Medicxi

MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.

Xenikos B.V. is a biotechnology company based in Nijmegen, the Netherlands, focused on developing innovative therapies for T-cell mediated diseases. Founded in 2009, the company specializes in toxin-loaded anti-T-cell antibodies that serve as a therapeutic tool to reset the immune system. These antibodies are designed to specifically identify and eliminate adult T cells, particularly the activated ones, which is crucial for treating serious immune conditions and preventing rejection after organ transplantation. By advancing its immunotherapy medicines, Xenikos aims to enhance treatment outcomes and improve the health and well-being of patients suffering from severe immune-related disorders.

Versanis Bio focuses on developing therapies to address health issues prevalent among older adults, particularly obesity and its associated complications. The company's lead product, bimagrumab, is a human monoclonal antibody that targets activin type II receptors, effectively blocking the binding of ligands such as activin A and myostatin. This treatment has undergone extensive clinical evaluation, with over 1,500 participants in various controlled trials previously conducted by Novartis. Bimagrumab has shown the ability to induce significant fat loss without compromising lean muscle mass, while also improving important cardiometabolic markers such as HbA1c. Through its innovative approach, Versanis Bio aims to help overweight and obese adults achieve and maintain a healthier body composition.

Sydnexis specializes in developing a proprietary treatment for pediatric progressive myopia, addressing a significant global medical need. The company manufactures a topical eyedrop formulation aimed at treating this condition in children, allowing healthcare professionals to effectively manage and reduce the progression of myopia. Through its innovative approach, Sydnexis seeks to enhance eye care for pediatric patients and mitigate the prevalence of eye disorders in this demographic.

Rivus Pharmaceuticals is a biopharmaceutical company focused on enhancing cardio-metabolic health through the development of controlled metabolic accelerators (CMAs). These innovative therapeutics aim to improve cellular metabolism and target the root causes of prevalent metabolic and cardiovascular diseases. By providing oral small-molecule treatments, Rivus addresses critical metabolic risk factors associated with cardiovascular mortality and morbidity, thereby contributing to better health outcomes for patients suffering from these conditions.

Kurome Therapeutics, Inc. develops a series of inhibitors for treating cancer. Its therapy target cancer cells that have co-opted immune signaling pathways in order to avoid destruction by traditional therapeutic agents and subvert adaptive resistance mechanisms. The company helps in treating hematological cancers. Kurome Therapeutics, Inc. was incorporated in 2019 and is based in Cincinnati, Ohio.

Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.

Janpix Ltd. is a biopharmaceutical company focused on developing innovative treatments for cancer through the discovery and manufacture of inhibitors targeting signal transducer and activator of transcription (STAT) proteins. Founded in 2017 and headquartered in Cambridge, Massachusetts, with a research facility in Mississauga, Canada, Janpix specializes in ultra-selective STAT inhibitors aimed at treating hematological tumors and glioblastomas. By modulating immune cells and the tumor microenvironment, the company's inhibitors seek to enhance cancer treatment efficacy and improve patient outcomes. Janpix's efforts are underscored by the challenge of effectively inhibiting STAT proteins, particularly STAT3 and STAT5, which have been implicated in various cancers.

Palladio Biosciences, Inc. is a biotechnology company focused on developing transformative medicines for orphan diseases of the kidney. Founded in 2015 and based in Horsham, Pennsylvania, the company is in the clinical stage and specializes in therapeutics aimed at renal diseases, particularly polycystic kidney disease (PKD). One of its key developments is Lixivaptan, a selective vasopressin V2 receptor antagonist designed to treat PKD, a life-threatening genetic disorder characterized by the growth of numerous fluid-filled cysts in the kidneys. Palladio Biosciences aims to provide innovative treatment options that can help prevent the progression of kidney diseases.

Checkmate Pharmaceuticals is a clinical-stage biotechnology company based in Cambridge, Massachusetts, that specializes in developing novel immunotherapies for cancer treatment. Founded in 2015, the company focuses on leveraging CpG oligonucleotides to enhance anti-tumor T-cell responses and overcome the mechanisms that allow tumors to evade immune detection. By combining its proprietary technology with checkpoint inhibition, Checkmate aims to improve the efficacy of existing immunotherapies and provide new treatment options for patients. The company has formed strategic alliances with major pharmaceutical firms, including Merck KGaA and Pfizer, to further its research and development efforts in the field of cancer immunotherapy.

