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.

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, led by a team of experienced biopharmaceutical executives aiming to address significant unmet medical needs in this field.

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.

RAPT Therapeutics is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing orally delivered small-molecule therapies for oncology and inflammatory diseases. It uses a proprietary discovery and development engine to create highly selective compounds that modulate immune responses. The company's lead oncology candidate, FLX475, targets the C-C motif chemokine receptor 4 and is in a Phase 1/2 program as monotherapy and in combination with pembrolizumab. The lead inflammation candidate, RPT193, is designed to selectively inhibit migration of type 2 T helper cells into inflamed tissues. Additional targets such as general control nonderepressible 2 and hematopoietic progenitor kinase 1 are in discovery. Founded in 2015 and headquartered in South San Francisco, the company aims to address unmet medical needs in cancer and inflammatory diseases.

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

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

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

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

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

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

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.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies to transform the treatment of inflammatory bowel disease (IBD).

BioAge Labs develops medicines to treat aging and age-related diseases using its proprietary BioAge platform, which combines systems biology and AI. Its pipeline includes BGE-117 for multiple aging-related conditions, BGE-175 for immune aging and respiratory infections, and azelaprag for obesity in the elderly.

Basking Biosciences is developing a reversible thrombolytic therapy to restore blood flow to the brain during ischemic stroke, with the aim of preventing hemorrhage and long-term damage. The platform combines a primary agent that targets clot stability with a designed reversal agent that can be administered if bleeding occurs to neutralize the active compound and restore normal clotting, enabling treatment for acute ischemic stroke.

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

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company’s asset pipeline includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. Jixing Pharmaceuticals has in-licensed a small molecule cardiac myosin inhibitor, designed to address the specific needs of patients suffering from hypertrophic cardiomyopathy. By providing advanced treatment options, the company aims to meet the significant unmet medical needs of patients in China and beyond, particularly those facing serious, life-threatening health challenges in the fields of cardiology and ophthalmology.

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.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies to transform the treatment of inflammatory bowel disease (IBD).

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 is a clinical-stage biopharmaceutical company established in 2014, headquartered in Sioux Falls, South Dakota with additional offices in Cambridge, Massachusetts. The company specializes in developing immunotherapies using its proprietary DiversitAb platform, which employs transchromosomic cattle to produce large quantities of targeted human polyclonal antibodies without relying on human plasma or serum. SAB's pipeline includes therapies for conditions such as Type 1 diabetes (SAB-142), seasonal influenza (SAB-176), and Middle East Respiratory Syndrome Coronavirus (MERS-CoV, SAB-301).

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Abdera is an oncology company developing targeted alpha therapies for patients with relapsed, refractory, or metastatic cancers. Its precision oncology platform integrates drug development and antibody engineering technologies to create novel radiopharmaceuticals that specifically target tumors while minimizing systemic toxicities.

Milestone Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing etripamil, a rapid-onset calcium channel blocker for cardiovascular conditions. The lead product is etripamil, administered as a nasal spray for self-treatment of episodes of paroxysmal supraventricular tachycardia, with ongoing Phase III studies in the United States and Canada. The company is also advancing development for other cardiovascular indications, including atrial fibrillation and angina, with a Phase II program for atrial fibrillation with rapid ventricular response. Founded in 2003, Milestone is headquartered in Montreal.

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

OriCell Therapeutics is a biopharmaceutical company that specializes in the development of innovative cellular drugs aimed at addressing unmet clinical needs in tumor immunotherapy. The company focuses on creating therapeutics for a range of cancers, including liver cancer, myeloma, ovarian cancer, gastric cancer, cervical cancer, and non-small cell lung cancer. With a commitment to effective and affordable treatment options, OriCell Therapeutics aims to enhance patient care through the development of clinically valuable therapies, seeking to establish itself as a leader in the field of novel oncological treatments.

