Janus Henderson Investors

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Ottimo Pharma is a biopharmaceutical company dedicated to developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that simultaneously targets two critical pathways in cancer growth: immune checkpoint inhibition and angiogenesis. By addressing these pathways concurrently, Ottimo Pharma aims to improve treatment outcomes and reduce the burden of cancer on patients and the healthcare system.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

35Pharma is a biopharmaceutical company that designs and develops innovative biologics to address diseases with high unmet medical need. The company applies protein engineering and a deep understanding of transforming growth factor-beta structure-function relationships to create potent ligand traps for cardio-pulmonary and metabolic diseases, including pulmonary hypertension and musculoskeletal disorders.

Forte Biosciences is a biotechnology company specializing in dermatology. Its lead product, FB-401, is a topically applied live biotherapeutic developed with the National Institutes of Health and National Institute of Allergy and Infectious Diseases for treating inflammatory skin diseases.

Metsera is a clinical-stage biopharmaceutical company specializing in developing innovative therapies for obesity and metabolic conditions. It focuses on advancing oral and injectable treatments, integrating proprietary health technology tools for personalized care.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Alpha9 Theranostics is a biopharma company that focuses on radiotherapeutics for the treatment of solid and hematologic malignancies. The company is creating a strong pipeline of radiopharmaceuticals to target solid and hematologic malignancies. Its systematic approach to radiotherapeutic design is fueled by a toolbox of technologies and chemistries, which offers broad potential for expansion into a multitude of oncology targets. Alpha9 Theranostics was founded in 2019 and is headquartered in Vancouver, Canada.

Aktis Oncology is a biotechnology company developing targeted radiopharmaceuticals for treating various solid tumor cancers. Its proprietary platforms generate tumor-targeting agents with optimal properties for alpha radiotherapy, ensuring high tumor penetration and long residence time to maximize tumor elimination while minimizing side effects.

Tenax Therapeutics, Inc. is a specialty pharmaceutical company dedicated to the identification, development, and commercialization of products aimed at addressing cardiovascular and pulmonary diseases with significant unmet medical needs. Headquartered in Morrisville, North Carolina, Tenax focuses on levosimendan, a pharmaceutical product designed to reduce morbidity and mortality in cardiac surgery patients at risk for low cardiac output syndrome. The company is also actively conducting a Phase 2 clinical trial to explore the efficacy of levosimendan in treating pulmonary hypertension associated with heart failure with preserved ejection fraction. Additionally, Tenax is involved in developing novel formulations of imatinib mesylate, a kinase inhibitor with the potential to serve as a disease-modifying therapy for pulmonary arterial hypertension. Founded in 1967, Tenax Therapeutics underscores its commitment to advancing treatments within these critical therapeutic areas.

Entrada Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on treating diseases through the intracellular delivery of biologics. Founded in 2016, the company has developed innovative technologies that facilitate the efficient delivery of proteins, peptides, and nucleic acids, targeting intracellular sites that have traditionally been difficult to access. Entrada's approach includes Intracellular Enzyme Replacement Therapy (IC-ERT) and Endosomal Escape Vehicle (EEV) therapeutics, which aim to improve the therapeutic index by enabling the delivery of a wide range of therapeutics across various organs and tissues. The company's pipeline addresses conditions related to neuromuscular diseases and expands into additional therapeutic areas, positioning Entrada at the forefront of advancing treatment options for patients with devastating illnesses.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Lycia Therapeutics operates as a biotechnology company focused on discovering and developing first-in-class therapeutics using its proprietary Lysosomal Targeting Chimeras (LYTACs) platform. This platform enables the degradation of extracellular and membrane-bound proteins associated with challenging-to-treat diseases like cancer and autoimmune disorders, aiming to provide effective treatments for patients.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Eliem Therapeutics is a clinical-stage biotechnology company focused on developing treatments for nervous system disorders driven by neuronal hyperexcitability. It leverages neuroscience and translational medicine to advance analgesics and other therapies for conditions such as chronic pain, aiming to deliver best-in-class drug candidates that alleviate suffering and improve function. Founded in 2018 and based in Boston, Eliem concentrates on translating scientific insights into clinical solutions to address unmet medical needs in hyperexcitability disorders.

