Janus Henderson Investors

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

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.

Bambusa Therapeutics operates as biotechnology company, that focuses on developing bispecific antibodies.

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.

nChroma Bio is a biotechnology company specializing in targeted genetic medicine. It operates an integrated product engine for safe, accurate, and targeted in vivo administration, aiming to overcome limitations of current genetic medicine techniques. The company focuses on liver-targeted therapies, initially for chronic hepatitis B and D, leveraging epigenetics to enable precise and specific gene regulation. This approach allows for therapies with programmable tissue specificity, potentially offering durable and targeted cures for the liver and other organs.

Angitia Biopharmaceuticals is a global biotechnology company dedicated to the discovery and development of innovative therapeutics targeting serious musculoskeletal diseases. The company specializes in researching and developing breakthrough therapies for conditions affecting bones, muscles, and joints. By focusing on creating novel drugs, Angitia aims to provide effective treatments that enhance patient recovery from severe musculoskeletal ailments.

Maze Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, established in 2017. The company specializes in developing genetic modifier therapeutics aimed at addressing unmet medical needs. Utilizing its innovative Compass platform, Maze Therapeutics focuses on identifying rare genetic variants that offer protective effects against diseases. By analyzing large-scale human genetic data and employing functional genomics, the company maps these variants to the biological pathways that contribute to specific conditions in patient populations. This approach enables a deeper understanding of target biology and facilitates the development of novel therapies, positioning Maze Therapeutics as a key player in translating genetic insights into effective medicines.

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 focused on designing and developing innovative biologics to address diseases with high unmet medical needs, particularly pulmonary hypertension and musculoskeletal disorders. The company leverages its expertise in protein engineering and its understanding of transforming growth factor-beta (TGF-beta) structure-function relationships to create highly potent ligand traps. These developments aim to enhance treatment options for patients suffering from cardio-pulmonary and metabolic diseases. By combining advanced scientific knowledge with a commitment to addressing critical health challenges, 35Pharma seeks to contribute significantly to the biopharmaceutical landscape.

Forte Biosciences is a biotechnology company specializing in dermatology and biopharmaceuticals. The company's lead product, FB-401, is a topically applied live biotherapeutic designed to treat inflammatory skin diseases, developed in collaboration with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte Biosciences is advancing another product candidate, FB-102, which is a proprietary therapeutic molecule aimed at addressing a range of autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata. The company is currently in the preclinical trial stage for FB-102, reflecting its commitment to developing innovative treatments for dermatological and autoimmune disorders.

Metsera is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for obesity and metabolic conditions. The company focuses on advancing a variety of treatment options, including both oral and injectable therapies, specifically targeting incretin, non-incretin, and combination approaches. By integrating proprietary health technology tools, Metsera aims to provide personalized care that meets the evolving demands of weight loss treatment. Its commitment to addressing multiple therapeutic targets positions the company to significantly impact the future of obesity management and related metabolic diseases.

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 focused on the discovery and development of a new class of targeted radiopharmaceuticals aimed at treating various solid tumor cancers. The company has developed proprietary platforms to create tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered for high tumor penetration and prolonged residence time, allowing for effective tumor elimination while reducing the side effects typically associated with cancer treatments. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing treatment precision and efficacy.

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.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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 gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

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, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, that specializes in developing therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating innovative treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact millions of individuals. Eliem utilizes its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, including its lead candidate, ETX-155, aimed at addressing major depressive disorder and focal onset seizures. The company's mission centers on meeting unmet medical needs and improving patients' quality of life through its novel therapies.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

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.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Crinetics Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company headquartered in San Diego, California, dedicated to the discovery, development, and commercialization of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials aimed at treating acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is developing several other therapeutics, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, and an oral somatostatin type 5 receptor agonist for congenital hyperinsulinism. The company is also working on an oral adrenocorticotrophic hormone antagonist for conditions such as Cushing’s syndrome and congenital adrenal hyperplasia. Founded in 2008, Crinetics Pharmaceuticals is focused on leveraging its internal discovery efforts to create effective treatments while utilizing hormonal biomarker endpoints to enhance the efficiency and cost-effectiveness of its clinical studies.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for serious diseases that currently lack approved therapies. Established in 2016 and based in New York, the company is advancing a pipeline of novel product candidates that target high unmet medical needs, including cardiovascular diseases and metabolic disorders. Its lead candidate, AT-007, is in Phase I/II trials aimed at treating galactosemia. Additionally, AT-001 is undergoing Phase II trials for diabetic cardiomyopathy and Phase I trials for diabetic peripheral neuropathy, while AT-003 is in Phase I trials for diabetic retinopathy. The company also has preclinical candidates such as AT-104, intended for orphan hematological oncology conditions, demonstrating its commitment to addressing critical health challenges through targeted therapeutics.

