Caroline Phillips

ELRIG UK Drug Discovery 2024 is only a week away!   Nicholas Clare, Malek Haddad, Haris Choudhery and CEO, Chris Kirton will be attending and ready to showcase our cutting-edge iPSC-derived disease models and screening services. Celine Gomez will also be in attendance and presenting our poster: iPSC-derived hepatocytes as a novel platform for modelling Metabolic dysfunction-Associated Steatotic Liver Disease (MASLD) in vitro. So, whether you are looking for efficacy screening services, looking to predict hepatic safety, or in need of an iPSC-derived disease model to study #MASLD, #A1ATD, UTC disorders, Wilson’s disease or PFIC2, we have you covered.   Swing by our booth, D24, and check out Poster (no. 219), to discuss how our #CRISPR-edited disease models and in vitro screening platforms can de-risk your therapeutic research.   #DrugDiscovery #iPSC #CRISPR #Biotech #ELRIGDD24 #hepatocytes #liver #liverdisease #DefiniGEN

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Check out the key highlights from our recently released CGT Catapult Annual Review 2024 in the video below 👇 Read the Annual Review 2024: https://ow.ly/MA8k50ThbHZ #CellTherapy #GeneTherapy #AdvancedTherapies

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In Episode 4 of our pipeline analysis series, we explore the groundbreaking advancements within Bristol-Myers Squibb (BMS). Here are some key takeaways: - Oncology Breakthroughs: BMS’s oncology pipeline showcases promising candidates like Opdivo® (nivolumab) and Yervoy® (ipilimumab), expanding treatment options for various cancers such as melanoma and lung cancer. - Cardiovascular Health: BMS is making significant progress with therapies like Eliquis® (apixaban) for stroke prevention and heart health. Additionally, pipeline candidates are targeting heart failure and other cardiovascular conditions. - Immunology Advancements: BMS leads the way with innovative treatments like Orencia® (abatacept) for rheumatoid arthritis. Moreover, new therapies in the pipeline aim to address autoimmune diseases, offering hope for enhanced patient outcomes. Follow RAS LifeScience Solutions for more insightful analyses. #BMS #PipelineAnalysis #Innovation #Healthcare #Oncology #CardiovascularHealth #Immunology #Pharmaceuticals #ResearchAndDevelopment #RASLSSConsulting

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Important article in Pharmafile.com by our client Dr Dan Williams at SynaptixBio Ltd. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. Many thanks to Betsy Goodfellow. The full article is here: https://lnkd.in/e_58twVs.

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Want to improve your understanding of the key barriers facing #AdvancedTherapies and how we are working to overcome these? ➡️ https://buff.ly/44lGFOu Our experience and knowledge of #cell and #gene therapies has enabled us to identify these barriers which we call “Challenge Themes”, where we focus our innovation activities, and utilise our unique, collaborative approaches to identify and demonstrate effective solutions. Discover the Challenge Themes ☝️ #CellTherapy #GeneTherapy #ATMPs

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Congratulations Purespring Therapeutics 🎉 Recently they announced raising £80m in an oversubscribed Series B financing round. This funding will be used to accelerate the development of their novel podocyte-targeting AAV #GeneTherapy platform, which has the potential to transform the lives of the 840 million people globally with kidney diseases. This includes advancing their lead programme, PS-002, which targets IgA Nephropathy (IgAN), a common, chronic kidney disease affecting young adults, towards a Phase I/II #ClinicalTrial. There are currently no therapeutic options to stop disease progression, nor a cure, and nearly one third of IgAN patients will go on to lose their kidney function within five years and require a kidney transplant or dialysis. #genetherapy #kidneydisease https://lnkd.in/gjBZbDkD

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The UK has seen a dramatic drop in commercial clinical trial participation in recent years. Can we turn the tide? Clinical research is crucial for advancing medical innovation, and at Barts Life Sciences we believe our diverse population provides a unique opportunity for research that is both representative and impactful. Next year, thanks to funding from Barts Charity, we’re thrilled to open a much larger, pioneering Clinical Research Facility (CRF) at The Royal London Hospital. Led by Consultant Nephrologist Dr. kieran mccafferty, this state-of-the-art facility will greatly enhance our research capabilities and offer new opportunities for our local community. In our latest article, Dr. McCafferty highlights that research-active healthcare organisations are crucial for high-quality care. Yet, despite the UK’s strong global reputation, delays in study approvals have made our country less appealing for internationally-sponsored studies. How are we addressing these challenges and driving progress? Read the full article here: https://lnkd.in/egcuTjaq Barts Health NHS Trust Queen Mary University of London #BartsLifeSciences #UKLifeSciences #EastLondon #HealthcareInnovation #ClinicalResearch #Innovation