Nkarta Therapeutics is a clinical-stage biopharmaceutical company focused on developing allogeneic, off-the-shelf engineered natural killer (NK) cell therapies. The company aims to harness the inherent power of NK cells to target and destroy tumor cells, offering a potent and better-tolerated alternative to traditional T-cell therapies for a broad range of cancer indications.

Mind Medicine Inc. is a biopharmaceutical company focused on discovering, developing, and deploying psychedelic-inspired medicines and therapies aimed at addressing addiction and mental health issues. Headquartered in New York, the company is building a diverse drug development pipeline that includes innovative treatments derived from psychedelic substances such as Psilocybin, LSD, MDMA, DMT, and an Ibogaine derivative known as 18-MC. Founded in 2010 by JR Rahn, Scott Freeman, Leonard Latchman, and Stephen L. Hurst, MindMed is actively engaged in forming collaborations and conducting clinical trials to further explore the therapeutic potential of these substances in treating brain health disorders.

Crinetics Pharmaceuticals is a clinical-stage company focused on discovering, developing, and commercializing therapeutics targeting peptide hormone receptors for treating rare endocrine diseases and related cancers. Its lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist in Phase I trials for acromegaly and neuroendocrine tumors.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

EnGene specializes in developing a mucosal immunotherapy platform for treating inflammatory bowel disease and diabetes. Its core technology enables localized delivery of immune-modulating proteins to intestinal mucosa using non-integrating biopolymer-based nucleotides, impacting diseases affecting mucosal tissues and facilitating systemic protein release.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Benitec Ltd is a biotechnology company that specializes in developing innovative genetic medicines using its proprietary technology platform, which combines RNA interference with gene therapy. The company focuses on creating therapeutics for chronic and life-threatening diseases, aiming to provide long-lasting gene silencing through a single administration. Among its key product candidates is BB-301, an adeno-associated virus-based gene therapy designed to treat oculopharyngeal muscular dystrophy and chronic hepatitis B virus infection. Benitec's technology, known as ddRNAi, selectively targets and silences specific disease-related genes, offering potential cures for conditions that currently require ongoing treatment or have limited therapeutic options. The company holds over 40 global patents protecting its technology and has an active pipeline of drug development programs addressing various diseases, including hepatitis and cancer-related conditions. Established in 1995, Benitec is headquartered in Hayward, California.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

