RTW Investments

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.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

Windward Bio is a clinical-stage biopharmaceutical company dedicated to enhancing outcomes for patients with advanced immunological diseases, focusing on severe respiratory conditions. The company is advancing a fully human monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into Phase 2 clinical trials. This antibody, administered subcutaneously, potently binds to the TSLP ligand, extending its half-life and silencing its effector functions. Windward Bio is also developing innovative bispecific programs for other immunological disorders, aiming to address significant unmet medical needs in these areas. Led by an experienced team of biopharmaceutical executives, the company is committed to accelerating drug development to improve the lives of patients.

Alesta Therapeutics is engaged in developing innovative therapies aimed at treating cancer by targeting chronic stress response pathways. The company focuses on small molecule biotech programs that address synthetic lethal metabolic dependencies essential for cancer cell survival. By targeting specific control mechanisms involved in these pathways, Alesta Therapeutics aims to provide new therapeutic approaches that can effectively disrupt the growth of cancer cells, thereby enhancing treatment options for patients.

RAPT Therapeutics, Inc., established in 2015 and headquartered in South San Francisco, California, is a clinical-stage biopharmaceutical company specializing in immunology-based therapies. The company focuses on developing oral small molecule treatments for patients with unmet needs in oncology and inflammatory diseases. Its lead drug candidates are FLX475, an oral C-C motif chemokine receptor 4 antagonist in Phase 2 clinical trials for various cancer types, and RPT193, which selectively inhibits type 2 T helper cell migration into inflamed tissues. Additionally, RAPT is exploring other targets like general control nonderepressible 2 and hematopoietic progenitor kinase 1 in the discovery phase.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

Ottimo Pharma specializes in developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that targets both immune checkpoint inhibition and angiogenesis, two key pathways involved in cancer growth. By simultaneously addressing these pathways, Ottimo Pharma's approach offers healthcare professionals a dual-pronged strategy to potentially enhance treatment outcomes and reduce the burden of cancer on patients and the healthcare system.

Crescent Biopharma is a biotechnology firm focused on developing novel precision-engineered medicines targeting verified biology to improve care for patients with solid tumors.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

Obsidian Therapeutics develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies. It focuses on addressing vision-related issues, particularly presbyopia, which affects the near vision of individuals, especially those over the age of 45. The company's primary product is an aceclidine-based eye drop designed to restore near vision loss associated with this condition. Through its advancements in ophthalmic pharmaceuticals, LENZ Therapeutics aims to improve the quality of life for those impacted by vision challenges.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

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.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

SAB Biotherapeutics, Inc. is a clinical-stage biopharmaceutical development company based in Sioux Falls, South Dakota, with an additional office in Cambridge, Massachusetts. Founded in 2014, the company focuses on developing immunotherapies for various human diseases using its proprietary DiversitAb platform. This innovative platform enables the rapid production of fully targeted human polyclonal antibodies from genetically engineered transchromosomic cattle, eliminating the need for human plasma or serum. SAB Biotherapeutics' product pipeline includes SAB-142 for Type 1 diabetes, SAB-176 for seasonal influenza, and SAB-301 for Middle East Respiratory Syndrome Coronavirus (MERS-CoV). The company's approach aims to address public health challenges, rare diseases, and pandemic threats through advanced antibody science.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Abdera Therapeutics is an oncology company focused on developing targeted alpha therapies (TATs) for patients suffering from relapsed, refractory, and metastatic cancers. The company specializes in targeted radiotherapies that employ purpose-built vectors to deliver high-energy radioisotopes directly to tumors and metastatic lesions. This innovative approach aims to provide more effective treatment options for patients, capitalizing on the significant therapeutic and commercial potential of TATs. With the nuclear medicine market projected to grow substantially, Abdera's targeted therapies are positioned to play a crucial role in the evolving landscape of cancer treatment.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

OriCell Therapeutics is a biopharmaceutical company dedicated to developing innovative, effective, and affordable tumor immunotherapy treatments. It focuses on unmet clinical needs, with a pipeline targeting various cancers including liver, myeloma, ovarian, gastric, cervical, and non-small cell lung cancers.

Apogee Therapeutics is a biotechnology company based in San Francisco, California, founded in 2022. The company focuses on developing differentiated biologics for the treatment of immunological and inflammatory disorders, specifically targeting atopic dermatitis and chronic obstructive pulmonary disease. Apogee Therapeutics aims to address high unmet medical needs by advancing novel therapies that utilize advanced antibody engineering to enhance therapeutic properties such as half-life. The company's key programs, APG777 and APG808, are designed to leverage established mechanisms of action to improve treatment outcomes for patients suffering from these conditions.