Vaxcyte, Inc., a preclinical-stage biotechnology vaccine company, develops vaccines to prevent or treat infectious diseases worldwide. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine. The company also develops VAX-XP to protect against emerging strains causing significant invasive pneumococcal disease and antibiotic resistance; VAX-A1, a conjugate vaccine candidate for protection against subtypes of Group A Strep; and VAX-PG, a novel protein vaccine candidate targeting porphyromonas gingivalis. The company was formerly known as SutroVax, Inc. and changed its name to Vaxcyte, Inc. in May 2020. Vaxcyte, Inc. was founded in 2013 and is headquartered in Foster City, California.

Alderaan Biotechnology is a Biotechnology company develops antibodies targeting new and original targets in immuno-oncology, expressed by NK cells in particular.

Divide & Conquer is a biotechnology company based in Cambridge, United Kingdom, established in 2018. The company specializes in developing therapeutics for glioblastoma, a highly aggressive form of brain cancer, as well as other solid tumors with poor prognoses, including breast, pancreatic, prostate, lung, and colorectal cancers. It collaborates with the University of Heidelberg and the German Cancer Research Center to advance its research and development efforts. Funded by the venture capital firm Medicxi, Divide & Conquer aims to create innovative treatments that address significant unmet medical needs in oncology.

Breakpoint Therapeutics GmbH, located in Hamburg, Germany, specializes in the discovery and development of innovative anti-cancer drugs. The company focuses on targeting the DNA damage response (DDR) in cancer cells, specifically aiming to disrupt pathways related to DNA repair and replication stress tolerance. By doing so, Breakpoint Therapeutics seeks to advance first-in-class oncology drugs that not only complement existing treatments, such as PARP inhibitors, but also address the challenge of therapy-resistant cancers. Through its research and development efforts, the company aspires to enhance treatment options for patients facing difficult-to-treat cancer types.

Yukin Therapeutics is a French biotechnology company. The company focuses on the development of new therapeutic molecules, entitled kinase inhibitors, specifically targeting a protein involved in the development of several types of cancer: the NIK protein (NF-κB inducing kinase). Yukin Therapeutics led studies that demonstrated proof of concept in animal models and is now actively seeking to identify a first drug candidate to launch a clinical trial in oncology.

Phathom Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada to vonoprazan, a potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach. It is also developing vonoprazan, which is in Phase III clinical trials for the treatment of erosive gastroesophageal reflux disease; and in combination with antibiotics for the treatment of Helicobacter pylori infection. The company was incorporated in 2018 and is based in Buffalo Grove, Illinois.

Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.

Orexia Limited is a biotechnology company based in London, United Kingdom, focused on developing innovative therapies for neurological diseases. Incorporated in 2018, the company specializes in oral orexin positive modulators aimed at treating narcolepsy, a rare neurological condition that disrupts the brain's regulation of the sleep-wake cycle. By targeting the G protein-coupled receptors Orexin OX1 and OX2, Orexia seeks to restore orexin levels in the brain, thereby addressing the symptoms associated with this disorder.

Inexia aims to develop intranasally administered orexin positive modulators for the treatment of neurological diseases.The orexin system is a key regulator of behavioural arousal, wakefulness and sleep. The loss of the orexin neurons has been shown to be strongly linked to multiple neurological conditions including narcolepsy. In this indication, orexin receptors remain intact and functional, providing an opportunity for therapeutic intervention.

Morphogen-IX Limited is a biotechnology company based in Cambridge, United Kingdom, focused on developing novel treatments for pulmonary arterial hypertension (PAH), a serious condition affecting lung blood vessels that can lead to heart failure. Founded in 2015 by Index Ventures, the company is exploring the therapeutic potential of bone morphogenetic proteins (BMPs), particularly BMP9 and BMP10, which have shown promise in enhancing BMPR-II receptor signaling in endothelial cells. This signaling is crucial for reversing the effects of PAH, as compromised BMPR-II signaling is central to the disease's pathology. Current PAH treatments primarily address symptoms, and effective long-term solutions are limited, often requiring risky lung transplants. Morphogen-IX employs a semi-virtual business model, leveraging a network of experienced professionals in drug discovery while maintaining operational efficiency through outsourcing. The company's founders are recognized experts in both PAH and BMP research, positioning Morphogen-IX to potentially transform the treatment landscape for this life-limiting disease.