CG Oncology is a clinical-stage biopharmaceutical company dedicated to developing novel immunotherapies for cancer patients. It specializes in oncolytic immunotherapy, focusing on bladder cancer treatments.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, it focuses on discovering and developing innovative therapies for patients with metabolic diseases, including obesity and diabetes. Utilizing its proprietary Chemotype Evolution technology, Carmot Therapeutics aims to address the underlying causes of these conditions and accelerate the discovery of effective treatments. The company’s portfolio includes several clinical-stage drug candidates, such as CT-388 and CT-996, which are designed as GLP-1 receptor agonists for the treatment of obesity and type 2 diabetes, and CT-868, intended for type 1 diabetes patients who are overweight or obese. Through these efforts, Carmot Therapeutics seeks to create life-changing therapeutics that improve the health outcomes of individuals affected by metabolic diseases.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Founded in 2014, Compass Therapeutics is a clinical-stage biopharmaceutical company developing antibody therapeutics to treat solid tumors and hematological malignancies. Its pipeline includes CTX-471, an agonistic antibody of CD137 for immune cell activation, currently in Phase I trials; CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, in IND-enabling studies; and CTX-009, a bispecific antibody inhibiting DLL4 and VEGF-A signaling, completed Phase I and is in Phase Ib combination with chemotherapy.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Founded in 2019, Enliven Therapeutics is a precision oncology company based in Boulder, Colorado. It focuses on developing small molecule therapies to extend and improve patient lives by targeting clinically validated biological targets with industry-leading chemistry.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Quell's vision is to become a leading engineered Treg cell therapy company. The company will seek to develop next generation engineered T-regulatory cell (Treg) therapies for a range of solid organ transplant and autoimmune conditions. Quell has been founded by Syncona in partnership with six leading experts in the Treg field, cell engineering, solid organ transplantation and autoimmune diseases: Giovanna Lombardi, Professor of Human Transplant Immunology at Kings College London (KCL), Alberto Sanchez-Fueyo, Professor of Hepatology in the Institute of Liver Studies at KCL, Hans Stauss, Professor of Tumour Immunology and Director of the Institute of Immunity and Transplantation at University College London (UCL), Emma Morris, Professor of Clinical Cell & Gene Therapy and Inflammation, Immunity and Immunotherapeutics Theme Director, National Institute for Health Research University College London Hospitals Biomedical Research Centre, Marc Martinez-Llordella, Senior Lecturer in the Institute of Liver Studies at KCL and Elmar Jaeckel, Co-Leader Liver Transplant program MHH, Group Leader "Immune tolerance" in the Department of Gastroenterology, Hepatology and Endocrinology at Hannover Medical School.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Founded in 1999, SomaLogic specializes in the discovery, development, and commercialization of life science research tools and clinical diagnostic products centered around its proprietary SOMAmer/SOMAscan technology. This platform enables protein measurement at scale, facilitating biomarker discovery for various diseases, drug development, and clinical diagnostics.

Sonoma Biotherapeutics, Inc. is focused on developing adoptive T regulatory (Treg) cell therapies aimed at treating autoimmune and degenerative diseases. Established in 2019 and based in South San Francisco, California, with an additional office in Seattle, the company employs next-generation genome editing and target-specific cell therapy techniques to innovate in the field of Treg cell therapeutics. The firm aims to create disease-modifying and potentially curative therapies that promote self-tolerance and inhibit harmful inflammatory responses associated with conditions such as rheumatoid arthritis, inflammatory bowel disease, multiple sclerosis, amyotrophic lateral sclerosis, and Alzheimer's disease. Founded by experts in Treg biology and cell therapy, Sonoma Biotherapeutics integrates advanced methodologies to enhance its therapeutic offerings.

Synthekine Inc. is a biotechnology company based in Menlo Park, California, specializing in the development of engineered cytokine therapies and immunotherapies aimed at treating cancer and autoimmune disorders. Founded in 2018, Synthekine utilizes advanced platform technologies, including engineered partial agonists and orthogonal cell therapies, to enhance the efficacy of cytokines while minimizing their toxic side effects. The company's innovative Synthekine Platform employs surrogate agonists instead of traditional mutant cytokines to create selective immunotherapies. Its product pipeline features candidates such as STK-009, an orthogonal ligand paired with SYNCAR-001, an orthogonal receptor-modified CAR-T therapy targeting CD-19. Additionally, Synthekine is developing STK-012, partial agonists of IL-2, and other novel immunotherapies that integrate cytokine receptors to initiate new signaling pathways without relying on wild-type cytokines.

BioTheryX is a clinical-stage biopharmaceutical company focused on restoring protein homeostasis through targeted protein degradation. It specializes in developing innovative small molecule degraders, initially targeting cancer, using its proprietary PRODEGY platform built upon expertise in Cereblon binding.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Nuvalent is a biotechnology company focused on developing targeted therapies for clinically proven kinase targets in cancer. Leveraging expertise in structure-based design, Nuvalent creates innovative small molecules with exquisite target selectivity to overcome resistance and minimize adverse events.