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.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for immunologic diseases. The company specializes in leveraging Nanobody technology to create new treatments aimed at addressing inflammatory conditions, particularly in the skin and joints. Its lead investigational product, Sonelokimab, represents a novel approach to managing inflammation, with the potential to significantly improve patient outcomes. Through its research and development efforts, MoonLake aims to advance the understanding and treatment of various inflammatory diseases, contributing to the evolving landscape of biopharmaceuticals.

Acelyrin is a late-stage clinical biopharmaceutical company dedicated to advancing new treatment options for patients. The company specializes in identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. By leveraging its expertise, Acelyrin aims to rapidly bring transformative medicines to market, ultimately improving patient outcomes and quality of life.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly ABI-009, aimed at treating patients with alterations in the TSC1 or TSC2 genes, where existing mTOR inhibitors have limitations in pharmacology, drug delivery, or safety. Aadi's primary focus is on providing transformational therapies for patients with ultra-rare cancers, such as perivascular epithelioid cell tumors (PEComa). Since its founding in 2011, Aadi Bioscience has aimed to address significant unmet medical needs in oncology and related therapeutic areas.

Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia, Pennsylvania, dedicated to developing gene therapies for cardiovascular diseases. Founded in 2013, the company focuses on innovative treatments for dilated cardiomyopathy (DCM), particularly cases caused by mutations in the BAG3 gene. This condition affects over 3 million individuals in the United States, with around 35,000 patients estimated to have BAG3 mutations, classifying it as an orphan disease. Patients with BAG3-associated DCM, often younger and experiencing faster disease progression, currently have limited treatment options, primarily consisting of standard heart failure care. Renovacor's lead program involves a recombinant adeno-associated virus (AAV)-based gene therapy aimed at replacing the defective BAG3 gene, which holds the potential to prevent disease progression and improve outcomes for this vulnerable patient population.

Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing innovative gene therapies for rare and severe pediatric diseases. The company specializes in lentiviral vector and adeno-associated virus platforms to target genetic disorders. Its lead program focuses on treating Fanconi Anemia, a serious condition that leads to bone marrow failure. In addition to this, Rocket is actively researching therapies for several other bone marrow-derived disorders, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis. Furthermore, the company is developing an AAV-based gene therapy for Danon Disease, a multi-organ disorder associated with increased risk of cardiovascular complications. To advance its research, Rocket Pharmaceuticals maintains numerous collaborations and license agreements with prestigious institutions, enhancing its capabilities in gene therapy development. The company is headquartered in New York, New York.

Neurogastrx, Inc. is a venture-stage biopharmaceutical company focused on developing innovative therapies for gastrointestinal disorders. Established in 2017 and located in Campbell, California, the company aims to address the unmet medical needs associated with functional and motility disorders of the gastrointestinal tract. One of its key products, NG101, is designed to enhance gastric motility while also possessing antiemetic properties. Neurogastrx's specialty therapies target the enteric nervous system, addressing issues related to gut contractions, sensation, and the brain-gut axis, ultimately striving to alleviate the burden of these disorders on patients and the healthcare system.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

UroGen Pharma Ltd. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, focused on developing innovative therapies for specialty cancers and urologic diseases. The company is dedicated to transforming the standard of care in uro-oncology through its diverse pipeline of product candidates. Its lead products, including UGN-101 and UGN-102, aim to ablate tumors non-surgically and address various forms of non-muscle invasive urothelial cancer, such as low-grade upper tract urothelial carcinoma and low-grade non-muscle invasive bladder cancer. UroGen is also advancing UGN-201, an immunotherapy candidate targeting high-grade non-muscle invasive bladder cancer. Furthermore, the company's proprietary RTGel technology enhances drug delivery by enabling sustained release, improving therapeutic efficacy. UroGen has established licensing agreements with Allergan Pharmaceuticals for the development of RTGel-based products and with Agenus Inc. to commercialize treatments for urinary tract cancers. Founded in 2004, UroGen Pharma continues to innovate in the field of urology and oncology.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Biomea Fusion is a preclinical-stage biopharmaceutical company dedicated to advancing precision medicine for cancer treatment. The company's focus is on the discovery and development of irreversible small molecule drugs that target specific genetic alterations associated with tumor growth. Its lead product candidate, BMF-219, is designed as a potent and selective irreversible inhibitor of menin, a key transcriptional regulator involved in oncogenic signaling across various cancers. Biomea Fusion aims to expedite the development of these innovative therapies to provide highly effective treatment options for patients with genetically defined cancers.

Nuance Biotech Inc. is a pharmaceutical development company based in Shanghai, China, founded in 2014. The company focuses on the research and development of innovative drug formulations, offering products in the form of capsules, injections, and tubes. In addition to its core pharmaceutical development activities, Nuance Biotech also provides commercial and academic platforms for drug-related ventures and offers contract sales organization services. Through its diverse capabilities, the company aims to advance healthcare solutions and contribute to the pharmaceutical industry.

