NEURO-SYS’ Post

The following is from my Sage CSO Ashley Barnes in our webinar "Using the power of iPSCs for patient stratification". “In my 25 years working in biopharma, clinical trial failure rate has not really improved- particularly in the neuroscience space". Together with Duncan Borthwick (our Head of Sales and Marketing) Ashley discussed the exciting potential of iPSCs and "clinical trial in a dish" (CTIAD) models to address key challenges in Alzheimer's Disease drug discovery that have driven a 99.6% failure rate for potential new therapies. Ashley described the key potential benefits as: • More human-relevant insights: Traditional animal models (including mouse amyloid overexpression models) can be limited in their ability to recapitulate human disease processes, so the use of human iPSC-derived cells could help to close this translational gap • Patient stratification: Potential therapies could be tested on the CTIAD model to find the profile of patients that would most benefit from subsequent clinical trials, known as patient stratification. This could even help to identify key biomarkers to predict drug efficacy • Increasing patient diversity: With a CTIAD model, a diverse range of patient samples can be used to widen demographic representation. Even recent breakthroughs (such as the drug donanemab) have entered clinical trials with an overwhelming proportion of patients coming from a white background Click here to read more and access the webinar: https://hubs.la/Q02kSMMK0 What do you think about an Alzheimer's Disease CTIAD model? Let us know your thoughts! #drugdiscovery #research #biopharma #pharma #clinicaltrials #biotech #drugdevelopment #ipscs

"In my 25 years working in biopharma, clinical trial failure rate has not really improved- particularly in the neuroscience space". Those were the words of our CSO Ashley Barnes in our webinar "Using the power of iPSCs for patient stratification". Together with Duncan Borthwick (our Head of Sales and Marketing) Ashley discussed the exciting potential of iPSCs and "clinical trial in a dish" (CTIAD) models to address key challenges in Alzheimer's Disease drug discovery that have driven a 99.6% failure rate for potential new therapies. Ashley described the key potential benefits as: • More human-relevant insights: Traditional animal models (including mouse amyloid overexpression models) can be limited in their ability to recapitulate human disease processes, so the use of human iPSC-derived cells could help to close this translational gap • Patient stratification: Potential therapies could be tested on the CTIAD model to find the profile of patients that would most benefit from subsequent clinical trials, known as patient stratification. This could even help to identify key biomarkers to predict drug efficacy • Increasing patient diversity: With a CTIAD model, a diverse range of patient samples can be used to widen demographic representation. Even recent breakthroughs (such as the drug donanemab) have entered clinical trials with an overwhelming proportion of patients coming from a white background Click here to read more and access the webinar: https://hubs.la/Q02kSQjn0 What do you think about an Alzheimer's Disease CTIAD model? Let us know your thoughts! #iPSCs #AlzheimersDisease #PatientStratification #ClinicalTrialInADish #StemCells

Could a "clinical trial in a dish" model transform drug discovery for Alzheimer's Disease? Here are the key facts: • Alzheimer's Disease (AD) is the most common form of dementia worldwide, affecting over 40 million individuals (and rising) • 99.6% of potential AD therapies fail to reach the market, meaning limited treatment options for patients. • AD is a complex condition with genetic and molecular heterogeneity, which is difficult to incorporate into traditional animal models and post-mortem brain tissue studies. This has posed a major challenge for drug discovery, resulting in significant costs and high attrition rates So how do we incorporate this heterogeneity in a human-relevant, scalable way? Enter the "clinical trial in a dish" model. We previously announced an exclusive license with StrataStem to commercialize a large-scale collection of diverse AD patient samples. Using our iPSC expertise, we want to turn these samples into iPSC-derived cells and incorporate them into a cohort-scale in vitro model for drug discovery, aka a “clinical trial in a dish”. The power of this platform comes from patient stratification, where we can test potential therapies on these models to identify the profiles of patients who respond better, maximizing later-stage clinical trials and de-risking the process. It's an incredibly exciting area of research and we're actively looking for partners to collaborate on this project. Click here to read more: https://hubs.la/Q02jD95h0 #iPSCs #AlzheimersDisease #ClinicalTrialInADish #PatientStratification #StemCells