Vaxcyte, Inc., a preclinical-stage biotechnology vaccine company, develops vaccines to prevent or treat infectious diseases worldwide. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine. The company also develops VAX-XP to protect against emerging strains causing significant invasive pneumococcal disease and antibiotic resistance; VAX-A1, a conjugate vaccine candidate for protection against subtypes of Group A Strep; and VAX-PG, a novel protein vaccine candidate targeting porphyromonas gingivalis. The company was formerly known as SutroVax, Inc. and changed its name to Vaxcyte, Inc. in May 2020. Vaxcyte, Inc. was founded in 2013 and is headquartered in Foster City, California.

Xenikos B.V. is a biotechnology company based in Nijmegen, the Netherlands, focused on developing innovative therapies for T-cell mediated diseases. Founded in 2009, the company specializes in toxin-loaded anti-T-cell antibodies that serve as a therapeutic tool to reset the immune system. These antibodies are designed to specifically identify and eliminate adult T cells, particularly the activated ones, which is crucial for treating serious immune conditions and preventing rejection after organ transplantation. By advancing its immunotherapy medicines, Xenikos aims to enhance treatment outcomes and improve the health and well-being of patients suffering from severe immune-related disorders.

Synthorx, Inc. is a biopharmaceutical company based in La Jolla, California, that specializes in developing innovative protein therapeutics through synthetic biology. The company focuses on creating novel cytokine Synthorin programs aimed at treating cancer and autoimmune disorders. Its lead candidate, THOR-707, is a modified version of recombinant human IL-2 being tested for various solid tumors, both as a monotherapy and in combination with immune checkpoint inhibitors. Additionally, Synthorx is developing Synthorins for other indications, including IL-10 for immuno-oncology and IL-15 as an immunoregulatory cytokine. The company leverages proprietary engineered organisms to produce these advanced proteins with high fidelity, enhancing their efficacy, safety, and convenience for patients. Founded in 2014 with technology licensed from The Scripps Research Institute, Synthorx has attracted investment from various venture capital firms and operates as a subsidiary of Sanofi.

Critical Pressure is a provider of drugs designed to treat to treat vascular dysfunction and shock. The company's technology has the potential to change clinical practice without the potentially harmful effects of excess fluid administration and toxicities associated with the currently available catecholamine-based vasoactive agents, providing the possibility of supportive therapy during the critical period of haemodynamic compromise.

Impact Biomedicines, Inc. engages in the research, development, and commercialization of treatments for patients with myeloproliferative neoplasms and other cancers. Its product under late-stage development pipeline is fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor for the treatment of myelofibrosis and polycythemia vera. The company was founded in 2016 and is headquartered in San Diego, California. As of February 28, 2018, Impact Biomedicines, Inc. operates as a subsidiary of Bristol-Myers Squibb Company

Janpix Ltd. is a biopharmaceutical company focused on developing innovative treatments for cancer through the discovery and manufacture of inhibitors targeting signal transducer and activator of transcription (STAT) proteins. Founded in 2017 and headquartered in Cambridge, Massachusetts, with a research facility in Mississauga, Canada, Janpix specializes in ultra-selective STAT inhibitors aimed at treating hematological tumors and glioblastomas. By modulating immune cells and the tumor microenvironment, the company's inhibitors seek to enhance cancer treatment efficacy and improve patient outcomes. Janpix's efforts are underscored by the challenge of effectively inhibiting STAT proteins, particularly STAT3 and STAT5, which have been implicated in various cancers.

Sydnexis specializes in developing a proprietary treatment for pediatric progressive myopia, addressing a significant global medical need. The company manufactures a topical eyedrop formulation aimed at treating this condition in children, allowing healthcare professionals to effectively manage and reduce the progression of myopia. Through its innovative approach, Sydnexis seeks to enhance eye care for pediatric patients and mitigate the prevalence of eye disorders in this demographic.

Diasome Pharmaceuticals, Inc. is a clinical stage company focused on developing innovative diabetes therapeutics. The company specializes in insulin-based therapies, notably its hepatocyte directed vesicle (HDV), which acts as a nanoscale carrier delivering insulin directly to the liver. This technology allows for the delivery of 100 insulin molecules per disc and is compatible with various insulin delivery methods, including insulin pumps. In addition to HDV, Diasome offers products such as Oral HVD-I, a targeted oral insulin, and both injectable short-acting (HDV-I) and long-acting (HDV-B) insulins. The company aims to improve treatment options for patients with Type 1 and Type 2 diabetes. Founded in 2004, Diasome Pharmaceuticals is based in Conshohocken, Pennsylvania.