enGene, Inc. is a biotechnology company based in Vancouver, Canada, specializing in mucosal immunotherapy to address inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables the localized delivery of immune-modulating proteins to mucosal tissues, targeting conditions affecting the gastrointestinal tract, lung, and bladder. This innovative platform allows for the systemic release of proteins from the gut, which can be beneficial for treating a variety of immune disorders, as well as other conditions such as diabetes, anemia, and hemophilia. Founded in 1999, enGene has formed a strategic alliance with Takeda Pharmaceutical Company to further advance its therapeutic offerings.

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.

Claris Biotherapeutics develops a topical formulation intended to cure corneal wounds, ulcers, and scars. The formulation creates a recombinant hepatocyte growth factor which accelerates the healing process, providing healthcare providers noninvasive therapeutic treatment.

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.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

AN2 Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Menlo Park, California, focused on the research, development, and commercialization of innovative medicines for infectious diseases. Founded in 2017, the company aims to address significant unmet medical needs through its development of novel therapeutics. Its lead product candidate, epetraborole, is a once-daily oral treatment designed for patients suffering from chronic non-tuberculous mycobacterial lung disease. AN2 Therapeutics is committed to leveraging advanced biomedical expertise to create transformative solutions for patients dealing with serious and rare infectious diseases. The company has also established a strategic partnership with Brii Biosciences to enhance its development efforts.

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, formerly known as Cold Genesys, is a privately held clinical-stage biopharmaceutical company specializing in the development of innovative oncolytic immunotherapies aimed at treating cancer. The company focuses on creating therapies that offer alternatives to conventional cancer treatments, particularly for patients with bladder cancer. Its lead candidate, cretostimogene grenadenorepvec, is a targeted intravesical immunotherapy agent designed to be delivered directly to the bladder. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials addressing high-risk and intermediate-risk non-muscle invasive bladder cancer (NMIBC), as well as a phase two study in combination with a checkpoint inhibitor for high-risk NMIBC. Through its research and clinical endeavors, CG Oncology aims to provide effective and less invasive treatment options for bladder cancer patients.

Immunome Inc., founded in 2006 and headquartered in Exton, Pennsylvania, is a biotechnology company focused on discovering, developing, and commercializing highly targeted and native human cancer antibodies against universal cancer antigens. The company utilizes its proprietary RealMAb technology to identify novel antigens and the corresponding native human antibodies that target them. Additionally, Immunome employs ScreenMab multiplex functional screening technology to pinpoint monoclonal antibodies with high specificity for tumor neoantigens.

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 focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Entact Bio is a biotechnology company that is in the preclinical stages of developing medications to improve protein function. It was founded by a team with deep roots in deubiquitinase (DUB) biochemistry, chemical biology, disease biology, and small-molecule drug development. The company's proprietary EncompassTM platform was designed to create enhancement-targeting chimeric (ENTACTM) medicines. ENTACs take advantage of DUBs' ability to regulate proteins.

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.

Compass Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative antibody therapeutics for the treatment of solid tumors and hematological malignancies. Founded in 2014 and headquartered in Cambridge, Massachusetts, the company utilizes its proprietary StitchMabs and common light-chain platforms to discover and create multispecific antibody combinations. Its lead product candidate, CTX-471, is an agonistic monoclonal antibody targeting CD137, currently undergoing Phase 1 clinical trials in patients with prior PD-1/PD-L1 checkpoint inhibitor treatment. Another significant candidate, CTX-009, is a bispecific antibody that inhibits DLL4-mediated Notch signaling and VEGF-A signaling, having completed a Phase 1 dose escalation study. Additionally, CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, is in the IND-enabling stage. Through its innovative approaches, Compass Therapeutics aims to enhance immune engagement and improve therapeutic outcomes for cancer patients.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Elektrofi is a preclinical-stage company based in Boston, Massachusetts, focused on revolutionizing drug delivery to enhance patient experiences. Founded in 2016, Elektrofi specializes in a formulation system capable of processing a broad spectrum of therapeutic molecules, including large and complex proteins such as monoclonal antibodies, bispecifics, and fusion proteins. The company's technology aims to improve subcutaneous administration, enabling drug manufacturers to create innovative therapies that offer patients greater control over how they receive life-changing medicines.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

Ceptur Therapeutics is focused on developing innovative genetic medicines for patients with unmet medical needs. The company utilizes its proprietary U1 Adaptor technology to achieve non-genotoxic, long-lasting silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and has potential applications in cancer treatment, allowing healthcare providers to offer new therapeutic options for various cancer types. Through its advancements, Ceptur aims to improve patient outcomes by providing effective solutions for difficult-to-treat conditions.