"In my 25 years working in biopharma, clinical trial failure rate has not really improved- particularly in the neuroscience space". Those were the words of our CSO Ashley Barnes in our webinar "Using the power of iPSCs for patient stratification". Together with Duncan Borthwick (our Head of Sales and Marketing) Ashley discussed the exciting potential of iPSCs and "clinical trial in a dish" (CTIAD) models to address key challenges in Alzheimer's Disease drug discovery that have driven a 99.6% failure rate for potential new therapies. Ashley described the key potential benefits as: • More human-relevant insights: Traditional animal models (including mouse amyloid overexpression models) can be limited in their ability to recapitulate human disease processes, so the use of human iPSC-derived cells could help to close this translational gap • Patient stratification: Potential therapies could be tested on the CTIAD model to find the profile of patients that would most benefit from subsequent clinical trials, known as patient stratification. This could even help to identify key biomarkers to predict drug efficacy • Increasing patient diversity: With a CTIAD model, a diverse range of patient samples can be used to widen demographic representation. Even recent breakthroughs (such as the drug donanemab) have entered clinical trials with an overwhelming proportion of patients coming from a white background Click here to read more and access the webinar: https://hubs.la/Q02kSMMK0 What do you think about an Alzheimer's Disease CTIAD model? Let us know your thoughts! #iPSCs #AlzheimersDisease #PatientStratification #ClinicalTrialInADish #StemCells

Could a "clinical trial in a dish" model transform drug discovery for Alzheimer's Disease? Here are the key facts: • Alzheimer's Disease (AD) is the most common form of dementia worldwide, affecting over 40 million individuals (and rising) • 99.6% of potential AD therapies fail to reach the market, meaning limited treatment options for patients. • AD is a complex condition with genetic and molecular heterogeneity, which is difficult to incorporate into traditional animal models and post-mortem brain tissue studies. This has posed a major challenge for drug discovery, resulting in significant costs and high attrition rates So how do we incorporate this heterogeneity in a human-relevant, scalable way? Enter the "clinical trial in a dish" model. We previously announced an exclusive license with StrataStem to commercialize a large-scale collection of diverse AD patient samples. Using our iPSC expertise, we want to turn these samples into iPSC-derived cells and incorporate them into a cohort-scale in vitro model for drug discovery, aka a “clinical trial in a dish” The power of this platform comes from patient stratification, where we can test potential therapies on these models to identify the profiles of patients who respond better, maximizing later-stage clinical trials and de-risking the process. It's an incredibly exciting area of research and we're actively looking for partners to collaborate on this project. Click here to read more: https://hubs.la/Q02jDdZh0 #iPSCs #AlzheimersDisease #ClinicalTrialInADish #PatientStratification #StemCells

🔊 Weekly round-up! 🔬 Translating ‘nature’s cues’ into breakthrough immunotherapies Discover how Cue Biopharma harnesses the curative potential of the body’s intrinsic immune system to develop treatments for oncology and autoimmune diseases. Here, Anish Suri Ph.D., Chief Scientific Officer of Cue Biopharma, explains how its platforms and biologics are designed to selectively modulate disease-specific T cells to restore immune balance, aiming to improve efficacy and minimise adverse effects. 👩🔬 Women in STEM with Juliet Williams We spoke to Juliet Williams, Head of Research at Kymera Therapeutics. She discusses what inspired her early passion for science, the power of mentorship and what excites her most about the future of targeted protein degradation – and its potential to deliver results in previously undruggable conditions. 🐁 New model offers a unique method to study Parkinson’s disease Researchers at Weill Cornell Medicine have shown that mice with rod-specific VPS35 deletion demonstrate a pathology more similar to human Parkinson’s disease, compared to other mouse models. 💊 The role of KDR in the survival of HTLV-1-infected T cells Researchers at Penn State College of Medicine have discovered that KDR inhibitors could be very impactful for treatment of ATLL and HAM/TSP or prevent disease by lessening the viral load of HTLV-1. Click on the links in the comments to read more! 👇 #drugdiscovery #lifescience #drugtargets 

🔲 F1000 Media: Coya Therapeutics, Inc. Accelerates Alzheimer’s Research with Key Collaborations Coya Therapeutics is making waves in the biotech industry with its recent advances in neurodegenerative disease treatment. In the last two months, the company has been focusing on expanding research and development efforts, particularly around #Alzheimer’s and other neurodegenerative diseases. 🔳 Collaboration with Dr. Reddy’s Laboratories: Coya Therapeutics is leveraging its partnership with Dr. Reddy’s Laboratories to enhance its flagship biologic, #COYA302. This dual-mechanism treatment for Alzheimer’s disease shows promising results in modulating the immune system, specifically targeting the inflammation that contributes to the progression of neurodegenerative disorders. 🔳 Presenting at Major Healthcare Conferences: In September and October 2024, Coya is scheduled to present its latest findings at several key conferences, including the Alzheimer’s Drug Discovery Foundation (ADDF) Summit. Their focus on innovative biologics and therapies designed to restore immune balance is set to change how the medical community approaches diseases like #Alzheimer’s, #Parkinson’s, and #ALS. 🔳 Advancing Neuroinflammation Research: These collaborations and conference presentations highlight Coya’s commitment to advancing science in neuroinflammation and regulatory T cell therapies. By combining forces with top industry players, Coya is positioning itself at the forefront of finding effective treatments for neurodegenerative diseases. #CoyaTherapeutics #AlzheimersResearch #Biotechnology #Neurodegeneration #TregTherapies #Coya302 #F1000Media #DrReddys #ADDF #Neuroinflammation #Parkinsons #ALS #HealthcareInnovation

