PolyActiva’s Post

A FAU Erlangen-Nürnberg study explored CAR T-cell therapy for severe #autoimmunediseases. Early results show promise, with all patients experiencing significant clinical improvement and discontinuing medication, highlighting the potential of this treatment approach. SCTbio has a rich history producing #GMP CAR T-cell therapies over a span of four years from process development beginnings to late-stage Phase III clinical trials. #celltherapy

Today, we announced first quarter 2024 financial results and highlighted recent business updates. The first quarter was a period of significant clinical progress for Entrada. We initiated dosing for the fourth and final cohort of our Phase 1 clinical trial of ENTR-601-44 for DMD, and we expect to report data from the clinical trial in October of 2024. Additionally, our partner Vertex announced that enrollment and dosing are underway in their global Phase 1/2 clinical trial of VX-670 for DM1.   With a cash runway through the second quarter of 2026, we are well positioned to execute on several important clinical milestones across our pipeline of intracellular therapeutic candidates to create value for patients and shareholders.    For further details, visit: https://lnkd.in/eb6NcE3k   #EntradaHere #DucheneMuscularDystrophy #FinancialResults

Emerging Options in Uncomplicated UTI - Do you know the latest Therapeutic Strategies? Watch Neil J. Kocher, MD, FPMRS, Catriona Anderson, MBChB, BSc (HON), MRCGP, DFSRH and Paula Cobb, MPT as they discuss the Impact of #uUTI and evaluate current and emerging therapies! Watch it now>> https://cmeo.me/WCV-085 #FOAMed #MedEd #CME #UTI

If we are really looking into these precision medicine initiatives- what we are doing right now is fishing for those patients who have a chance to respond to immune therapy and make sure they get it. As for the rest, it's stable disease and the hope for the occasional responder and gratefulness for any life saved. So basically, it's super-easy to be snarky and criticise and lament we should have just *prevented it all*. The kicker is this- this is the worst-off cancer patient population there is. Those who have been through all lines of treatment- and the cohorts are full of those cancers with no good treatments to start with. These are the people who are usually sent off to some more or less reasonable Ph1- or directly to palliative care to hopefully die at least without being in pain. This is the stage of disease that gives cancer its bad reputation and causes the fear- it makes it the Voldemort where people only talk about the 'c-word'. After having been through this with my husband and having become a person where people go when there's nothing left- I think it's down to 2 things: 1. we aren't particularly good at dealing with our own mortality in general and working close to death gives you a lot of pause for thought. 2. it's a clear demonstration of what we have not understood and cannot control in medicine and that's a particularly tough one when you are a clinician or a cancer researcher. And yes, we should prevent whatever is possible. But we cannot prevent everything. And we cannot undo the past- what we see now is the outcomes of prevention gone wrong for decades. And with age being one of the biggest risk factors for cancer, ageing societies don't have a good way out anyway. So the real question is whether we have the guts to tackle a setting with very little happy survivor stories. Or whether we somehow deep down think that we just aren't smart enough to crack this.

Evidence for the feasibility, early efficacy and safety of incorporating comprehensive #GenomicProfiling into clinical practice to provide #personalised treatments based on Drug Rediscovery Protocol-like Clinical Trials (DLCTs) was presented at #ESMO24. DLCTs explore the effects of #biomarker-driven treatments outside their approved indication(s) across a broad range of tumour types, thus enabling the study of #PrecisionOncology in large patient numbers, according to Vivek Subbiah, MD. 👇Read the full article on the #ESMODailyReporter https://ow.ly/tVNg50ToglA

We are excited to share data from the first cohort of the LIGHTHOUSE study, our ongoing Phase I/II clinical trial evaluating our investigational #GeneTherapy ATSN-201 for the treatment of X-linked retinoschisis (#XLRS). For the first time in patients, we have validated the ability of AAV.SPR, our novel spreading #capsid, to spread laterally well beyond the subretinal injection blebs. Additionally, we have demonstrated the ability to safely administer subretinal injections in patients with extensive retinal schisis. Subretinal injection of ATSN-201 was well tolerated in all three patients in the first cohort with two patients showing extensive resolution of schisis beginning at 8 weeks after dosing. Additional and continued resolution of schisis was observed through week 24, the latest time point available. Read more about the data in our press release: https://lnkd.in/eAcpdUHS

2 steps forward. 1 step back. F.D.A. Declines to Approve MDMA Therapy, Seeking More Study https://lnkd.in/eHjUf9hH

In the phase 3 RATIONALE-312 study, researchers aimed to evaluate efficacy & safety of tislelizumab + chemo as first-line treatment for ES-SCLC. This treatment demonstrated statistically significant clinical benefit & manageable safety compared to placebo + chemo. https://bit.ly/4akAXPN #LCSM

LCMC3 evaluated neoadjuvant atezolizumab in pts w/resectable #NSCLC & reported a 20% major PathR rate. The dev of AI-powered approaches in clinical settings may aid pathologists in clinical operations to support improved pt care & long-term outcomes. https://bit.ly/4akxjos #LCSM