Enliven Therapeutics is a clinical-stage precision oncology company based in Boulder, Colorado, focused on developing innovative small molecule therapies to enhance patient outcomes. Established in July 2019 by Sam Kintz, Joe Lyssikatos, and Anish Patel, the company employs a unique discovery process that leverages clinically validated biological targets alongside advanced chemistry. This methodology aims to overcome the limitations of current therapies by creating potentially first-in-class treatments that address critical challenges such as tolerability, combinability, resistance, and disease progression, particularly in cases involving brain metastases. Enliven's product pipeline includes ELVN-001, a selective small molecule kinase inhibitor targeting the BCR-ABL gene fusion in chronic myeloid leukemia, and ELVN-002, an irreversible HER2 inhibitor designed to penetrate the central nervous system and act against various HER2 variants.

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.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Shoreline Biosciences, Inc. is a biotechnology company based in La Jolla, California, founded in 2020. The company specializes in developing cell-based immunotherapies aimed at treating seriously ill patients. Shoreline's proprietary technology platform leverages advanced methods in induced pluripotent stem cell (iPSC) differentiation and genetic programming, specifically targeting the IL-15/CISH pathway. This approach enhances the metabolic fitness and persistence of engineered natural killer (NK) cells, improving their anti-cancer activity. Shoreline's focus includes creating clinically effective immunotherapies using iPSC-derived NK cells and macrophages, providing innovative and cost-effective treatment options for oncology and other serious health conditions.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Attralus, Inc. is a biopharmaceutical company dedicated to addressing the systemic nature of amyloidosis and enhancing the quality of life for affected patients. Incorporated in 2010 and headquartered in South San Francisco, California, Attralus develops proprietary peptide-based pan-amyloid targeting agents aimed at diagnosing and treating various forms and stages of systemic amyloidosis. Its product pipeline includes AT-01, a polybasic peptide radiotracer for diagnostic and monitoring purposes; AT-02, a peptide-immunoglobulin fusion for therapeutic use; and AT-03, an Fc-fusion protein designed for therapeutic applications. By targeting the common pathology present in all systemic amyloidosis diseases, Attralus seeks to create effective treatments for all subtypes, particularly those that currently lack available treatment options. The company was previously known as Aurora Bio, Inc., before rebranding in August 2020.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. Founded in 1999, the company has developed advanced technologies such as SOMAmers, slow off-rate modified aptamers that specifically bind to target proteins, and the SOMAscan platform, which facilitates comprehensive protein measurement. This innovative approach allows for the analysis of protein biomarker signatures, significantly enhancing the understanding of health and disease beyond traditional genomic methods. SomaLogic's products are utilized in a variety of applications, including diagnostics for conditions in oncology, neurology, and cardiovascular and metabolic diseases, as well as in drug discovery and development. The company also offers diagnostic tests and protein measurement solutions for life science and biopharma researchers, contributing to advancements in medical science.

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.

DICE Therapeutics is a biopharmaceutical company dedicated to developing novel oral therapeutic candidates aimed at treating chronic diseases, particularly in immunology. Utilizing its proprietary DELSCAPE technology platform, DICE focuses on creating selective oral small molecules that can effectively modulate protein-protein interactions, similar to the efficacy of existing systemic biologics. The company's lead candidate, S011806, targets interleukin-17 (IL-17), a key pro-inflammatory molecule associated with various immunological conditions. Additionally, DICE is advancing oral therapies aimed at α4ß7 integrin and αVß1/αVß6 integrin, which are intended for the treatment of inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively. Through its innovative approach, DICE Therapeutics aims to bring effective oral treatments to patients suffering from chronic inflammatory diseases.

Tango Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, is a biotechnology company focused on developing innovative cancer therapies. The company employs the genetic principle of synthetic lethality to identify and target driver genes in cancer cells. Tango Therapeutics concentrates its efforts on three key areas: counteracting tumor suppressor gene loss, reversing immune evasion by cancer cells, and discovering novel drug combinations for enhanced efficacy. Its pipeline includes TNG908, a precision therapy targeting MTAP-deleted cancers, and several other discovery programs aimed at various cancer types with limited treatment options.