Palladio Biosciences, Inc. is a biotechnology company focused on developing transformative medicines for orphan diseases of the kidney. Founded in 2015 and based in Horsham, Pennsylvania, the company is in the clinical stage and specializes in therapeutics aimed at renal diseases, particularly polycystic kidney disease (PKD). One of its key developments is Lixivaptan, a selective vasopressin V2 receptor antagonist designed to treat PKD, a life-threatening genetic disorder characterized by the growth of numerous fluid-filled cysts in the kidneys. Palladio Biosciences aims to provide innovative treatment options that can help prevent the progression of kidney diseases.

Z Factor Limited is a drug discovery company based in Cambridge, United Kingdom, established in 2014. The company focuses on identifying and developing therapeutic agents to address alpha-1-antitrypsin deficiency, a genetic disorder that leads to significant health complications. Z Factor's research aims to design novel therapeutic solutions that correct the misfolding of the Z variant of alpha-1-antitrypsin, addressing a critical medical need. Current treatment options are limited, primarily relying on lung or liver transplantation and augmentation therapy. Through its innovative approach, Z Factor seeks to create effective therapies that could improve the quality of life for those affected by this condition.

SuperX is a preclinical biopharmaceutical company based at the Babraham Research Campus, focused on developing novel anticoagulant therapies. The company is engaged in the discovery of antibodies targeting components of the blood coagulation cascade, aiming to create anti-thrombotic drugs that reduce the risk of bleeding. By specifically blocking thrombosis, which is a leading cause of heart attacks and strokes, SuperX seeks to provide healthcare institutions and physicians with effective anticoagulation treatments suitable for chronic use. Through its innovative approach, the company aspires to improve patient outcomes in the management of cardiovascular diseases.

ApcinteX develops and markets anticoagulant APC pathway to treat haemophilia. Apcintex has developed a new treatment that seeks to turn down the activity of a key natural anticoagulant pathway to produce normal blood clotting in patients with haemophilia. This means that the drug could potentially treat patients with all types of haemophilia, including those who develop antibodies to replacement factors. Furthermore, the drug does not cause anti-clotting antibodies to form and could be administered fortnightly by simple injection under the skin.

Mavalon Therapeutics is a company focused on stopping the progression of Parkinson’s disease. The biotechnology company is developing mGluR3 allosteric modulators for the treatment of Parkinson's disease.

Kymo Therapeutics’s ambition is to move forward Metabrain Research’s proprietary program on kynurenine metabolism (KMO) inhibitors towards clinical proof of concept, before seeking partners to complete development and ensure commercialization.

Gadeta B.V. is a research and development company based in Utrecht, the Netherlands, focused on creating immunotherapies for cancer. Founded in 2015, Gadeta has developed a technology platform utilizing combinatorial T Cell receptor exchange (CTE) to create optimized gamma delta (γδ) T-cell receptors (TCRs) that enhance anti-cancer reactivity. The company’s key product, T Cells Engineered To Express A Defined Gamma Delta TCR (TEGs), shows strong therapeutic potential against both hematological and solid tumors by employing high-affinity γδ TCRs expressed on αβ T cells, which possess superior proliferation capabilities. Gadeta has also established a strategic collaboration with Kite Pharma to further advance its innovations in cancer treatment.

Capella Bioscience Limited is involved in the discovery and development of monoclonal antibodies (mAbs) for therapeutic applications. The company utilizes proprietary technologies to create and commercialize mAb-based medicines targeting oncology, autoimmune diseases, and other conditions. Founded in 2014 and headquartered in London, the firm operates both independently and in collaboration with biotechnology firms and academic institutions to advance its research and development efforts. Through its innovative approach, Capella Bioscience aims to contribute to the development of advanced medical therapies that address significant health challenges.

Stealthyx Therapeutics Ltd. focuses on developing a patented drug-targeting platform designed for precise drug delivery at disease sites. The company’s innovative platform utilizes a fusion protein that combines a protective shell-like structure with a pharmacologically active agent connected by an enzyme-sensitive cleavage site. This technology enables targeted release of therapeutic agents, such as cytokines, small peptides, and antibody fragments, specifically at locations affected by disease, while minimizing toxicity and enhancing the effectiveness of the treatment. Founded in 2002 and based in Babraham, United Kingdom, Stealthyx Therapeutics aims to improve the pharmacokinetic and pharmacodynamic properties of biological agents, thereby advancing therapeutic applications in medicine.