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 is a clinical-stage biopharmaceutical company developing antibody-based therapeutics targeting immunomodulatory receptors on immune effector cells. Its pipeline focuses on allergic, inflammatory, and proliferative diseases, with lead programs targeting Siglec-6 (AK006) and Siglec-8.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

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 developing novel therapies for inflammatory skin and joint diseases. Its flagship product, SLK, is an investigational Nanobody targeting IL-17A and IL-17F, showing potential for disease modification in dermatology and rheumatology patients.

Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for severe allergies and inflammation. The company is focused on advancing its lead product candidate, THB001, which is a first-in-class, highly selective oral inhibitor of the KIT receptor, a key regulator of mast cell function and survival. Mast cells play a crucial role in mediating inflammation in allergic diseases, making this targeted approach significant for treatment. Additionally, Third Harmonic Bio is developing THB335, another potent and selective oral small-molecule KIT inhibitor aimed at addressing various mast cell-driven inflammatory conditions affecting the skin, airway, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to provide new options for patients suffering from inflammatory diseases.

Acelyrin is a biopharmaceutical company dedicated to advancing treatments for diseases involving excessive immune system activation. It achieves this by discovering, acquiring, and expediting the development and market launch of innovative drug candidates.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these conditions.

Science 37 specializes in decentralized clinical trials, offering a technology-driven approach that enables patients to participate from their homes. The company's platform connects researchers with patients via telemedicine investigators and mobile nurses, accelerating trial enrollment and retention while reaching a more diverse patient population.

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

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

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

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2016, the company is dedicated to creating first-in-class therapies that selectively kill cancer cells by exploiting their reliance on DDR mechanisms. Artios’ product pipeline includes a range of promising candidates, such as a therapy that targets DNA polymerase theta, a protein involved in DNA repair processes, and another program focusing on a novel DDR target. The company collaborates with prominent research organizations, including Cancer Research Technology, to enhance its development efforts and leverage expertise in DNA repair. Artios is led by a skilled team with extensive experience in drug discovery, positioning it to advance its next-generation DDR programs and contribute significantly to the field of cancer treatment.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company’s asset pipeline includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. Jixing Pharmaceuticals has in-licensed a small molecule cardiac myosin inhibitor, designed to address the specific needs of patients suffering from hypertrophic cardiomyopathy. By providing advanced treatment options, the company aims to meet the significant unmet medical needs of patients in China and beyond, particularly those facing serious, life-threatening health challenges in the fields of cardiology and ophthalmology.

Alcyone Therapeutics is focused on advancing precision genetic therapies for central nervous system (CNS) disorders. The company develops innovative neural intervention technology aimed at addressing neurological conditions through targeted drug therapies. By integrating micro-fabrication technologies with advanced biomedical engineering, Alcyone Therapeutics seeks to enhance treatment options for patients suffering from chronic neuropathological conditions, including hydrocephalus. Their work aims to unlock new possibilities in CNS therapy development, addressing the unique challenges associated with treating these complex disorders.

GH Research is a clinical-stage biopharmaceutical company focused on developing therapies for psychiatric and neurological disorders, with an emphasis on treatment-resistant depression. It is advancing novel and proprietary mebufotenin (5-MeO-DMT) therapies, including inhalable and intravenous product candidates, to provide transformative treatment options for patients who do not respond to existing therapies. The portfolio includes GH001, an inhalable mebufotenin candidate, and GH002, an intravenous candidate. The company concentrates its activities in research and development to create practice-changing solutions for depression.

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 cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

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

Visus Therapeutics is a clinical-stage company developing innovative ophthalmic therapies to improve vision worldwide. Its primary focus is on creating eye drops for correcting presbyopia, with additional product candidates targeting ocular surface disease, glaucoma, and age-related macular degeneration.

Encoded Therapeutics is a biotechnology company focused on precision gene therapies to treat a broad range of severe genetic disorders. It develops therapies and a platform that identifies human genome sequences that regulate gene expression using screening and computational approaches. The company's pipeline targets conditions across multiple pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease, aiming to help medical practitioners treat patients and improve outcomes. Based in South San Francisco, California, Encoded Therapeutics was founded in 2014 and was previously known as Encoded Genomics.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

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 is a biopharmaceutical company developing targeted protein degradation therapies using its proprietary Degronimid platform. This platform enables the selective destruction of disease-causing proteins, expanding the scope of treatable conditions and potentially reducing drug resistance. The company focuses on oncology indications and has strategic collaborations with industry leaders.

