de tuinman schreef op 4 november 2022 21:22 :
[...]
Uuh, oneens.
De koers is voor mij hoofdzaak!
de tuinman. ik wil enkel de zaak momenteel niet opdrijven. Of anders gezegd : de koers is dit jaar enkel koopwaardig. Voor de rest tegenvallend. Zo'n twee dagen voor het feestmaal zie ik het liever via data binnenstromen. Trouwens bedankt voor uw interventie elders. Er staat daar nu ineens ook "even" leuke tekst gepost over Belgische bio.
Achtergrond: Lipoproteïne(a) [Lp(a)] is een risicofactor voor atherosclerotische hart- en vaatziekten (ASCVD). Olpasiran is een kleine interfererende RNA-molecule die de Lp(a)-synthese in de lever vermindert.Methoden: Wij voerden een gerandomiseerd, dubbelblind, placebo-gecontroleerd, fase 2 dosis-onderzoek uit bij 281 proefpersonen met vastgestelde ASCVD en Lp(a) niveaus >150 nmol/L. De proefpersonen werden willekeurig toegewezen aan een van de 4 doses olpasiran (10 mg q12 weken, 75 mg q12 weken, 225 mg q12 weken of 225 mg q24 weken) of een overeenkomstige placebo, subcutaan toegediend. Het primaire eindpunt was de procentuele verandering in Lp(a) vanaf de uitgangswaarde tot week 36. Resultaten: De mediane basislijn Lp(a)-concentratie was 260,3 nmol/L en de mediane low-density lipoproteïne-cholesterol was 67,5 mg/dL. Bij de uitgangswaarde gebruikte 88% een statine, 52% een ezetimibe en 23% een PCSK9-remmer. Na 36 weken was het Lp(a) gemiddeld met 3,6% gestegen in de placebo-arm, terwijl olpasiran het Lp(a)-gehalte op dosisafhankelijke wijze verminderde met voor placebo gecorrigeerde gemiddelde percentages van 70,5% voor 10 mg om de 12 weken, 97,4% voor 75 mg om de 12 weken, 101,1% voor 225 mg om de 12 weken en 100,5% voor 225 mg om de 24 weken (alle P <0,001; figuur 1). Meer dan 98% van de proefpersonen bereikte een Lp(a)-niveau lager dan 125 nmol/L bij doses van 75 mg of hoger (figuur 2). De totale percentages bijwerkingen waren vergelijkbaar in de olpasiran- en placebo-armen: Olpasiran gedoseerd 75 mg of hoger elke 12 weken verminderde Lp(a) niveaus met meer dan 95%.
Arrowhead wordt niet genoemd, Amgen heeft daar blijkbaar geen belang bij
nelis h schreef op 7 november 2022 09:56 :
Arrowhead wordt niet genoemd, Amgen heeft daar blijkbaar geen belang bij
Oei, dan lopen wij risico dat AMGEN arrowhead wil opslokken. In ieder geval begint fase 3 in december voor Olparisan.
FDA places clinical hold on Verve Therapeutics gene-editing treatmentwww.statnews.com/2022/11/07/fda-place...
Ik ging hier zelf bijn awat details en uren opvragen : Bij deze voor deze week !(AHA) Scientific Sessions 2022, Nov 7, Aro-Ang3, Aro-Apoc3, 3:00 p.m. CST (22:00 hier ) Start of Phase 2b Study of GSK4532990 in Adults With NASH (HORIZON), Nov 8 AASLD, Nov 4-8, 3 abstracts with JNJ-3989,Virtual ARO-APOC3 and ARO-ANG3 Investor Event, Nov 9, 10:00 AM EST (16:00 uur hier)
- ARO-ANG3 decreased triglycerides by 59%, LDL-C by 32%, and was associated with a relative reduction in liver fat fraction in patients with mixed dyslipidemia - Olpasiran reduced lipoprotein(a) levels by more than 95% in patients with established ASCVD - Company will host a virtual analyst and investor event on November 9, 2022 PASADENA, Calif.--(BUSINESS WIRE)--Nov. 7, 2022-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced the presentation of new clinical data on its pipeline of investigational RNAi-based cardiometabolic medicines, ARO-APOC3, ARO-ANG3, and olpasiran, which is being developed by Amgen, in three late-breaking oral presentations at the American Heart Association (AHA) Scientific Sessions 2022, being held in Chicago. The company will also host a virtual analyst and investor event on November 9, 2022, at 10:00 am ET to discuss these data and Arrowhead’s plans for future clinical development of ARO-APOC3 and ARO-ANG3. “Our team and our partners at Amgen collectively showed promising new clinical data across three late-breaking AHA 2022 presentations on investigational candidates, ARO-APOC3, ARO-ANG3, and olpasiran, which were all developed utilizing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) technology. The totality of these data demonstrates the significant progress achieved in RNAi drug development and they specifically suggest a potential future treatment paradigm where RNAi may be prominently leveraged in preventive cardiology,” said Javier San Martin, M.D, Arrowhead’s chief medical officer. “ARO-APOC3, ARO-ANG3, and olpasiran were all highly active at silencing their respective gene targets, which resulted in encouraging changes in multiple relevant lipid and lipoprotein levels. Our confidence in the potential of these investigational medicines continues to grow as we approach the start of multiple potentially pivotal Phase 3 studies with the goal to get these important medicines to the patients that need them.” In the SHASTA-2 study in subjects with severe hypertriglyceridemia who had baseline triglycerides (TGs) of greater than 500 mg/dL, treatment with ARO-APOC3 at doses of 10 mg, 25 mg, and 50 mg all durably decreased APOC3 up to 87%, TGs up to 86%, non-HDL-C up to 45%, and increased HDL-C up to 99% through the week 16 timepoint. ARO-APOC3 has been well tolerated with treatment emergent adverse events