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In a recent podcast, Allucent experts Sugato De, MS (VP, Regulatory Strategy, Head of MedTech) and Alex MacDonald, PhD (VP, Model Informed Drug Development) explored the exciting world of antibody drug conjugates (ADCs) and radionuclide drug conjugates (RDCs), discussing the tremendous growth potential of these targeted oncology therapies, the challenges faced by small and mid-sized biotech companies, and strategies for navigating the complex regulatory landscape. This podcast was hosted by BioPharma Dive and can be accessed here: https://lnkd.in/g8uSZp7X.   #Allucent #BringNewTherapiesToLife #DrugDiscovery #AntibodyDrugConjugates #Radionuclides #Oncology

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📢🔬Just announced: Exa-cel becomes the UK’s first CRISPR-based therapy to be made available on the NHS. Today, NICE has issued final draft guidance recommending exa-cel, also known as Casgevy, should be funded for patients between the ages of 12 and 35 with transfusion-dependent beta thalassaemia (TDT) – an inherited condition that affects red blood cells, which are used to carry oxygen around the body. Prior to this decision, the only curative treatment available to people with beta-thalassaemia was a donor stem cell transplant - but not all patients have a suitable donor available. Exa-cel can now provide a ‘functional cure’ for people unable to have a stem cell transplant by introducing genetically modified, healthy red blood cells into eligible patients. Whilst this is fantastic news for patients with thalassemia, we’re still awaiting a decision from NICE around the use of Exa-cel for patients with sickle cell, as Yasmin Sheikh, head of policy and public affairs at Anthony Nolan notes: “We hope this game-changing therapy will also be funded for people with sickle cell, where it also has huge promise and is desperately needed. The sickle cell community has been waiting for many months now for NICE and the manufacturer of Casgevy, Vertex, to come to an agreement over bringing this therapy to the NHS for people with sickle cell.” Find out more about the approval of Exa-cel for thalassemia: https://lnkd.in/eVk5bdAe

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We bring you the latest updates from the 17th Translational Research Conference! ✨ We summarised the amazing research talks by leaders in the field! Massive thanks to our incredible sponsors: Sarepta Therapeutics, Pfizer, ITF Pharma UK, Lupin Healthcare, and Dyne Pharmaceuticals – your support makes these events possible! Here's to continued collaboration and accelerating progress in neuromuscular research. Read the full blog here: https://lnkd.in/ebTQ8bdS #NeuromuscularResearch #TranslationalResearch #InnovationInScience

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In the latest issue of Drug Development & Delivery , Dr Suzanne Saffie-Siebert and her team review the current shortcomings and accompanying manufacturing challenges of lipid nanoparticles, and they show how these shortcomings can be addressed by SiSaf’s silicon-stabilized hybrid LNPs (#sshLNPs). ⬇️Read the article https://lnkd.in/dGXT22va 🔗 Learn more about SiSaf and our Bio-Courier technology: sisaf.com #RNA #LNP #sshLNP #BioCourier

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Two recent Deallus blogs – written by Anna Scott, Ph.D., Deallus Senior Principal - explore the outlook for pharma’s prospects in neuroscience and why working in this therapeutic area is more exciting than ever. Read them here: https://lnkd.in/e8VPeG4M   While there remains an ongoing need for further research, a number of scientific breakthroughs in our understanding of the brain saw an uptick in investments in early-stage neuroscience drug programs in the late 2010s, bringing hope to millions of patients and their families whose lives are impacted by brain health conditions.      Neuroscience continues to be one of Deallus’ leading therapeutic areas, both in project volume and the expertise held by our consulting staff. We’ve had the privilege of partnering with many pharma organisations over the years as they navigate the challenges and complexities of drug development and commercialisation in neuroscience.    Read our blogs to find out more here: https://lnkd.in/e8VPeG4M   #blog #neuroscience #brainhealth #inspire #brainscience #sciencebreakthrough #expertise #brainawarenessweek #worldbrainday #advancements #equality #experience #leadingtheconversation