Athira Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for brain disorders. Its lead product candidate, ATH-1017, aims to treat neurodegenerative diseases like Alzheimer's and Parkinson's by restoring neuronal health.

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

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 is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

Avadel Pharmaceuticals is a specialty pharmaceutical company focused on developing and marketing differentiated drug products. It operates internationally with headquarters in Dublin and a presence in St. Louis and Lyon. The portfolio centers on urology, central nervous system/sleep, and hospital products, including LUMRYZ, an extended-release formulation of sodium oxybate for narcolepsy-related cataplexy and excessive daytime sleepiness in adults. The company markets three sterile injectable medicines for hospital use: Akovaz for hypotension during anesthesia, Bloxiverz for reversal of non-depolarizing neuromuscular blocking agents after surgery, and Vazculep for treatment of hypotension. Avadel pursues new product development through formulation innovation and partnerships to improve patient adherence and outcomes.

Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, focused on developing innovative oral therapeutics for autoimmune diseases, particularly inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Founded in 2017, the company aims to address unmet medical needs through its unique approach to treatment, leveraging novel mechanisms of action. Its lead product candidate, NX-13, is a gut-selective NLRX1 agonist designed to modulate immune responses, and it is part of a broader pipeline that includes other internally discovered compounds targeting various pathways at the intersection of immunity and metabolism. Landos Biopharma's commitment to developing first-in-class therapeutics underscores its dedication to improving patient outcomes in the field of autoimmune disorders.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Encoded Therapeutics is a biotechnology company focused on precision gene therapies to treat a broad range of severe genetic disorders. It develops therapies and a platform that identifies human genome sequences that regulate gene expression using screening and computational approaches. The company's pipeline targets conditions across multiple pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease, aiming to help medical practitioners treat patients and improve outcomes. Based in South San Francisco, California, Encoded Therapeutics was founded in 2014 and was previously known as Encoded Genomics.

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 is a biopharmaceutical company focused on developing and commercializing etripamil, a rapid-onset calcium channel blocker for cardiovascular conditions. The lead product is etripamil, administered as a nasal spray for self-treatment of episodes of paroxysmal supraventricular tachycardia, with ongoing Phase III studies in the United States and Canada. The company is also advancing development for other cardiovascular indications, including atrial fibrillation and angina, with a Phase II program for atrial fibrillation with rapid ventricular response. Founded in 2003, Milestone is headquartered in Montreal.

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

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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.

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

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

REGENXBIO is a clinical-stage biotechnology company developing gene therapy product candidates using its proprietary NAV Technology Platform. This platform delivers genes to cells to address genetic defects or produce therapeutic proteins. REGENXBIO's pipeline includes RGX-314 for wet age-related macular degeneration, RGX-121 and RGX-111 for mucopolysaccharidoses, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II disease, and RGX-501 for homozygous familial hypercholesterolemia. The company also licenses its platform to other biopharmaceutical companies.

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 is a clinical-stage oncology-focused biopharmaceutical company that develops proteolytically-activated antibodies based on its Probody technology platform. Probody therapeutics are designed to be activated by the tumor microenvironment, enabling targeted cancer immunotherapies with reduced activity in healthy tissues. The company advances antibody and antibody-drug conjugate programs such as CX-072 (PD-L1), CX-2009 (CD166-targeted PDC), BMS-986249 (CTLA-4 Probody), CX-2029 (CD71-targeted PDC), and BMS-986288 (CTLA-4 Probody) in clinical research, and maintains collaborations with AbbVie, Amgen, Bristol-Myers Squibb, ImmunoGen, Pfizer, and Astellas to develop Probody therapeutics. Founded in 2008 and headquartered in South San Francisco, the company aims to improve the precision and safety of antibody-based therapies for cancer and inflammatory diseases.