reported to date that reflect the underlying comorbidities and conditions of the population under study. In the ARCHES-2 study in subjects with mixed dyslipidemia who had baseline median TGs of 226 mg/dL, treatment with ARO-ANG3 at doses of 50 mg, 100 mg, or 200 mg resulted in substantial reductions of ANGPTL3 up to 71% at week 8, TGs up to 59% at week 16, and LDL-C up to 32% at week 16. ARO-ANG3 was also associated with relative reduction in liver fat fraction at week 24, with no adverse events related to liver function test changes reported to date. ARO-ANG3 has been well tolerated with treatment emergent adverse events reported to date consistent with those expected in this patient population and with associated underlying comorbidities. Amgen also presented end-of-treatment data from its Phase 2 OCEAN(a)-DOSE study of TRiM-enabled investigational olpasiran in adults with elevated lipoprotein(a) [Lp(a)] levels (greater than 150 nmol/L) and a history of atherosclerotic cardiovascular disease (ASCVD). These data were presented during the Nov. 6 Late-Breaking Science Session and simultaneously published in the New England Journal of Medicine. At week 36, Lp(a) increased by a mean of 3.6% in the placebo arm, whereas there were substantial reductions of Lp(a) levels in all olpasiran arms. Placebo-adjusted mean percent reductions were 70.5% for patients receiving 10 mg every 12 weeks, 97.4% for patients receiving 75 mg every 12 weeks, 101.1% for patients receiving 225 mg every 12 weeks and 100.5% for patients receiving 225 mg every 24 weeks. The November 9, 2022 analyst and investor event will feature presentations from key opinion leaders, Christie M. Ballantyne, M.D. (Baylor College of Medicine) and Robert S. Rosenson, M.D. (Icahn School of Medicine at Mount Sinai), who will discuss the data presented at AHA, the current treatment landscape for various lipid disorders with unmet medical needs, and the potential of ARO-APOC3 and ARO-ANG3 to address dysregulated lipids and lipoproteins that may contribute to the substantial residual risk of cardiovascular disease that persists despite existing therapies. A copy of the presentation materials and a webcast link for the analyst and investor event will be available on the Events and Presentations page under the Investors section of the Arrowhead website. ARO-ANG3 is the company’s investigational RNA interference (RNAi) therapeutic designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase, being developed as a treatment for patients with mixed dyslipidemia. In the Phase 2 ARCHES-2 clinical study (NCT04832971), eligible subjects (n=203) were randomized 3:1 to receive subcutaneous injections of 50, 100, or 200 mg ARO-ANG3 or placebo on day 1 and at week 12. Subjects were on a stable diet and optimal statin/lipid-lowering therapies. In subjects with hepatic steatosis, liver fat was assessed at baseline and week 24 by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF). The primary endpoint is the percent change from baseline in fasting triglycerides (TGs) at week 24. This interim analysis (data cutoff July 6, 2022) evaluated data when all subjects reached week 12. Serum lipid and lipoprotein levels, including LDL-C, were determined at week 16 after all most subjects had received both doses. ARO-APOC3 is the company’s investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with hypertriglyceridemia (HTG), severe hypertriglyceridemia (SHTG), and familial chylomicronemia syndrome (FCS). In the Phase 2 SHASTA-2 clinical study (NCT04720534), eligible subjects (n=177/216 planned subjects) were randomized 3:1 to receive subcutaneous injections of 10, 25, or 50 mg ARO-APOC3 or placebo on day 1 and at week 12. Patients with FCS were excluded. The primary endpoint is percent change from baseline in fasting TGs at week 24. This interim analysis (data cutoff July 25, 2022) evaluated data when greater than 50% of subjects had reached week 12 and received both doses. Serum lipid, lipoprotein, and apolipoprotein levels were reported at week 16, four weeks after the second dose of ARO-APOC3 or placebo. OCEAN(a)-DOSE is Amgen’s multicenter, randomized, double-blind, placebo-controlled dose-finding study of olpasiran in 281 patients with established ASCVD and Lp(a) levels >150 nmol/L. Patients were randomized to one of four doses of olpasiran (10 mg Q12 weeks, 75 mg Q12 weeks, 225 mg Q12 weeks or 225 mg Q24 weeks) or placebo, given subcutaneously. Across cohorts, the median baseline Lp(a) concentration was 260.3 nmol/L. Patients who received 75 mg or higher every 12 weeks had a 95% or greater reduction in Lp(a) compared to placebo at week 36. At these doses (75 mg or higher), more than 98% of patients achieved an Lp(a) level of 125 nmol/L or less at week 36. Overall, the rates of adverse events were similar in the olpasiran and placebo arms. The most common treatment-related adverse events were injection site reactions, primarily pain.
Het lijkt dat de halve pijplijn een face opschuift. Nu JnJ nog.
JNJ zoekt nog steeds in alle keukenkastjes naar de juiste cocktail en wordt links en rechts ingehaald door de concurrentie
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