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.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

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.

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 clinical stage biotechnology company specializing in the development of targeted therapies aimed at clinically validated kinase targets in cancer. Focusing on small molecules, Nuvalent employs a structure-based design approach to create treatments that possess high selectivity, intending to address the limitations of existing therapies by overcoming resistance and minimizing side effects. The company is advancing a promising pipeline that includes lead programs targeting ROS1-positive and ALK-positive non-small cell lung cancer (NSCLC). Additionally, Nuvalent is engaged in various discovery-stage research initiatives that aim to expand its therapeutic offerings.

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 is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at treating cancer. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux aims to generate targeted immune responses that effectively combat tumors while minimizing effects on healthy tissue. The company's approach seeks to enhance all three stages of the anti-tumor immune response, making it a promising player in the field of cancer treatment.

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, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines utilizing its proprietary virus-like particle (VLP) platform technology. This innovative approach allows for the multivalent, particle-based display of complex viral antigens, which is aimed at generating broad and durable immune responses. Icosavax focuses primarily on life-threatening respiratory diseases, with its lead vaccine candidate, IVX-121, targeting respiratory syncytial virus (RSV) in older adults. The company's pipeline also includes candidates for human metapneumovirus (hMPV) and SARS-CoV-2, reflecting its commitment to advancing vaccine solutions for infectious diseases.

Tyra Biosciences, Inc. is a clinical-stage biotechnology company based in Carlsbad, California, dedicated to developing precision oncology therapies targeted at overcoming drug resistance in cancer treatment. Founded in 2018, the company utilizes its proprietary SNÅP platform to facilitate rapid and precise drug design by generating iterative molecular snapshots. This innovative approach allows Tyra to create next-generation therapies specifically aimed at addressing acquired resistance in tumors. The company's primary focus is on developing selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. Tyra's lead product candidate, TYRA-300, is designed to selectively inhibit FGFR3, initially targeting patients with metastatic urothelial carcinoma of the bladder and urinary tract.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

Amunix Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to discovering and developing innovative protein and peptide therapeutics aimed at treating cancer. The company employs several proprietary platforms, including XTEN for extending drug half-life, XPAT for analyzing protease activity in the tumor microenvironment, and ProTIA, which focuses on immuno-oncology therapeutics. Amunix's product pipeline includes prodrugs and transformative T-cell engagers designed to activate the immune system against solid tumors. The company collaborates with various academic, biotechnology, and pharmaceutical sectors, maintaining strategic partnerships with organizations such as Janssen and Biogen-Idec. Founded in 2006 and headquartered in Mountain View, California, Amunix strives to deliver breakthroughs that can harness the immune system to combat cancer more effectively.

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.

Artiva Biotherapeutics, Inc. is a San Diego-based biotechnology company focused on developing and manufacturing cellular immunotherapies for cancer treatment. Founded in 2019, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating both hematologic cancers and solid tumors. The company's product pipeline includes therapies that target CD20 and CD19 in B-cell lymphomas, as well as HER2 in various solid tumors. Artiva is committed to harnessing the therapeutic potential of NK cells while addressing challenges in their scalability and manufacturing. Its mission centers on delivering effective and safe cell therapies that are readily accessible to cancer patients in need.

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 clinical-stage biopharmaceutical company focused on innovating the drug development process through an asset-centric research and development model. The company manages a diverse portfolio of therapeutics across several therapeutic areas, including SerpinPC for Hemophilia A and B, LB101 for solid tumors, ORX750 for Narcolepsy Type 1 and other sleep disorders, and MGX292 for pulmonary arterial hypertension. Each therapeutic program is developed by a dedicated subsidiary, leveraging a centralized infrastructure and experienced management team to ensure efficient advancement of its pipeline assets.

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.

HemoShear Therapeutics, LLC is a biopharmaceutical company focused on discovering and developing novel treatments for rare metabolic disorders, particularly Organic Acidemias and Nonalcoholic Steatohepatitis. Founded in 2008 and based in Charlottesville, Virginia, the company utilizes its proprietary REVEAL-Tx platform to create human-relevant systems that accurately replicate the biology of organ systems and diseases, thereby providing valuable insights into human disease mechanisms. HemoShear’s approach generates meaningful human response data that aids in reducing the risk of drug development failures and enhances the identification of effective therapies. The company collaborates strategically with pharmaceutical, biotechnology, and medical device organizations to accelerate drug discovery and improve patient outcomes in the realm of metabolic health.