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Crosswalk Therapeutics is pleased to share our plans to develop a novel, next-generation enzyme replacement therapy (ERT) for Fabry disease. Our discussions with physicians and patients at this year’s WORLDSymposium, MPS Society UK, here for those with rare diseases Fabry Matters conference, and the 8th Update on Fabry Disease event from @Kidneys for Life have further illuminated for us the unmet need in the patient community and the transformative potential of our investigational technology to improve the lives of people living with Fabry disease. Natalia Boukharov, our Head of Nonclinical Research, and John Chapin, our Head of Clinical Research, presented a poster at the 8th Update on Fabry Disease Conference in Hamburg, Germany. The aim of the poster presentation, “An analysis of clinical endpoints and industry-sponsored drug development in Fabry disease,” was to foster a conversation on the challenges of designing Fabry clinical trials. Crosswalk received invaluable feedback and insights from experts. It established new connections that will help facilitate the development of our novel investigational ERT to address the current unmet needs of Fabry patients. "Patients with Fabry disease have had marked improvement in the quality of their lives with the availability of enzyme replacement therapies and more recently chaperone therapy. It is very exciting for these patients, and their treaters, to see the second generation of therapies being developed. The current unmet clinical needs of these patients include the development of anti-drug antibodies, and tissue distribution and efficacy, especially for the cardiomyocytes. The present development efforts are directly targeting these issues and may well usher in an exciting new chapter in the treatment of Fabry disease."- David Warnock, MD, Professor of Medicine (Emeritus), University of Alabama at Birmingham. We are so excited to continue our momentum. Everyone at Crosswalk remains dedicated to the Fabry patient community (patients, caregivers, and loved ones) and we will continue to work tirelessly to develop technology and therapies intended to offer new hope and new options. Learn more about the exciting work we’re doing: https://lnkd.in/eXw_HBAK #raredisease #drugdevelopment #startup #lifesciences #biotech #healthinnovation #fabrydisease #healthcare #pharmaceuticals #healthtechnology #ERT #enzymereplacementtherapy

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Last weeks Advanced Therapies Integrates was full of interesting fireside chats and panels discussions about the future of cell and gene therapies. Here's a few of our key takeaways 👇 💡“CGT developers need to lean into people with lived experience early in the development of CGTs to harness their knowledge. They might not currently do that because it is a highly emotive area, but patient led organisations have a wealth of resources and are motivated to move the needle” For the people who need them- Nicola Redfern (NJ Redfern Ltd), James Kelly (MEF2C Foundation) 💡 “Digital 5.0 is all about having a human centric approach to digital technologies. If we're to get to manufacturing thousands of batches, we need resilience in our processes. This requires visibility and transparency across the manufacturing process that humans can then interrogate and respond to.” Make Them Better- Stephen Ward (Cell and Gene Therapy Catapult), Rebecca Street (RoslinCT), Katy Newton (Achilles Therapeutics plc), Antoine Espinet (MFX) Edwin Stone (Cellular Origins) Kwok Pang (adthera bio) 💡 “For CGT investment, it's important to clearly define your patient population and have access to them. Not doing this as a company and suffering the negative consequences can have a knock-on effect to the wider industry with regards to investor confidence.” Funding Battles- Jeanette Evans (Cell and Gene Therapy Catapult), Karolina Zapadka, PhD MBA (Parkwalk Advisors), Stefanos Theoharis (OneChain Immunotherapeutics), Abigail Martin (ImmuONE), Sam Goldsmith PhD (Cell and Gene Therapy Catapult) 💡 “When looking at digital technologies. You have to understand how to bring people on board instead of fear mongering with new technologies or processes. We also shouldn't use digital technologies just for the sake of it, and make sure it works with people.” It’s an upskill battle- Ian M. Parnham, PhD (Cell and Gene Therapy Catapult), James Kusena PhD, (MFX), Mustafa Munye (Complement Therapeutics), Saerah Khan (Rentschler Biopharma), Dharmesh Vara (Cytiva), Anji Miller Ph.D CLP RTTP (LifeArc) For a full run down of the day, head to our founder Maaria's article: https://lnkd.in/e35EZX85 Thanks to all of the panellists and to Life Science Integrates for an excellent event! #advancedtherapiesintegrates #cgt #celltherapies #genetherapies #lifescience #events #mowbi

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Are you facing specific challenges or looking for tailored solutions in drug discovery? Come and say hello to Lorraine Hendry and find out how Concept Life Sciences can help expedite your drug discovery journey from Concept through to Clinic. #drugdiscovery #antimicrobials #concept2clinic

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