Prometheus Biosciences, established in 1995 and headquartered in San Diego, California, specializes in developing and commercializing innovative pharmaceutical and diagnostic products. The company's portfolio includes tests that enable physicians to provide personalized patient care, such as monitoring drug and anti-drug antibody levels, diagnosing and prognosticating inflammatory bowel disease (IBD), and detecting celiac disease and lactose intolerance. Prometheus also offers therapeutic products like Entocort EC for treating Crohn's disease and has agreements to develop and market other drugs. The company's commitment to innovation positions it as a leader in precision healthcare, particularly in gastroenterology.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

Avadel Pharmaceuticals plc is a specialty pharmaceutical company based in Dublin, Ireland, with operations in the United States and France. The company focuses on developing and commercializing innovative pharmaceutical products, particularly in the fields of urology, central nervous system disorders, and hospital settings. Avadel's key product, FT218, is in Phase 3 clinical trials for treating narcolepsy-related excessive daytime sleepiness and cataplexy. Additionally, Avadel markets several sterile injectable drugs for hospital use, including Akovaz for hypotension during anesthesia, Bloxiverz for reversing neuromuscular blockade effects, and Vazculep for treating hypotension. The company also offers LUMRYZ, an extended-release formulation of sodium oxybate for narcolepsy management. Avadel Pharmaceuticals was incorporated in 2015 and was previously known as Flamel Technologies SA before rebranding in January 2017.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, specializing in the development of oral therapeutics for autoimmune diseases. Founded in 2017, the company focuses on innovative treatments for conditions such as inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Its lead product candidate, NX-13, is a novel, gut-selective agonist targeting the NLRX1 pathway. The company also has a diverse pipeline that includes other internally discovered compounds aimed at novel immunometabolic pathways. By developing first-in-class therapeutics, Landos Biopharma seeks to provide effective treatment options for patients suffering from autoimmune conditions.

Attune Pharmaceuticals, Inc. is a biotechnology research and development company based in New York, established in 2015. The company specializes in discovering and developing novel orally administered small molecule therapeutics aimed at treating rare diseases. Its focus includes the creation of heterocyclic derivative compounds and pharmaceutical compositions that address conditions such as hereditary angioedema, thereby providing clinicians with effective treatment options for rare medical conditions.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

ARMO BioSciences is a biotechnology company based in Redwood City, California, focused on developing immune modulatory biologic therapeutics. The company specializes in immuno-oncology, aiming to activate the immune system of cancer patients to recognize and eradicate tumors. Its pipeline includes several product candidates: AM0010, a long-acting form of interleukin 10 (IL-10) that stimulates the immune system; AM0001, an anti-PD-1 monoclonal antibody; AM0003, an anti-LAG-3 checkpoint inhibitor; AM0015, a form of recombinant human interleukin-15 (IL-15); and AM0012, a form of recombinant human interleukin-12 (IL-12). The company was incorporated in 2010 and formerly known as Targenics.

AveXis, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

Verastem, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative treatments aimed at improving the survival and quality of life for cancer patients. Based in Needham, Massachusetts, Verastem markets COPIKTRA (duvelisib), an oral medication indicated for adult patients with relapsed or refractory chronic lymphocytic leukemia and follicular lymphoma after multiple prior therapies. In addition to this approved therapy, the company is advancing its pipeline with the focal adhesion kinase inhibitor defactinib, which is being studied in combination with immunotherapies for various cancers, including pancreatic and ovarian cancers, as well as non-small cell lung cancer. Verastem's research emphasizes inhibiting critical signaling pathways that promote cancer cell survival and tumor growth, particularly those associated with the RAS/MAPK pathway. The company has formed collaborative agreements with Yakult Honsha Co., Ltd. and CSPC Pharmaceutical Group Limited to enhance its development efforts. Founded in 2010, Verastem continues to focus on late-stage development of its anticancer agents.

CytomX Therapeutics, Inc. is an oncology-focused biopharmaceutical company based in South San Francisco, California. It specializes in developing a novel class of antibody therapeutics utilizing its Probody technology platform, which aims to enhance the targeting of tumors while minimizing drug activity in healthy tissues. The company's clinical-stage product candidates include CX-072, a therapeutic targeting programmed cell death ligand 1; CX-2009, a drug conjugate targeting CD166; BMS-986249 and BMS-986288, both targeting CTLA-4 for metastatic melanoma and solid tumors; and CX-2029, a drug conjugate for CD71 in solid tumors. CytomX collaborates strategically with several industry leaders, including AbbVie, Amgen, and Bristol-Myers Squibb, to advance its Probody therapeutics. Founded in 2008, CytomX Therapeutics is committed to providing innovative and less toxic treatment options for patients with severe illnesses such as cancer.