Day One Biopharmaceuticals is a clinical-stage biotechnology company dedicated to developing targeted cancer therapies for patients of all ages, with a particular emphasis on pediatric patients who have historically been underserved in cancer treatment advancements. The company is committed to addressing the unique needs of children and adults facing cancer by creating therapies that are informed by the biology of childhood cancers. Day One Biopharmaceuticals aims to bring promising treatments rapidly to market, focusing on those that can benefit both younger and older patients. The company's lead product candidate, DAY101, is an oral, brain-penetrant pan-RAF kinase inhibitor designed to target genetically defined cancers. By licensing and acquiring innovative products from research institutions and other biopharmaceutical companies, Day One Biopharmaceuticals seeks to provide effective treatment options for families confronted with the challenges of a cancer diagnosis.

Shoreline Biosciences, Inc. is a biotechnology company based in La Jolla, California, founded in 2020. The company specializes in developing cell-based immunotherapies aimed at treating seriously ill patients. Shoreline's proprietary technology platform leverages advanced methods in induced pluripotent stem cell (iPSC) differentiation and genetic programming, specifically targeting the IL-15/CISH pathway. This approach enhances the metabolic fitness and persistence of engineered natural killer (NK) cells, improving their anti-cancer activity. Shoreline's focus includes creating clinically effective immunotherapies using iPSC-derived NK cells and macrophages, providing innovative and cost-effective treatment options for oncology and other serious health conditions.

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.

Scorpion Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on advancing precision oncology for cancer treatment. Founded in 2020, the company specializes in developing targeted small-molecule drugs that address validated cancer targets, as well as previously undruggable and novel targets. Scorpion Therapeutics utilizes an integrated approach that combines target discovery, medicinal chemistry, and translational medicine to create a diverse pipeline of optimized compounds. By leveraging its precision medicine platform, the company aims to enhance the efficacy of cancer therapies and expand treatment options for patients, ultimately striving to overcome the limitations of existing cancer treatments.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Biomea Fusion is a privately held biopharmaceutical company focused on developing precision medicines for cancer patients. The company targets specific gene alterations that drive tumor growth, aiming to create more effective therapies. Its lead program focuses on inhibiting the interaction between menin and the MLL complex, which is crucial in various tumors. Biomea Fusion's goal is to expedite the development process of these potentially groundbreaking medicines to deliver impactful treatments for patients with genetically defined cancers.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. Founded in 1999, the company has developed advanced technologies such as SOMAmers, slow off-rate modified aptamers that specifically bind to target proteins, and the SOMAscan platform, which facilitates comprehensive protein measurement. This innovative approach allows for the analysis of protein biomarker signatures, significantly enhancing the understanding of health and disease beyond traditional genomic methods. SomaLogic's products are utilized in a variety of applications, including diagnostics for conditions in oncology, neurology, and cardiovascular and metabolic diseases, as well as in drug discovery and development. The company also offers diagnostic tests and protein measurement solutions for life science and biopharma researchers, contributing to advancements in medical science.

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

Elevation Oncology, Inc. is a biotechnology company dedicated to creating precision medicines for patients with genomically defined cancers. Founded in 2019 and headquartered in New York, it specializes in developing targeted therapeutics that inhibit specific genetic alterations known to drive cancer progression. The company's lead candidate, seribantumab, aims to address tumors associated with NRG1 fusions, which are rare genomic changes that can be effectively targeted through HER3 inhibition. Currently, seribantumab is undergoing clinical evaluation in the Phase 2 CRESTONE study, which focuses on patients with tumors of any origin exhibiting NRG1 fusions. Elevation Oncology is committed to making genomic testing actionable by advancing innovative treatments tailored to the unique genetic profiles of cancer patients.

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 focused on developing small-molecule therapeutics to address severe diseases, particularly fibrosis and cancer. Established in 2011, Galecto builds on over a decade of research into the roles of galectin-3 and lysyl oxidase-like 2 (LOXL2) in fibrotic diseases. The company's lead product candidate, GB0139, is an inhaled galectin-3 inhibitor specifically designed for treating severe fibrotic lung diseases, such as idiopathic pulmonary fibrosis (IPF), where there is a significant unmet medical need. Additionally, Galecto is advancing GB1211, an oral selective galectin-3 inhibitor aimed at addressing fibrosis related to non-alcoholic steatohepatitis. With a robust patent portfolio, Galecto leverages its unique therapeutic platform to target the underlying biological processes associated with fibrosis and related diseases.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.