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Gene&Cell Therapy >> Merck partners with small UK biotech to drug fibroblasts for fibrosis, I&I diseases: As Merck builds out its immunology research lab in London, it’s partnering with a local biotech startup that has remained relatively quiet since its seed round in 2021. The pharma giant said Tuesday morning it is pairing up with Cambridge, UK-based Mestag Therapeutics to find new targets for fibrosis and inflammatory disease treatments. Merck will deliver an upfront payment of undisclosed size to the nearly four-year-old Mestag, which is working on drugging fibroblasts. These cells are integral to the formation of connective tissue and also help communicate with immune cells and give them navigation orders. If all goes to plan, Mestag stands to gain as much as $1.9 billion from the yearslong partnership that the duo has inked. Mestag could deliver a “number of targets,” but the startup’s CEO, Susan Hill, declined to disclose how many to Endpoints News. Merck will be responsible for developing any drugs that arise from the pact. The move comes as Merck makes bold bets on the I&I field, dishing out $10.8 billion to buy Prometheus Biosciences last year and $700 million upfront to buy an early clinical-stage T cell engager from Curon Biopharmaceutical this summer. “We’re also very happy to work with a partner that is very actively and demonstrably building out inflammatory disease and autoimmune discovery capabilities,” Hill said in an interview. For Mestag, the money infusion gives runway to get closer to the clinic. It disclosed a $45 million seed round in 2021 from SV Health Investors, Johnson & Johnson Innovation, GV, Northpond Ventures and Forbion. It also worked on an inflammatory disease collaboration with J&J for undisclosed terms, but that tie-up is “completing” and coming to an end, Hill said. The three dozen-employee startup will likely begin fundraising a Series A in the first half of 2025 so it can get into the clinic with its first experimental drug about 15 months from now, Hill said. The company’s first program, MST-0300, is a bispecific antibody that is meant to agonize lymphotoxin beta receptors in solid tumors and co-engage fibroblast activation protein, or FAP. It aims to induce tertiary lymphoid structures, or TLS, in solid tumors to drum up an immune response to the cancer. “There’s an incredible amount of data in the literature now showing that patients that have TLS in their tumors do better on overall survival and PFS,” Hill said. Behind that project, Mestag has inflammatory and autoimmune programs, including checkpoint agonists that target myeloid biology rather than T cell biology that other hot I&I drug modalities are going after, the CEO said. For example, multiple drugmakers are attempting to turn CAR-T cell therapies and T cell engagers into I&I medicines. Hill said she doesn’t believe there are any other I&I…

Today is Rare Disease Day. Millions of people are affected by often overlooked and under-researched rare diseases. The path to future treatments is complex, and many scientific challenges remain. But I and many others believe that RNA therapeutics hold the promise of precise, targeted treatments. At Abzu, we are acutely aware of the scientific challenges that impede the progress of RNA therapeutics. Our technology enables a deeper understanding of biological interactions, paving the way for more effective, successful, and fast drug development. We are proud to be at the forefront of accelerating the development of innovative RNA-based drugs. Last year, a record 10% of all new drugs were RNA-based short oligos. In 5 years, I see no reason why it shouldn't be 50%, making a real dent in the sad list of currently untreatable diseases.

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

FDA has selected the first participants in this pilot program. The START program has the potential to accelerate development of CBER and CDER products for rare diseases by offering frequent advice and enhance communication on topics such as "clinical study design, choice of control group, fine-tuning the choice of patient population, leveraging nonclinical information and product characterization". Eligible CBER products "must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first decade of life." Eligible CDER products "must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic etiology." #FDA #STARTProgram #RareDiseases #MedicalInnovation

LucidQuest Views >>> How we helped a Top-10 BioPharma company enhance its strategic decision-making in the Sickle Cell Disease space. #Casestudies #casestudy Comment below! >>> lqventures.com

🔬 Unlock New Possibilities with Complement System Humanized Mouse Models! 🧬 The complement system, one of the most ancient components of the immune system, is increasingly recognized for its roles beyond traditional immunity, including maintaining immune homeostasis, supporting development, and regulating synaptic pruning. Since the FDA's approval of eculizumab—the first complement-specific inhibitor—in 2007, interest in complement-targeting therapies has grown exponentially. At Biocytogen, we are driving this innovation forward with a suite of complement system-related humanized mouse models, featuring humanized genes such as C3, C5, and C5AR1/2. These advanced tools enable robust preclinical in vivo evaluation of your drug candidates against human targets, helping you explore the untapped potential of complement system therapeutics with confidence and precision. #ComplementTherapeutics #DrugDevelopment #HumanizedModels #PreclinicalResearch #Biocytogen #ImmuneSystem #Innovation