The Alzheimer's Gamble by Jocelyn Kaiser. Further readings: 1) Novel Approach to Treatment and Prevention of Alzheimer's Disease https://lnkd.in/eqHm424J 2) Alzheimer's Disease: Barriers and Solutions to Progress https://lnkd.in/d6tXXTHJ 3) Alzheimer's Disease: The Amyloid Cascade Hypothesis Turns 30 https://lnkd.in/dDZCMUwW 4) Biomarker, epigenetics, preclinical model and repurposing resources for AD https://lnkd.in/d6Gm_cMC 5) When will there be a cure https://lnkd.in/dzQhedN2 6) Alzheimer’s disease successes—aducanumab and oligomannate—against 98 failures gives a 2.0% phase II and III clinical drug development success rate since 2003 https://lnkd.in/dXw5Ub6F 7) 41 Alzheimer's Disease Targets prioritized by the biological intelligence-powered BRAINCURES Discovery Engine (BDE) from a list of 187 unique AD-genes published within "Multiomic prediction of therapeutic targets for human diseases associated with protein phase separation" https://lnkd.in/dvENqagJ 8) Precision pharmacology for AD https://lnkd.in/dxs_WTgj 9) The price of progress against AD https://lnkd.in/ddhYpEJR 10) Leveraging preclinical models in AD research https://lnkd.in/dKNUEqCM

🔔 Conference Early Bird Registrations are now Open! Join us in London on Oct 1st for the 1st Getting Cilia Moving Conference Bringing together the key opinion leaders, UK Government, UKRI accelerators, industry KOLs, investors, experts in regulation and the patients to work together to leverage the progress made in cystic fibrosis into primary ciliary dyskinesia. If you have an interest in #respiratory #rare diseases, #brochiecstasis or #genetherapy, join us. Key highlights: 🌟 Focus on translational medicine for genetic respiratory diseases, and lessons learned from Cystic Fibrosis  🌟 Engage with experts and stakeholders in genomics, translational medicine, genetic therapies 🌟 Networking, expert panels, patient perspective and more more Save over £600! Register by 30th July for Early bird pricing 👉 https://lnkd.in/ezN7RDuz Ethris ReCode Therapeutics Skip Therapeutics SpliSense Vertex Pharmaceuticals Insmed Incorporated Parion Sciences Simbec-Orion LifeArc Prime Medicine, Inc. AXON Moderna Alexion Pharmaceuticals, Inc. FibroGen, Inc. Grifols Eloxx Pharmaceuticals CRISPR Therapeutics Weatherden #rarediseases #genetherapy #biotech #mRNA #PCDresearch #geneandcelltherapy #advancedtherapies

ACD856’s anti-inflammatory effects to be presented at a leading Alzheimer’s conference AlzeCure´s abstract with preclinical data on NeuroRestore ACD856 demonstrating its anti-inflammatory and immunomodulatory effects has been accepted for presentation at the annual Alzheimer's conference CTAD, Clinical Trials in Alzheimer's Disease, which this year is being held in Madrid, Spain, on October 29 – November 1. The abstract, titled Preclinical evidence for anti-inflammatory and immunomodulatory effects of NeuroRestore ACD856, a Trk-PAM in clinical development for the treatment of Alzheimer’s disease, will be presented at the conference by Dr. Cristina Parrado-Fernández, Senior Scientist at AlzeCure. The other authors are Veronica Lidell, Azita Rasti, Maria Backlund, Gunnar Nordvall, Martin Jönsson, Johan Sandin and Pontus Forsell from AlzeCure Pharma, and Ruchi Gera, Sumonto Mitra and Prof. Maria Eriksdotter from Karolinska Institutet. The presentation includes new preclinical results with ACD856, the lead clinical drug candidate within the NeuroRestore platform. The data show that the substance shows both anti-inflammatory and immunomodulating properties, something that has been demonstrated after treatment in both aged animals and in Alzheimer's models. This indicates potentially disease-modifying properties of this class of molecules. Read the press release here: https://lnkd.in/dVsTCtRU The abstract and the poster will be available on AlzeCure’s website after the presentation https://lnkd.in/dnVm8J3f #alzecure #CTAD24 #neurorestore #alzheimer #ACD856 #alzheimerfonden