TwinStrand Biosciences, established in 2015 and based in Bellevue, Washington, specializes in advancing DNA sequencing technology. The company's core innovation, Duplex Sequencing, enhances sequencing accuracy by over 10,000 times, enabling the detection of previously invisible mutations. This breakthrough technology has wide-ranging applications, including oncology, infectious disease diagnosis, organ transplant monitoring, genetic health, and forensics.

Janux Therapeutics develops immunotherapies that target tumors without harming healthy tissue. Its proprietary technology enables two distinct bispecific platforms: Tumor Activated T Cell Engagers (TRACTr) and Tumor Activated Immunomodulators (TRACIr), designed to stimulate the immune system's response against cancer.

ValenzaBio focuses on developing safe and effective treatments for patients with autoimmune and inflammatory diseases. The company employs biomarker-driven approaches to guide its research and clinical development. Its pipeline features several monoclonal antibodies designed to target surface antigens, aiming to reduce pathogenic subpopulations of autoreactive cells. ValenzaBio's most advanced program has achieved Phase 1 proof-of-concept in patients, while additional preclinical programs are exploring differentiated mechanisms of action. The organization is committed to addressing the unmet needs of patients through innovative therapeutic options.

Icosavax develops virus-like particle (VLP) vaccines using its proprietary computational design platform. Its primary focus is creating vaccines for life-threatening respiratory diseases, with current candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Founded in 2017, the company aims to discover, develop, and commercialize vaccines against infectious diseases.

Tyra Biosciences is a biotechnology company focused on developing precision therapies for cancer. It uses its proprietary SNAP platform to create next-generation drug candidates that target acquired drug resistance, with an initial focus on the Fibroblast Growth Factor Receptor (FGFR) family.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Viracta Therapeutics, Inc. is a clinical-stage drug development company based in Cardiff, California, that specializes in precision oncology focused on virus-associated malignancies. The company utilizes a viral activation therapy platform to develop treatments for cancers linked to the Epstein-Barr Virus (EBV) and other virus-related diseases. Its lead investigational drug, nanatinostat, is being evaluated in combination with the antiviral valganciclovir as a potential oral therapy for EBV-positive lymphoma, including post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma. Viracta is also exploring the application of this approach to other EBV-associated conditions, such as nasopharyngeal carcinoma and gastric carcinoma. Founded in 2007 and rebranded from HemaQuest Pharmaceuticals in 2015, Viracta Therapeutics is actively conducting multiple clinical trials to advance its therapeutic candidates.

Centessa Pharmaceuticals is a biopharmaceutical company that focuses on advancing validated drug programs through an asset-centric R&D model. Each program is developed by dedicated subsidiaries, supported by centralized infrastructure and expert management.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Founded in 2008 and headquartered in Charlottesville, Virginia, HemoShear Therapeutics develops treatments for rare metabolic disorders, including organic acidemias and nonalcoholic steatohepatitis. The company employs its proprietary REVEAL‑Tx platform to create human‑relevant systems that replicate organ biology and disease states, providing detailed insights into disease mechanisms. By generating meaningful human response data, HemoShear aims to reduce the risk of costly failures and accelerate the discovery of effective therapies. It collaborates with pharmaceutical, biotechnology, and medical device partners to advance drug development programs across liver diseases, gout, and other metabolic conditions.

Day One Biopharmaceuticals is a clinical-stage biotechnology company focused on developing targeted therapies for genetically defined cancers across all ages, with an initial emphasis on pediatric patients. The company aims to bring effective cancer treatments to families by advancing therapies that can benefit both children and adults, prioritizing programs that can progress to the market rapidly. Day One pursues licensing or acquisition of promising products from research institutions and industry partners to expand its pipeline. Its lead candidate, DAY101, is an oral, brain-penetrant, highly selective type II pan-RAF kinase inhibitor designed to target cancers driven by the RAF signaling pathway. By applying insights from childhood cancer biology, the company seeks to address unmet needs in oncology and broaden access to precision therapies.

