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.

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It focuses on discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. RAPT's proprietary discovery engine has led to the advancement of two drug candidates: FLX475, which selectively inhibits the migration of immunosuppressive regulatory T cells into tumors, and RPT193, which targets type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short time frame since the company's inception. Additionally, RAPT is exploring further therapeutic targets, including general control nonderepressible 2 and hematopoietic progenitor kinase 1, which are currently in the discovery phase.

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 is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

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 for chronic inflammatory and autoimmune diseases. The company has a pipeline that includes three small molecule products. Its lead program, IMU-838, is a selective immune modulator that works by inhibiting the enzyme DHODH, targeting conditions such as multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. The second product, IMU-935, functions as an inverse agonist of the transcription factor RORγt and is being developed for psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Lastly, IMU-856 aims to restore intestinal barrier function and is intended for diseases associated with bowel barrier dysfunction. Through these innovative therapies, Immunic Therapeutics aims to address significant unmet medical needs in the field of immunology.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of therapies designed to degrade disease-causing proteins. The company’s lead product candidates include ARV-110, a proteolysis-targeting chimera (PROTAC) currently in phase I clinical trials for treating metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. Additionally, Arvinas is developing other PROTACs aimed at degrading various androgen receptor mutations and has initiatives focused on treating neurodegenerative diseases, including tauopathies. Arvinas collaborates with major pharmaceutical companies such as Pfizer, Genentech, Roche, and Bayer to advance its innovative therapeutic approaches. Founded in 2015, Arvinas is dedicated to improving the lives of patients with debilitating and life-threatening conditions through its unique protein degradation 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 develop drugs with good efficacy and affordable prices to satisfy the globally unmet clinical needs through innovations and strives to become the world-leading developer of novel drugs for tumor immunotherapy.

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.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies for chronic inflammatory and autoimmune diseases. The company has a pipeline that includes three small molecule products. Its lead program, IMU-838, is a selective immune modulator that works by inhibiting the enzyme DHODH, targeting conditions such as multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. The second product, IMU-935, functions as an inverse agonist of the transcription factor RORγt and is being developed for psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Lastly, IMU-856 aims to restore intestinal barrier function and is intended for diseases associated with bowel barrier dysfunction. Through these innovative therapies, Immunic Therapeutics aims to address significant unmet medical needs in the field of immunology.

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 is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company crating novel therapies, and unlocking the potential of Sonelokimab, the novel investigational Nanobody® for the treatment of inflammation, to revolutionize outcomes for patients.

ACELYRIN is a biopharma company focused on providing patients life-changing new treatment options by identifying, acquiring, and accelerating the development and commercialization of promising drug candidates and by leveraging its expertise to rapidly advance these medicines to patients.

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 is a biotechnology company dedicated to developing gene therapies aimed at treating rare pediatric diseases. The company's primary focus is on its lead program, a lentiviral-based gene therapy for Fanconi Anemia, a genetic disorder that leads to bone marrow failure. In addition to this, Rocket is advancing therapies for several other conditions, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis, all of which are currently in various stages of clinical and preclinical trials. The company is also engaged in an adeno-associated virus program targeting Danon disease, a serious multi-organ disorder. Rocket Pharmaceuticals collaborates with various academic and research institutions, enhancing its research capabilities and expanding its therapeutic portfolio. The company is based in New York, New York.

Neurogastrx, Inc. is a venture-stage biopharmaceutical company developing products for the treatment of gastrointestinal disorders.

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 privately held precision medicine company with a mission to revolutionize drug development to create more effective therapies for cancer patients. Biomea Fusion has a development portfolio that targets specific gene alterations which occur in the DNA of patients that translate into key drivers of tumor growth. It is Biomea’s goal to move these potentially breakthrough medicines swiftly through the development process and deliver highly impactful treatments for patients in need. The lead program targets the protein-to-protein interaction between menin and the MLL complex for the treatment of various tumors.

Nuance Biotech Inc. is a pharmaceutical development company based in Shanghai, China, founded in 2014. The company specializes in the research and development of various drug delivery methods, including capsules, injections, and tubes. In addition to its focus on drug development, Nuance Biotech offers a commercial and academic platform for pharmaceuticals and provides contract sales organization services to support its initiatives in the healthcare sector.

Prometheus Biosciences, formerly known as Prometheus Laboratories, is a specialty pharmaceutical company headquartered in San Diego, California. The company develops and commercializes innovative pharmaceutical and diagnostic products aimed at enhancing individualized patient care, particularly in the fields of gastroenterology and autoimmune diseases. Prometheus offers a range of diagnostic tests, including assays for monitoring drug and anti-drug antibody levels, as well as tests for inflammatory bowel disease and celiac disease. Among its marketed products are Entocort EC, indicated for Crohn's disease, and Imuran, used in kidney transplantation and rheumatoid arthritis management. Additionally, the company is involved in developing new treatments, such as COLAL-PRED, for gastrointestinal disorders. Through its advanced diagnostics and therapeutic offerings, Prometheus Biosciences assists healthcare providers in optimizing treatment strategies and improving patient outcomes.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

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 (Nasdaq:AVDL) is a specialty pharmaceutical company that seeks to develop differentiated pharmaceutical products that are safe, effective and easy to take through formulation development, by utilizing its proprietary drug delivery technology and through in-licensing / acquiring new products; ultimately, helping patients adhere to their prescribed medical treatment and see better results. Avadel’s current portfolio of products and product candidates focuses on the urology, central nervous system (CNS) / sleep, and hospital markets. The Company is headquartered in Dublin, Ireland with operations in St. Louis, Missouri and Lyon, France. For more information, please visit www.avadel.com.

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 is a developer of an oral first-in-class therapeutics designed to target autoimmune diseases.The company's therapeutics are novel, locally-acting small molecules targeting inflammatory bowel disease (IBD), crohn's disease (CD) and ulcerative colitis (UC), enabling healthcare providers to cure their patients.

Attune Pharmaceuticals is a private biotechnology company focused on discovering and developing novel orally-administered small-molecule therapeutics for the treatment of rare diseases.

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 the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

ARMO BioSciences, Inc. is an immuno-oncology company based in Redwood City, California, focused on developing biologic therapeutics that stimulate the immune system to combat cancer. The company's product pipeline includes several innovative candidates, such as AM0010, a long-acting form of interleukin-10 that enhances immune response, and AM0001, an anti-PD-1 monoclonal antibody targeting various cancers. Other key products in development include an anti-LAG-3 checkpoint inhibitor, AM0015, a recombinant form of interleukin-15, and AM0012, a recombinant interleukin-12. Established in 2010 and formerly known as Targenics, ARMO BioSciences aims to activate and enhance the body's immune response against tumors, thereby improving outcomes for cancer patients. As of mid-2018, it operates as a subsidiary of Eli Lilly and Company.

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.

CytomX Therapeutics is an oncology-focused biopharmaceutical company based in South San Francisco, California. The company specializes in developing proteolytically-activated antibody therapeutics using its proprietary Probody technology platform. This innovative approach aims to create cancer immunotherapies that enhance targeting of tumor cells while minimizing effects on healthy tissues. CytomX's clinical-stage product candidates include CX-072, which targets programmed cell death ligand 1; CX-2009, a drug conjugate against CD166; and BMS-986249, a CTLA-4 Probody therapeutic for metastatic melanoma, among others. The company has established strategic collaborations with several major pharmaceutical companies, including AbbVie, Amgen, and Bristol-Myers Squibb, to further the development of its Probody therapeutics. Founded in 2008, CytomX is dedicated to providing patients with more effective and less toxic treatment options for cancer and inflammatory diseases.