💡 Kinetiq Therapeutics won a $295,923 award from the Department of Health and Human Services' National Institutes of Health! 🎉 This award is part of the Small Business Innovation Research (SBIR) program, which aims to support innovative research and development projects that have the potential for commercialization. Kinetiq Therapeutics' project focuses on developing a subcutaneous enzyme therapy for Fabry disease, a rare genetic disorder that affects the body's ability to break down certain fatty substances. 🌟 The award is for a Phase I project, which will last for 12 months, starting from July 1, 2024, and ending on June 30, 2025. The goal of this project is to demonstrate the feasibility of Kinetiq Therapeutics' approach, which could potentially transform the current standard of care for Fabry disease and other lysosomal diseases. #SBIR #STTR #KinetiqTherapeutics #FabryDisease #RareDisease #Innovation

LucidQuest Views >>> How we helped a Top-10 BioPharma company enhance its strategic decision-making in the Sickle Cell Disease space. #Casestudies #casestudy Comment below! >>> lqventures.com

Introducing Hornet Therapeutics: Pioneering Treatment for EBV-Driven Diseases 🚀 Exciting news for Hornet Therapeutics, as they emerge from stealth mode with groundbreaking data published in Science! 📰 This marks a significant milestone in the fight against Epstein-Barr Virus (EBV) and its related diseases. 🔑Key Highlights🔑 🐝 Revolutionary Treatment: First-ever small molecule drug for EBV-driven diseases. 🐝 Published Data: Demonstrates decreased EBV latency with IDO-1 inhibition. 🐝 Strategic Partnership: Collaboration with Kyowa Kirin. 🐝 Clinical Trials Ahead: Proof-of-concept studies for EBV-driven PTLD in transplant patients. 🌟 What Makes the Lead Asset (HTX-201) & This Antiviral Approach Significant? 🌟 👉 Addresses a significant unmet need in solid organ transplant populations. 👉 Broad Potential: Future targets include Multiple Sclerosis, Mononucleosis, and long-COVID. #Biotech #MedicalInnovation #EBV #HealthTech #Science #Transplantation #MS #LongCOVID #AntiviralTherapy #Healthcare

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Amgen shelves early-stage bispecific T cell engager: Amgen has stopped development of a Phase 1 bispecific T cell engager, a type of medicine that is core to its oncology portfolio and for which it has trademarked the BiTE acronym. The California drugmaker terminated a clinical investigation of its experimental treatment AMG 794 in patients with certain forms of Claudin 6-positive cancers, including non-small cell lung, epithelial ovarian and malignant solid tumors, according to a July 8 update to the federal trials database. The study, with sites in the US, Australia and Switzerland, ended up enrolling three patients out of a planned 98. In the update to clinicaltrials.gov, Amgen wrote that it “made the business decision to discontinue development of AMG 794. The safety profile of AMG 794 remains unchanged.” In the Claudin 6 landscape, BioNTech has an mRNA-based candidate and a cell therapy targeting CLDN6. Context Therapeutics is developing a CLDN6 and CD3 bispecific antibody. Meanwhile, Mark Alles’ TORL BioTherapeutics expects to start a pivotal trial of its antibody-drug conjugate for CLDN6+ tumors before the end of this year. Amgen’s candidate is still listed on its oncology pipeline, which includes a handful of assets in the class of bispecific T cell engagers. The class of medicines comes with two arms that bind a T cell and a tumor cell to bring them closer, with the ultimate goal of destroying a tumor. The company does not “have additional information to share” beyond the trial entry update, an Amgen spokesperson said in an emailed statement to Endpoints News on Tuesday afternoon. Amgen plans to release its second-quarter update on Aug. 6. Amgen “first pioneered the concept of BiTE with Blincyto,” chief scientific officer Jay Bradner said on an investor call in May, according to an AlphaSense transcript. Blincyto came by way of Amgen’s $1.16 billion acquisition of Micromet in 2012. It also received approval for Imdelltra in a particularly aggressive lung cancer in May. #lucidquest #genetherapy #celltherapy