Inivata Limited is a clinical cancer genomics company based in Cambridge, United Kingdom, that specializes in the analysis of circulating tumor DNA (ctDNA) to enhance cancer testing and treatment. Founded in 2014, Inivata has developed TAm-Seq technology, which enables the detection and analysis of genomic material from a patient's cell-free ctDNA, collected through non-invasive blood samples. This innovative approach, known as liquid biopsy, provides a significant advancement over traditional invasive biopsies by allowing for easier detection, monitoring, and treatment of cancer. Inivata's platform aims to assist oncologists in making informed treatment decisions by improving the understanding of cancer dynamics, including monitoring treatment responses and detecting relapses. The company has established a strategic collaboration with NeoGenomics, Inc. to further its mission in the field of cancer genomics.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Founded in 1999, SomaLogic specializes in the discovery, development, and commercialization of life science research tools and clinical diagnostic products centered around its proprietary SOMAmer/SOMAscan technology. This platform enables protein measurement at scale, facilitating biomarker discovery for various diseases, drug development, and clinical diagnostics.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

Elevation Oncology develops precision medicines for genomically defined cancers. Its lead candidate, seribantumab, inhibits tumor growth driven by NRG1 fusions, currently in Phase 2 trials.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

Galecto is a clinical-stage biotechnology company developing small molecule therapeutics targeting galectin-3 and lysyl oxidase-like 2 for treating severe diseases like fibrosis and cancer. Its lead product candidate, GB0139, is an inhaled inhibitor of galectin-3 for treating severe fibrotic lung diseases such as idiopathic pulmonary fibrosis.

Eledon Pharmaceuticals is a clinical-stage biotechnology company focused on developing treatments for patients undergoing organ or cellular transplantation and those with autoimmune and neurodegenerative diseases. Its lead compound, tegoprubart, is an anti-CD40L antibody targeting the CD40L pathway.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Chinook Therapeutics, Inc. is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently prepared for Phase III trials targeting IgA nephropathy and other primary glomerular diseases. In addition to atrasentan, Chinook is developing BION-1301, an investigational anti-APRIL monoclonal antibody evaluated in a Phase Ib trial for IgA nephropathy, and CHK-336, a preclinical candidate aimed at treating an undisclosed ultra-orphan kidney disease. Chinook Therapeutics is also engaged in research programs addressing other rare and severe chronic kidney conditions, including polycystic kidney disease.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Shattuck Labs is a clinical-stage biotechnology company focused on developing therapeutics for cancer and autoimmune diseases. Its proprietary Agonist Redirected Checkpoint (ARC) platform enables the creation of novel fusion proteins with applications in oncology and immunology. The company's lead program, SL-279252, is being studied in Phase I trials for advanced solid tumors and lymphoma, in collaboration with Takeda Pharmaceuticals.

Everest Medicines is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for critical unmet medical needs, initially focusing on Asia Pacific markets.

iTeos Therapeutics is a biotechnology company focused on immuno-oncology, developing cancer therapies that target the tumor microenvironment to overcome immune suppression and enhance efficacy of existing treatments. The company pursues small molecule and antibody approaches, including EOS-850, a small molecule antagonist of the adenosine A2A receptor, and EOS-448, an antagonist of TIGIT, with early-stage clinical programs designed to restore immune activity against tumors. By modulating metabolic and immune pathways within the tumor microenvironment, iTeos aims to improve responses across cancer types and complement immune checkpoint therapies.

Kymera Therapeutics is a biotechnology company focused on targeted protein degradation. It employs a proprietary predictive modeling platform to discover novel small molecule therapeutics that selectively degrade disease-causing proteins.

Vaxcyte is a biotechnology company developing vaccines to prevent or treat infectious diseases worldwide. Its lead product is VAX-24, a 24-valent pneumococcal conjugate vaccine candidate. The company also develops vaccines targeting emerging strains of pneumococcal disease (VAX-XP), Group A Strep infections (VAX-A1), and periodontal disease (VAX-PG).

