Biotrial’s Post

Timely update on the status of #CRISPR Clinical Trials. In late 2023, we saw the first-ever approval of CRISPR-based medicine: Casgevy, a cure for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This update tracks the progress CRISPR clinical trials since they first began, and this milestone has been anticipated for some time, but its speed is still noteworthy. #medicine #medicaltechnology #clinicaltrials https://lnkd.in/gaGv7PPN

LinkedIn network, looking for your help from those that market therapeutics in rare diseases. What are the best conferences you have participated as an exhibitor? Looking for all specialties. TIA for your input! Some I have participated in are ASH, Kidney Week, AAD, and Fall Clinical. #pharmamarketing #conference2024 #raredisease #healthcareanalytics

How can patients with rare disorders access cutting edge therapeutic discovery programs? Unravel Biosciences is using nasal swab kits to pioneer drug development for rare #CNS diseases. This innovative approach not only simplifies the sample collection process but also ensures a hassle-free, minimally invasive, and physically accessible experience for all #patients. By utilizing swab kits, patients around the world can access BioNAV™, our powerful drug discovery platform to facilitate rapid drug prototyping and accelerate the pace of urgent rare disease treatments. This means we’re bringing #personalized therapies more effectively to patients globally than ever before! #drugdiscovery #rarediseases #rarediseaseday2024

We are progressing with our personalized phage therapy against NTM lung disease, targeting M. abscessus. As we work our way toward clinical trials, we're introducing a limited Expanded Access Waitlist for patients. Expanded Access provides a pathway for patients to access investigational medical products outside of clinical trials. Interested individuals can join the waitlist here. https://lnkd.in/evEUAJQF Disclaimer: This drug is investigational and has not been approved for commercial distribution. It has not yet been proven to be safe and effective in randomized controlled clinical trials. #phagetherapy #ntm #expandedaccess #personalizedmedicine

📢 Upcoming Industry Symposium Alert! 📢 Join us on Wednesday, March 6, at 8:40 AM (GMT) in Auditorium I for an insightful industry symposium that promises to deepen your understanding and spark engaging discussions. 🔍 Focus Area: We will be diving deep into the latest research on alpha-synuclein targeting approaches, which are at the forefront of innovation in diagnostics and therapeutics. 🎯 What to Expect? • Get first-hand insights from leading experts in the field. • Explore cutting-edge diagnostic and therapeutic strategies. • Network with professionals and thought leaders passionate about neurological research. Don’t miss this opportunity to stay ahead of the curve in the dynamic landscape of neurological research. Let’s explore the future of diagnostics and therapeutics together! #IndustrySymposium #AlphaSynuclein #Diagnostics #Therapeutics #precisionmedicine #parkinsonsdisease #NeurologyResearch

May is Stroke Awareness Month! 🧠❤️ Let's come together to spread awareness about preventing recurrent strokes and supporting survivors. Did you know that for some patients, DAPT (Dual Antiplatelet Therapy) can significantly reduce the risk of recurrent strokes? (1) By assessing an individual's CYP2C19 genotype, their DAPT therapy can be further optimized to ensure effective treatment. (2) Discover how Genomadix is paving the way for personalized DAPT guidance for stroke patients. Visit Genomadix | Molecular testing. Where and When you need it to learn more. #StrokeAwarenessMonth #DAPT #Pharmacogenetics #PrecisionMedicine 1. Kleindorfer DO, et al. Stroke. 2021;52:e364–e467. 2. Lee CR, et al. J Clinical Pharmacol Therapeutics. 2022;122(5):959-967.

Carole Ho, Chief Medical Officer & Head of Development at Denali Therapeutics, emphasizes the urgent need for therapies to treat neuronopathic Mucopolysaccharidoses (MPS) and how Denali is focusing its novel technology to on these progressive and debilitating diseases. Read the full piece on BioSpace. ⬇ #RareDiseases #RareDiseaseDay 

OLOKER – FROM THE BENCH TO THE BEDSIDE. The case of cells called CPCPlus, endowed with impressive therapeutic potential, which can be transformed into a new pharmaceutical product indicated in refractory angina. In just a few years, Oloker, the start-up created to treat the most serious forms of cardiac ischemia, is reaping the first results. It all starts with a patent on CPCPlus (Cardiac Pro-angiogenic Cell Plus), a therapeutic method developed after 10 years of research, aimed at the isolation of a subtype of human heart stromal cells (CD90 negative), capable of inducing neo-vascularization . This research was conducted by the Monzino Cardiology Center and the University of Milan. #Oloker #CPCPlus #angina

Treatment with TAR-200 led to complete responses in over 75% of patients with BCG-unresponsive, high-risk NMIBC, according to results from the phase 2b SUNRISE-1 trial. https://lnkd.in/eifZtm5J

⚡New efficacy data from NETTER-2 Phase 3 trial on 177Lu-Dotatate published in The Lancet! 📊Snapshot: Between Jan 22, 2020, and Oct 13, 2022, 261 patients screened and 226 patients randomly assigned to 177Lu-Dotatate plus octreotide LAR (n=151) or control octreotide LAR (n=75). Median PFS was 8.5 months in the control group and 22.8 months in the 177Lu-Dotatate group (HR 0.276; p<0.0001). Adverse events occurred in 93% of 177Lu-Dotatate patients and 95% of control patients. No drug-related deaths occurred. 177Lu-Dotatate significantly extended PFS by 14 months in grade 2/3 gastroenteropancreatic neuroendocrine tumour patients and should be considered a new first-line standard of care. https://ow.ly/AwNS50ScNMT #MedEd