ALX Oncology is a clinical-stage immuno-oncology company based in Burlingame, California, established in 2015. The company specializes in developing innovative therapies aimed at treating various forms of cancer, with a particular focus on its lead product candidate, ALX148. This therapeutic agent is designed to block the CD47 protein, which cancer cells exploit to evade the immune system. ALX148 is currently being investigated for its efficacy in treating myelodysplastic syndromes, acute myeloid leukemia, and several solid tumor types, including head and neck squamous cell carcinoma and certain gastric cancers. The company leverages advanced protein engineering technologies to enhance the affinity of its CD47 blockers, aiming to minimize hematologic toxicities often associated with existing treatments. ALX Oncology's commitment to improving cancer therapies is rooted in pioneering research conducted by its founders at Stanford University.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

IGM Biosciences, Inc. is a biotechnology company that specializes in the research and development of engineered Immunoglobulin M (IgM) antibodies aimed at treating cancer and other diseases. The company’s lead product candidate, IGM-2323, is a bispecific IgM antibody currently undergoing Phase 1 clinical trials for the treatment of relapsed or refractory B cell Non-Hodgkin's lymphoma. In addition, IGM is developing IGM-8444, an IgM antibody targeting Death Receptor 5 for solid and hematologic malignancies, and IGM-7354, a bispecific IgM antibody designed to deliver interleukin-15 cytokines to PD-L1 expressing cells. IGM Biosciences has established collaborations focused on the discovery and development of novel IgM and IgA antibodies, including partnerships for potential COVID-19 treatments. Founded in 1993 and headquartered in Mountain View, California, the company was previously known as Palingen, Inc. and adopted its current name in 2010.

Phathom Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Buffalo Grove, Illinois, that specializes in developing and commercializing innovative treatments for gastrointestinal diseases. Incorporated in 2018, the company has rights in the United States, Europe, and Canada for vonoprazan, a potassium-competitive acid blocker (P-CAB) designed to inhibit acid secretion in the stomach. Currently, vonoprazan is undergoing Phase III clinical trials for the treatment of erosive gastroesophageal reflux disease and is also being evaluated in combination with antibiotics for addressing Helicobacter pylori infection. Phathom Pharmaceuticals aims to meet the unmet medical needs of patients suffering from acid-related disorders by leveraging the expertise of its team, which comprises seasoned professionals from the gastrointestinal and pharmaceutical sectors.

Aprea Therapeutics, Inc. is a biopharmaceutical company based in Boston, Massachusetts, that specializes in developing cancer therapeutics aimed at reactivating the mutant p53 tumor suppressor protein. Its primary product candidate, APR-246, is a small molecule p53 reactivator currently undergoing late-stage clinical development for hematologic malignancies, particularly myelodysplastic syndromes and acute myeloid leukemia. Founded in 2006, Aprea is focused on advancing innovative treatments that target critical mechanisms in cancer biology to improve patient outcomes.

Sojournix Inc., established in 2016 and headquartered in Waltham, Massachusetts, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for women's health and neuroendocrine disorders. Its primary focus is SJX-653, a selective neurokinin-3 (NK3) antagonist, designed as a non-hormonal treatment option for moderate to severe vasomotor symptoms associated with menopause.

Akero Therapeutics is a clinical-stage biotechnology company focused on developing treatments for serious metabolic diseases, with a primary emphasis on nonalcoholic steatohepatitis (NASH). Its lead program is a long-acting fibroblast growth factor 21 (FGF21) analog designed to restore metabolic balance and address liver fat, inflammation, and fibrosis. The company was founded in 2017 and is headquartered in South San Francisco, California.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

BioNTech is a Germany-based biotechnology company focused on developing cancer therapeutics and vaccines for infectious diseases. Its portfolio includes mRNA-based therapies to encode antigens and neoantigens, cytokines, and antibodies; cell therapies; bispecific antibodies; and antibody-drug conjugates. The company maintains an oncology pipeline and collaborates on vaccines for infectious diseases, including a COVID-19 vaccine developed with Pfizer.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.